[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Chiesi Farmaceutici S.p.A.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":368},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,15,0,[8,45,68,91,118,142,167,188,210,233,267,285,312,331,355],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":27,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":4},"100644137",false,"NCT07666490","BETRI-Prospective: Asthma Control in Single Inhaler ICS\u002FLABA\u002FLAMA (87\u002F5\u002F9 pMDI [BDP\u002FFF\u002FGB - 100\u002F6\u002F12.5 μg]) vs Single Inhaler ICS\u002FLABA (200\u002F6 pMDI [BDP\u002FFF - 200\u002F6 μg])","A Pragmatic, Phase IV, Randomized, Open-label, Multinational, Multicentre, 2-arm Parallel Group, Prospective Study Comparing Efficacy and Safety of Single Inhaler ICS\u002FLABA\u002FLAMA (Beclometasone\u002FFormoterol Fumarate\u002FGlycopyrronium Bromide [87\u002F5\u002F9 pMDI { BD\u002FFF\u002FGB -100\u002F6\u002F12.5 μg} ]) vs Single Inhaler ICS\u002FLABA (Beclometasone Dipropionate Plus Formoterol Fumarate [200\u002F6 pMDI { BD\u002FFF - 200\u002F6 μg} ]) in Asthma Subjects","BETRI","Inclusion Criteria:\n\n1 . Study participant's written informed consent obtained prior to any study related procedure.\n\n2\\. Male or female study participants aged ≥18 years old. 3. Physician confirmed documented asthma diagnosis as per current clinical practice.\n\n4\\. Stable asthma treatment prior randomisation: At least 3 months on any medium strength (MS) ICS\u002FLABA regular treatment.\n\n5\\. Poor asthma control at randomisation (ACQ-5 ≥1.5 and at least 1 exacerbation in the previous year).\n\n6\\. Women with childbearing potential (WOCBP) and with fertile male partners: they and\u002For their partner must be willing to use a highly effective birth control method from the signature of the informed consent and until the last visit.\n\n7\\. Study participants must have a cooperative attitude and the ability to be trained to use the patient app correctly, to be able to perform the required outcomes measurements (e.g. ePRO completion) and the ability to understand the risks involved.\n\nNote: Study participants with asthma diagnosis and no clinically relevant concurrent COPD diagnosis upon clinical judgement can be included. Study participants on maintenance and reliever therapy (MART) can be included if the ICS dosage does not exceed 400 µg\u002Fday of beclometasone or equivalent.\n\nExclusion Criteria: The presence of any of the following will exclude a study participant from study enrolment:\n\n1. Participation in another interventional clinical trial.\n2. Pregnant or breastfeeding women at the moment of enrolment.\n3. Other chronic respiratory disease: Lung Cancer, known alpha1-antitrypsin deficiency, active tuberculosis, clinically significant bronchiectasis, interstitial lung disease, pulmonary hypertension, or any other uncontrolled\u002Fclinically significant diseases (according to investigator's judgement).\n4. Contraindication for LAMA use.\n5. Study participants on biological therapies for asthma.\n6. For France only: Individuals under court protection (including protected adults) and individuals not affiliated to a social security system are excluded from participation in this study (Country\u002FRegion-Specific Differences - France), in accordance with Articles L.1121-6, L.1121-8, and L.1121-8-1 of the French Public Health Code.","ALL","18 Years",{"count":19,"type":20},644,"ESTIMATED","INTERVENTIONAL",[23],"PHASE4","This study will evaluate the effect of triple ICS\u002FLAMA\u002FLABA therapy with a BDP\u002FFF\u002FGB 100\u002F6\u002F12.5 µg on asthma control outcomes relative to ICS\u002FLABA therapy with BDP\u002FFF 200\u002F6 μg in a population with asthma poorly controlled.",[26],"Asthma",[28,29,30,31,32],"asthma control","adults","pragmatic study","BDP\u002FFF\u002FGB","BDP\u002FFF","NOT_YET_RECRUITING","2026-06-25",{"date":36,"type":37},"2026-06-29","ACTUAL",{"date":39,"type":20},"2026-06-12",{"date":41,"type":20},"2028-06-13",{"name":43,"class":44},"Chiesi Farmaceutici S.p.A.","INDUSTRY",{"id":46,"slug":4,"hasResults":10,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":50,"eligibilityCriteria":51,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":52,"enrollmentInfo":53,"targetDuration":4,"studyType":21,"phases":55,"briefSummary":57,"conditions":58,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":67},"100616140","NCT07301736","Therapeutic Equivalence of CHF5993 pMDI 100\u002F6\u002F12.5 µg HFA-152a in Subjects With Mild to Moderate Asthma","A Phase II Multinational, Multicentre, Double-blind, Randomised, Active-controlled, 3-way Cross-over Study to Evaluate the Therapeutic Equivalence of CHF5993 pMDI 100\u002F6\u002F12.5 µg HFA-152a Versus CHF5993 pMDI 100\u002F6\u002F12.5 µg HFA-134a in Subjects With Mild to Moderate Asthma","TRECONY","Inclusion Criteria:\n\n* Male and female adults (18 ≤ age ≤ 75 years) with a diagnosis of Asthma for at least 6 months prior to screening and with diagnosis before the age of 50 years;\n* Non-smokers, ex-smokers;\n* Body mass index: within the range of 18.0 to 35.0 kg\u002Fm2 inclusive;\n* Stable asthma therapy: a stable maintenance treatment for at least 4 weeks prior to screening with:\n\n  1. low or medium doses of ICS (Inhaled Corticosteroids) alone; or\n  2. low or medium doses of ICS + LABA (Long-acting β2-agonist) (fixed or free combination).\n* Controlled or partly controlled based on an Asthma Control Questionnaire - 7 Items (ACQ-7) score \\\u003C1.5 at screening and at randomisation.\n* Pre-BD (Bronchodilator) FEV1 \\>40% and \\\u003C90% of the predicted normal value, after appropriate wash out from BDs, at the Screening Visit (V1).\n* A demonstrated increase in either FEV1 or forced vital capacity of \\>12% and \\>200 mL from baseline within 30 minutes (min) after inhalation of 400 µg salbutamol (i.e. albuterol) pMDI at the Screening Visit (V1).\n\nExclusion Criteria:\n\n* History of near fatal asthma or hospitalisation for asthma in intensive care unit, inpatient setting or emergency room access for asthma in the previous 6 months prior to screening, which in the judgement of the Investigator may place the subjects at undue risk;\n* Recent asthma exacerbation requiring systemic corticosteroids (SCSs), or emergency room admission or hospitalisation within 3 months prior to screening and\u002For during the run-in period ;\n* Non-persistent asthma: exercise-induced, seasonal asthma (as the only asthma-related diagnosis) not requiring daily asthma control medicine;\n* Asthma subjects currently treated with any of the following :\n\n  1. High dose ICS;\n  2. Long-acting muscarinic antagonist (LAMA);\n  3. Systemic, depot or slow-release corticosteroids within 12 weeks prior to screening;\n  4. Any other asthma treatments (e.g. cromolyn sodium, nedocromil sodium, leukotriene modifiers) within 4 weeks prior to screening;\n  5. Any biologic therapy (e.g. omalizumab, mepolizumab, reslizumab, benralizumab, dupilumab, tezepelumab) within 6 months prior to screening;\n* Respiratory disorders other than asthma\n* Lung resection;\n* Lower respiratory tract infection;\n* Lung cancer and history of lung cancer;\n* Subjects with active cancer or a history of cancer (other than lungs) ;\n* Patients who have clinically significant cardiovascular condition;\n* Run-in compliance: e-Diary completion \\\u003C75% and run-in treatment compliance \\\u003C75% at randomisation;","75 Years",{"count":54,"type":20},780,[56],"PHASE2","This study will compare an asthma inhaler that uses a new climate friendly alternative propellant to an asthma inhaler with an existing propellant. We want to make sure both versions of the inhaler work the same way for people with mild to moderate asthma.",[26],"RECRUITING","2026-06-24",{"date":34,"type":37},{"date":63,"type":37},"2025-12-17",{"date":65,"type":20},"2027-06-03",{"name":43,"class":44},167,{"id":69,"slug":4,"hasResults":10,"nctId":70,"briefTitle":71,"officialTitle":72,"acronym":73,"eligibilityCriteria":74,"healthyVolunteers":10,"sex":16,"minAge":75,"maxAge":76,"enrollmentInfo":77,"targetDuration":4,"studyType":21,"phases":79,"briefSummary":81,"conditions":82,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":84,"lastUpdatePostDateStruct":85,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":4},"100637229","NCT07577804","A Study to Evaluate the Efficacy and Safety of CHF10196 Tablets (Florensocatib) Compared With Placebo in Male and Female Participants 12 to 85 Years of Age With Bronchiectasis","A Phase III, Randomised, Double-blind, Two-arm Study to Investigate the Efficacy and Safety of Treatment With CHF10196 Tablets (Florensocatib) Compared With Placebo for up to 78 Weeks, Followed by an Open-label Extension, in Male and Female Participants 12 to 85 Years of Age With Bronchiectasis","FABLE","Inclusion Criteria for Main Study:\n\n1. Signed and dated informed consent obtained prior to any study-related procedure; Adolescents: Written informed consent obtained from the parent(s)\u002Flegally authorised representative(s) (according to the local regulation) and written or verbal assent by the participant (when appropriate), obtained prior to any study-related procedures;\n2. Adults: Male or female participant ≥18 and ≤85 years of age; Adolescents: Male or female participant ≥12 and \\\u003C18 years of age;\n3. Adults: BMI (body mass index) \\>18.5 kg\u002Fm² at screening; Adolescents: Body weight \\>30 kg at screening;\n4. Clinical history consistent with bronchiectasis (cough, chronic sputum production, and\u002For recurrent respiratory infections) confirmed by centralised reading of a chest CT, not more than 5 years old demonstrating bronchiectasis affecting 1 or more lobes; Note: If no recent chest CT is available, or if the records\u002Freport is not available, a new CT chest will be performed during the screening period;\n5. A history of at least 2 pulmonary exacerbations treated with antibiotics, or 1 pulmonary exacerbation treated with antibiotics with additional risk factors (SGRQ Symptoms score at screening \\>40) in the past 12 months prior to screening; Adolescents: A history of at least 1 pulmonary exacerbation in the 12 months prior to screening;\n6. Post-bronchodilator FEV1 ≥30% of the predicted value, calculated using the Global Lung Function Initiative 2012 reference equations; Note: in case post-bronchodilator spirometry quality is not judged satisfactory as per ATS\u002FERS acceptability and usability criteria\\*, it may be re-checked on the same day (after a sufficient period of rest) or once before randomisation if it cannot be performed on the same day;\n7. Participants may either be:\n\n   * on stable background therapy for at least 3 months prior to screening;\n   * not on background therapy at screening and not planning to start background therapy;\n8. Able to provide sputum at screening (adults only);\n9. Cooperative attitude and ability to read\u002Fwrite, perform all study-related procedures, and use electronic device, ability to understand the risks involved;\n10. If female, the participant must be of non-childbearing potential, surgically sterile, postmenopausal, or she and her partner must use adequate methods of contraception\\*\\* during the study and for 90 days after the last dose of study intervention. Participants must not donate eggs during the study and for 90 days after the last dose of IMP (investigational medicinal product); Note: In case of hormonal contraception, an additional non-hormonal method (barrier method, preferably male condom) is required. The status of adolescent females being not of childbearing potential at screening will be monitored at each further visit (except the Follow-up visit);\n11. If male, the participant and his partner must agree to use adequate methods of contraception\\*\\* during the study and for at least 90 days after the last dose of study intervention.\n\nInclusion Criteria for OLE (Extension) Study:\n\n1. Participant's written informed consent for the OLE Phase obtained prior to any study-related procedures of the OLE Phase; Adolescents: Written informed consent for the OLE Phase obtained from the parent(s)\u002Flegal representative(s) (according to the local regulation) and written or verbal assent by the participant (when appropriate), obtained prior to any study-related procedures of the OLE Phase;\n2. Participants (adults or adolescents) who completed the treatment period of the Main Phase\n3. Participants with study intervention compliance ≥70% during the Main Phase\n4. If female, the participant must be of non-childbearing potential, surgically sterile, postmenopausal, or she and her partner must use adequate methods of contraception\\* during the study and for 90 days after the last dose of study intervention. Participants must not donate eggs during the study and for 90 days after the last dose of study intervention; Note: In case of hormonal contraception, an additional non-hormonal method (barrier method, preferably male condom) is required. The status of adolescent females being not of childbearing potential at screening will be monitored at each further visit (except the Follow-up visit);\n5. If male, the participant and his partner must agree to use adequate methods of contraception\\* during the study and for at least 90 days after the last dose of study intervention. Participants must not donate sperm during the study and for 90 days after the last dose of study intervention.\n\nExclusion Criteria for Main Study:\n\n1. Participants who have experienced any degree of pulmonary exacerbation or are experiencing a pulmonary exacerbation within 4 weeks prior to screening or during the screening period; Note: Participants can be rescreened only after recovery and 4 weeks after the last dose of antibiotic treatment;\n2. Current diagnosis of CF (cystic fibrosis) as determined by Investigator;\n3. Known history of invasive opportunistic infections (such as but not limited to histoplasmosis, pneumocystosis, or aspergillosis) and\u002For abnormally frequent or prolonged infections suggesting an immunocompromised status judged incompatible with the introduction of a DPP1 inhibitor by the Investigator;\n4. Diagnosis of A1ATD -Alpha-1-antitrypsin deficiency- (defined as A1AT serum level \\\u003C110 mg\u002FdL) currently being treated with augmentation therapy; Note: A prior test result of A1AT serum level to confirm the diagnosis will be acceptable;\n5. Participants with a diagnosis of non-tuberculosis mycobacteria, pulmonary infection or TB (tuberculosis), or allergic bronchopulmonary aspergillosis currently being treated or requiring treatment as determined by the Investigator;\n6. Participants receiving a systemic immunosuppressive therapy for the treatment of an autoimmune disease;\n7. History of malignancy in the past 5 years (excluding cured basal cell carcinoma of the skin, carcinoma in situ and papillary thyroid carcinoma) or treatment of malignancy in the past 5 years;\n8. Have significant haemoptysis (≥300 mL or requiring blood transfusion) within 4 weeks prior to screening or during the screening period;\n9. Participants with a severe concomitant disease or disorder that, in the opinion of the Investigator, may put the participant at risk by participating in the study, or interfere with the participant's treatment, assessment, or influence the results of the study, or have compliance issues with the study. Examples include, but are not limited to, cardiovascular conditions (e.g. NYHA Class III or IV cardiac failure), pulmonary conditions (e.g. severe pulmonary fibrosis or lung transplantation), infectious diseases (e.g. severe COVID-19 infection), gastrointestinal conditions, hepatobiliary conditions (e.g. Child-Pugh class B or C), severe renal conditions (e.g. severe nephrotic syndrome), neurological conditions (e.g. demyelinating diseases), musculoskeletal, endocrine, metabolic or psychiatric conditions;\n10. Have elevated liver function test results (ALT or AST \\>2x ULN) or have a bilirubin \\>1.5x ULN at screening (isolated bilirubin \\>1.5x ULN is acceptable if bilirubin is fractionated and direct bilirubin \\\u003C35%);\n11. Have an abnormal renal function test result (eGFR \\\u003C60 mL\u002Fmin by CKD-EPI formula for adults or CKiD-U25 formula for adolescents) at screening\n12. Participants with an absolute blood neutrophil count \\\u003C1000\u002F mm3 at screening;\n13. Participants with a history of alcohol or drug abuse within 6 months prior to screening as per DSM V criteria;\n14. Clinically significant abnormal laboratory values at screening that, in the opinion of the Investigator, may put the participant at risk by participating in the study, or interfere with the participant's treatment or assessment, or influence the results of the study;\n15. Blood donation or blood loss (≥450 mL) less than 2 months prior to screening;\n16. Participants who currently smoke tobacco or who stopped smoking within 3 months prior to screening or who are not willing to abstain from smoking for the duration of the trial; Note: any form of smoking (cigarettes, e-cigarettes, vaping, cannabis) is forbidden during the whole trial\n17. Introduction of new chronic treatment for NCFBE - Non-cystic fibrosis bronchiectasis- (oral or inhaled antibiotics, ICS (inhaled corticosteroids), or low-dose oral steroids\\*), if started \\\u003C3 months prior to screening or during the screening period\n\n    \\*Low-dose oral steroids defined as \\\u003C10 mg\u002Fday of prednisone or equivalent\n18. Use of any immunomodulatory agents (including but not limited to: bortezomib, ixazomib, thalidomide, cyclophosphamide, mycophenolate, Janus kinase inhibitors, IFN-γ, and azathioprine) or chronic treatment with high-dose oral steroids\\* within 4 weeks prior to screening or during the screening period\n\n    \\*High-dose oral steroids defined as ≥10 mg\u002Fday of prednisone or equivalent\n19. Use of strong CYP3A inducers (including but not limited to: apalutamide, carbamazepine, enzalutamide, mitotane, phenytoin, rifampicin) within 4 weeks prior to screening or during the screening period\n20. Previous treatment with another DPP1 (Dipeptidyl peptidase 1) inhibitor within 6 months before screening or during the screening period Note: participants who were randomised and only received placebo in studies with DPP1 inhibitor are allowed;\n21. Participant expected to take prohibited concomitant medications during the study\n22. Received any live attenuated vaccine within 4 weeks prior to randomisation Note: Recent administration of a live vaccine should be checked at screening. If a live vaccine has been administered, the participant should wait for 4 weeks prior to being randomised;\n23. Participants with medical conditions associated with periodontal disease (to be evaluated by a periodontist or dentist), defined as any tooth that can potentially cause pain or infection as noted in the oral exam unless they are corrected before randomisation visit (e.g. pulp necrosis); severe periodontal disease defined as having pocket depth measurements ≥6 mm on ≥2 teeth; Class 3 mobility or Class 3 furcation involvement; or scheduled tooth extraction during the study period;\n24. Participants with medical conditions known to be associated with the onset of non-hereditary palmoplantar keratosis should be carefully evaluated by the Investigator. The presence, severity, and clinical significance of such conditions must be assessed to determine their relevance to study eligibility. Examples include hypothyroidism, myxoedema, chronic lymphoedema, acrocyanosis, livedo reticularis, psoriasis, lichen planus, reactive arthritis (Reiter's disease\u002Fkeratoderma blennorrhagicum), pityriasis rubra pilaris, atopic dermatitis, chronic hand or contact dermatitis, chronic dermatophytosis, chloracne, extensive verruca vulgaris, and keratoderma climactericum;\n25. Medications that may cause palmoplantar keratoderma (including, but not limited to, BRAF inhibitors, MEK inhibitors, selected tyrosine kinase inhibitors, systemic retinoids, and capecitabine) within 4 weeks prior to randomisation\n26. Established diagnosis of hepatitis B or hepatitis C infection. Participants who have gained immunity for HBV infection after vaccination are eligible (HBsAg negative, HBsAb positive and HBcAb negative). Participants with positive HBcAb are eligible only if HBV DNA level is undetectable. Participants positive for HCV antibody are eligible only if HCV RNA is negative;\n27. Known HIV infection or positive serology test at screening;\n28. Known intolerance and\u002For hypersensitivity to DPP1 inhibitors or any of the excipients contained in the formulation used in the trial. A history of discontinuation of DPP1 inhibitors due to an adverse drug reaction is exclusionary;\n29. Clinical diagnosis of Papillon-Lefèvre syndrome;\n30. Abnormal and clinically significant 12-lead ECG at screening or prior to randomisation that results in an active medical problem, which may impact the safety of the participant, per the Investigator's judgement. Male participants with QTcF \\>450 ms and female participants with QTcF \\>470 ms cannot be enrolled (not applicable for participants with permanent atrial fibrillation and for participants with pacemakers). For participants aged 12 to \\\u003C18 years, QTcF values should be interpreted using age-appropriate normative reference ranges;\n31. Participation in other investigational trial: participants who have received an investigational drug within 1 month or 5 half-lives of the previous administered product (whichever is longer) prior to screening, or participants who have been previously randomised in the present trial, or participants who are currently participating in another clinical trial;\n32. For females only (adult or adolescent): pregnant or lactating women, where pregnancy is defined as the state of a female after conception and until termination of the gestation, confirmed by a positive serum β-hCG laboratory test. Both serum and urine pregnancy tests are to be performed at screening, and a urine pregnancy test is to be performed prior to randomisation;\n33. Participants legally incapacitated or participants accommodated in an establishment as a result of an official or judicial order.\n\nExclusion Criteria for OLE (Extension) Study:\n\n1. Pregnant or lactating woman;\n2. ADRs\\*: participants who experienced an ADR (defined as an AE assessed by the Investigator as possibly related to the IMP) during the Main Phase should not be enrolled in the OLE Phase unless the Investigator considers that the AE would not prevent them from safely and effectively participating in the OLE Phase;\n3. Ongoing SAEs\\*: participants with ongoing SAEs at the time of the last visit of the Main Phase should not be enrolled in the OLE Phase unless the Investigator considers that the ongoing SAE would not prevent them from safely and effectively participating in the OLE Phase;\n4. Changes in medical history: any clinically significant changes in participant's medical history, physical examination\u002Fvital signs\u002Flaboratory analysis; concomitant therapies, documented in the Main Phase or any newly identified disease or condition that might, in the judgement of the Investigator, place the participant at undue risk;\n5. Any use of prohibited medications during the Main Phase that requires to be prolonged during the OLE Phase.","12 Years","85 Years",{"count":78,"type":20},904,[80],"PHASE3","This study aims to find out whether the tablet CHF10196 is safe and effective for people with non-cystic fibrosis bronchiectasis, a long-term lung disease that causes widened airways, mucus buildup, infections, and flare ups of symptoms. The main goal is to see whether CHF10196 can reduce the number of lung flareups each year compared with placebo. The study will also assess whether it can improve lung function, quality of life, and overall safety.\n\nThe study has two phases. In the first phase, participants receive either CHF10196 tablets or placebo without knowing which one they are taking. In the second phase, all participants receive CHF10196 so its long-term safety can be further studied. Adults aged 18 to 85 years and adolescents aged 12 to under 18 years can take part, while continuing their usual stable bronchiectasis treatment.\n\nUp to about 2.5 years of participation are planned. Around 904 participants will be enrolled, with regular clinic visits and phone check ins. In the first phase, participants take either CHF10196 or inactive tablets once daily; in the second phase, everyone takes CHF10196 once daily. Temporary treatment interruptions are allowed if needed, and participants who stop treatment early may still continue study visits, depending on the study phase.",[83],"Bronchiectasis","2026-06-19",{"date":60,"type":37},{"date":87,"type":20},"2026-11",{"date":89,"type":20},"2030-04",{"name":43,"class":44},{"id":92,"slug":4,"hasResults":10,"nctId":93,"briefTitle":94,"officialTitle":95,"acronym":96,"eligibilityCriteria":97,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":52,"enrollmentInfo":98,"targetDuration":4,"studyType":21,"phases":100,"briefSummary":101,"conditions":102,"keywords":103,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":117},"100634999","NCT07546981","Efficacy and Safety of CHF 5993 100\u002F6\u002F12.5 μg HFA-152a pMDI in Patients With Asthma Uncontrolled on Medium Doses of Inhaled Corticosteroids in Combination With Long-acting ß2-agonists","A 24 Week, Randomised, Double Blind, Multicentre, Active Controlled, 2-arm, Parallel Group Trial Comparing Efficacy and Safety of CHF 5993 100\u002F6\u002F12.5 μg HFA-152a pMDI (Fixed Combination of Extrafine Beclometasone Dipropionate Plus Formoterol Fumarate Plus Glycopyrronium Bromide) to CHF 1535 100\u002F6 μg HFA-134a pMDI (Fixed Combination of Extrafine Beclometasone Dipropionate Plus Formoterol Fumarate) in Patients With Asthma Uncontrolled on Medium Doses of Inhaled Corticosteroids in Combination With Long-acting ß2-agonists","TRICHECO","Inclusion Criteria:\n\n* Subjects must meet all of the following inclusion criteria to be eligible for enrolment into the study:\n\n  1. Subjects must provide written informed consent prior to any study-related procedures;\n  2. Subjects must be male or female Chinese aged ≥18 and\n\n     ≤75 years;\n  3. Subjects must have a documented history of asthma for at least 1 year and asthma must have been diagnosed before the subject's age of 40;\n  4. Subjects must have uncontrolled asthma, receiving double therapy only on medium doses of ICS in combination with LABA at a stable dose for at least 4 weeks prior to screening;\n  5. Subjects must have a pre-bronchodilator FEV1 \\\u003C80% of their predicted normal value, after appropriate wash out from bronchodilators at the screening visit.\n  6. Subjects must have a positive response to a reversibility test at screening.\n  7. Subjects must have uncontrolled asthma evidenced by a score at the ACQ-7 ≥1.5 (this criterion must be met at screening and at randomisation).\n\n     Exclusion Criteria:\n* Subjects meeting any of the following criteria will be excluded from study enrolment:\n\n  1. Subjects who have a history of near fatal asthma or of a past hospitalisation for status asthmaticus or severe asthma exacerbation which, in the judgement of the Investigator, may place the subject at undue risk;\n  2. Subjects who have undergone hospitalisation, emergency room visit or use of systemic corticosteroids for an asthma exacerbation in the 4 weeks prior to the screening visit or during the run-in period;\n  3. Subjects with any asthma exacerbation or respiratory tract infection in the 4 weeks prior to the screening visit or during run-in period;\n  4. Subjects with any change in dose, schedule or formulation of the combination ICS+LABA in the 4 weeks prior to the screening visit;\n  5. Subjects who have used systemic corticosteroid medication in the 4 weeks prior to screening or slow-release corticosteroids in the 12 weeks prior to screening;\n  6. Subjects who have a history of a diagnosis of chronic obstructive pulmonary disease (COPD) (according to the Global Initiative for Chronic Obstructive Lung Disease \\[GOLD\\] document), cystic fibrosis, bronchiectasis or alpha- 1 antitrypsin deficiency or any other significant lung disease which may interfere with study evaluations;\n  7. Subjects who are current smokers; ex-smokers with total cumulative exposure ≥10 pack-years; having stopped smoking 1 year or less prior to the screening visit;\n  8. Subjects who are treated with monoclonal antibodies (e.g., anti-immunoglobulin (Ig)E or anti-IgG antibodies) or biological drugs;\n  9. Subjects who have received an investigational drug within 2 months or six half-lives (whichever is greater) prior to the screening visit, or have been previously randomised in this study, or are currently participating in another clinical study.",{"count":99,"type":20},554,[80],"This is a study comparing CHF 5993 pMDI HFA-152a with CHF 1535 pMDI HFA-134a in Chinese patients with asthma uncontrolled on medium doses of ICS+LABA.",[26],[104,105,106,107,108],"Uncontrolled asthma","Triple therapy","HFA-152a","Pressurized metered-dose inhaler","China","2026-04-16",{"date":111,"type":37},"2026-04-23",{"date":113,"type":20},"2026-05",{"date":115,"type":20},"2028-10-17",{"name":43,"class":44},2,{"id":119,"slug":4,"hasResults":10,"nctId":120,"briefTitle":121,"officialTitle":122,"acronym":123,"eligibilityCriteria":124,"healthyVolunteers":10,"sex":16,"minAge":125,"maxAge":4,"enrollmentInfo":126,"targetDuration":4,"studyType":21,"phases":128,"briefSummary":129,"conditions":130,"keywords":132,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":4},"100632689","NCT07516951","A Study to Find an Efficacious and Safe Dose of CHF10067 (Zampilimab) in Participants With Idiopathic Pulmonary Fibrosis","A Phase IIb, Multicentre, Randomised, Double Blind, Placebo Controlled, Three-arm Parallel-group Study to Evaluate the Efficacy, Safety, and Tolerability at Week 24 of 2 Doses of CHF10067 (Zampilimab), With an Optional 24-week Double Blind, Placebo Controlled Extension Phase in Participants With Idiopathic Pulmonary Fibrosis","ZAPPHIRE","Inclusion Criteria:\n\n* Informed consent: Participant's written informed consent obtained prior to any study-related procedure.\n* Sex and age: Male or female, of any race and ethnicity, aged ≥40 years with a life expectancy of at least 1 year at screening in the opinion of the Investigator.\n* Body weight ≥45 kg.\n* Diagnosis of IPF: Diagnosis as defined by the 2018 and 2022 American Thoracic Society\u002FEuropean Respiratory Society\u002FJapanese Respiratory Society\u002FLatin American Thoracic Society Guidelines for a maximum 8 years before screening. The most recent HRCT(High-resolution computed tomography) ≤6 months prior to screening, reviewed by central reading, should be used to confirm the diagnosis.\n* Lung function: Forced vital capacity (FVC) ≥45% of predicted normal value and a ratio of forced expiratory volume in the first second\u002FFVC ≥0.7 at screening.\n* Diffusing capacity of the lung for carbon monoxide (DLCO) corrected for haemoglobin ≥25% of predicted normal at screening.\n* Oxygen saturation measured by pulse oximetry (peripheral capillary oxygen saturation \\[SpO2\\]) \\>90% at rest when the maximum oxygen flow is 4 L\u002Fmin by standard nasal cannula or the equivalent oxygen delivery via reservoir nasal cannula (≤2 L\u002Fmin).\n\nExclusion Criteria:\n\n* Participant with a documented diagnosis of coeliac disease.\n* Low respiratory tract infection: Documented low respiratory tract infection in the last 4 weeks prior to screening or documented acute exacerbation of IPF (defined as acute worsening or development of dyspnoea typically \\\u003C1 month duration;\n* Lung cancer: Active diagnosis or history of lung cancer.\n* Emphysema: HRCT (refer to inclusion criterion #5 \\[Diagnosis of IPF\\]), reviewed by central reading, shows the presence of emphysema ≥20% or that the extent of emphysema is greater than the extent of fibrosis.\n* Organ transplantation: End-stage fibrotic disease expected to require organ transplantation within 6 months from screening.\n* Other medical conditions: Clinically relevant and uncontrolled pulmonary (including any non-IPF pulmonary diagnosis), cardiac, hepatic, gastrointestinal, renal, endocrine, metabolic, neurologic, psychiatric disorders, active or untreated latent tuberculosis\u002Ftuberculosis infection that may interfere with the participant's ability to complete this study according to the Investigator's judgement.\n* Any other comorbid non-IPF pulmonary condition that may impact FVC according to the Investigator's judgement. Emphysema is allowed, unless it meets the above exclusion criterion regarding emphysema.\n* Participant currently treated, or been treated with cytotoxic and immunosuppressant\u002Fmodulator drugs within 48 weeks prior to screening. Systemic (IV, intramuscular, or oral) corticosteroids prednisone- equivalent dose of \\>10 mg\u002Fday used for \\>10 days.\n* Hypersensitivity: Known intolerance and\u002For hypersensitivity to any of the excipients contained in the formulation or any other substance used in the study.\n* History of allergic or anaphylactic reaction to human, humanised, chimeric Igs (immunoglobulins), or murine monoclonal antibodies.","40 Years",{"count":127,"type":20},240,[56],"The purpose of this study is to evaluate the efficacy, safety, and tolerability at Week 24 (Part A) of 2 doses of CHF10067 (zampilimab), with an optional 24-week double-blind, placebo-controlled extension phase (Part B) in participants with idiopathic pulmonary fibrosis.\n\nIt is a phase IIb, multicentre, randomised, double-blind, placebo-controlled, three-arm parallel-group study.\n\nA total of 240 participants with IPF (Idiomatic Pulmonary Fibrosis) will be randomised in approximately 150 investigational sites in North and Latin America, Europe, Asia, and Oceania. The optional extension phase (Part B) is only applicable to the European Union and Macedonia.",[131],"Idiopathic Pulmonary Fibrosis",[133],"IPF","2026-04-01",{"date":136,"type":37},"2026-04-08",{"date":138,"type":20},"2026-06-01",{"date":140,"type":20},"2028-02-12",{"name":43,"class":44},{"id":143,"slug":4,"hasResults":10,"nctId":144,"briefTitle":145,"officialTitle":146,"acronym":147,"eligibilityCriteria":148,"healthyVolunteers":10,"sex":149,"minAge":4,"maxAge":4,"enrollmentInfo":150,"targetDuration":4,"studyType":152,"phases":4,"briefSummary":153,"conditions":154,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":158,"lastUpdatePostDateStruct":159,"startDateStruct":161,"completionDateStruct":163,"leadSponsor":165,"locationsCount":166},"100588409","NCT06941025","Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding","Maternal and Postnatal Outcomes Study (MOS) A Worldwide Decentralized Observational Registry to Evaluate the Safety in Women With Fabry Disease and Their Infants Exposed to Elfabrio® (Pegunigalsidase Alfa-iwxj\u002FPegunigalsidase Alfa) During Pregnancy and\u002For Lactation","MOS","Inclusion Criteria:\n\n* Female patients with Fabry disease who have been exposed to at least 1 dose of pegunigalsidase alfa at any time during pregnancy (defined as having received pegunigalsidase alfa within 30 days prior to the DOC and\u002For during pregnancy) and\u002For during lactation, and their infants.\n\n  o DOC, defined as 20\u002F7 gestational weeks, will be calculated from last menstrual period \\[LMP\\] or ultrasound\n* Patient or parent\u002Flegally authorized representative must be able to understand and provide consent through an Institutional Review Board \u002F Independent Ethics Committee (IRB\u002FIEC) approved Informed Consent Form.\n\nExclusion Criteria:\n\n* None","FEMALE",{"count":151,"type":20},10,"OBSERVATIONAL","The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and\u002For during pregnancy and lactation.\n\nThe main objectives are to:\n\n* Assess pregnancy outcomes, including maternal and infant health.\n* Evaluate the occurrence of congenital malformations and other neonatal outcomes.\n\nThis is a global, decentralized, single-arm, prospective and retrospective registry planned to enroll participants over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data will be collected through a secure web-based platform, allowing patients and physicians to enter information via electronic case report forms (eCRFs).\n\nPregnancy and clinical outcomes will be documented throughout pregnancy and up to 12 months post-birth. Data from self-enrolled patients will be confirmed by their primary care or attending physician. This registry is observational and does not impact clinical care or treatment decisions.",[155,156,157],"Fabry Disease","Pregnancy","Pregnancy Complications","2026-03-17",{"date":160,"type":37},"2026-03-19",{"date":162,"type":37},"2025-05-23",{"date":164,"type":20},"2034-12",{"name":43,"class":44},5,{"id":168,"slug":4,"hasResults":10,"nctId":169,"briefTitle":170,"officialTitle":171,"acronym":172,"eligibilityCriteria":173,"healthyVolunteers":10,"sex":16,"minAge":174,"maxAge":175,"enrollmentInfo":176,"targetDuration":4,"studyType":21,"phases":178,"briefSummary":179,"conditions":180,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":158,"lastUpdatePostDateStruct":181,"startDateStruct":182,"completionDateStruct":184,"leadSponsor":186,"locationsCount":187},"100541342","NCT06328608","A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease","Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease","FLY","Inclusion Criteria:\n\n* Participants with the provision of informed consent from their legal guardians\n* Boys and girls aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to \\\u003C18 years (Cohort C).\n* Confirmed diagnosis of Fabry disease\n* Presence of at least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and\u002For clustered angiokeratoma.\n* History of Fabry pain: Fabry crises OR chronic pain.\n* Clinical condition that, in the investigator's opinion, requires ERT treatment.\n\nExclusion Criteria:\n\nAll Subjects:\n\n* Estimated glomerular filtration rate (eGFR) at screening \\\u003C 80 mL\u002Fmin\u002F1.73 m2.\n* History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or any component of the study drug.\n* Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB) or a dose change in ongoing treatment in the four weeks before screening.\n* Urine protein to creatinine ratio (UPCR) \\> 0.5 g\u002Fg (0.5 mg\u002Fmg or 500 mg\u002Fg) if not treated with an ACE inhibitor or ARB.\n* Currently taking another investigational drug for any condition.\n* History of acute kidney injury in the 12 months before screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g., ischaemia, toxic injury); or extrarenal pathology (e.g., prerenal azotaemia, acute postrenal obstructive nephropathy).\n* History of renal dialysis or kidney transplantation.\n* History of or current malignancy requiring treatment.\n* Severe cardiomyopathy or significant unstable cardiac disease within six months before screening.\n* A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within three months before screening.\n* Presence of any medical, emotional, behavioural, or psychological condition that, in the Investigator's judgement, could interfere with the subject's compliance with the requirements of the study.\n\nAdditional Exclusion Criteria for Subjects Enrolled in Stage I:\n\n* Female\n* Non-classic form of Fabry disease\n* Receipt of treatment for Fabry disease within six months before screening\n* Positive for anti-PRX-102 antibodies at screening\n\nAdditional Exclusion Criteria for Subjects in Stage II (i.e., non-treatment naïve males or females):\n\n* Unwilling to discontinue current ERT treatment for Fabry disease before baseline.\n* Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until 30 days after the last infusion.","2 Years","17 Years",{"count":177,"type":20},22,[56,80],"A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.",[155],{"date":160,"type":37},{"date":183,"type":37},"2025-07-29",{"date":185,"type":20},"2031-04",{"name":43,"class":44},12,{"id":189,"slug":4,"hasResults":10,"nctId":190,"briefTitle":191,"officialTitle":192,"acronym":193,"eligibilityCriteria":194,"healthyVolunteers":10,"sex":16,"minAge":195,"maxAge":196,"enrollmentInfo":197,"targetDuration":4,"studyType":21,"phases":199,"briefSummary":200,"conditions":201,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":202,"lastUpdatePostDateStruct":203,"startDateStruct":205,"completionDateStruct":207,"leadSponsor":209,"locationsCount":151},"100493863","NCT05710692","Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease","A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease (RISE)","RISE","Inclusion criteria (all subjects)\n\n1. Must have been born in Japan and have their biological parents and all 4 grandparents of Japanese descent\n2. A documented diagnosis of Fabry disease, as determined by the following:\n\n   * Males: Plasma and\u002For leukocyte alpha-galactosidase-A activity (by activity assay) that is ≤ 5% of mean normal laboratory levels or, if the enzymatic activity is above the 5% limit but still under the normal level, a confirmed disease-causing mutation of the GLA gene\n   * Females: Historical genetic test results consistent with Fabry mutations or, in the case of novel mutations, a first-degree male relative with Fabry disease\n   * All subjects: At least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and\u002For clustered angiokeratoma\n3. Estimated glomerular filtration rate (eGFR) at screening ≥40 mL\u002Fmin\u002F1.73 m2. For adults, this will be calculated using the Japanese Modified Chronic Kidney Disease Epidemiology Collaboration (JPN-CKD-EPI) Creatinine equation (2009); and for adolescents, it will be calculated using the Creatinine Cystatin C-based Chronic Kidney Disease in Children (CKiD) equation.\n4. Clinical condition that in the opinion of the Investigator requires treatment with ERT\n5. A female subject (including an adolescent in Cohort C, if applicable) must meet one of the following criteria:\n\n   * If of childbearing potential, she must:\n\n     * Have a negative serum pregnancy test result at screening, AND\n     * Agree to undergo a urine pregnancy test at baseline and every 12 weeks thereafter up to the final treatment, AND\n     * Agree to use one of the following highly reliable methods of contraception from the day of the informed consent signature until 30 days after the last infusion received. The following methods are acceptable:\n\n       * Placement of an intrauterine device (IUD) or intrauterine system (IUS)\n       * Combined (both oestrogen and progestogen) hormonal contraception (oral) associated with inhibition of ovulation, supplemented with a barrier method (preferably male condom)\n       * Bilateral tubal occlusion\n       * Sexual abstinence, defined as refraining from heterosexual intercourse during the entire study period\n       * Partner vasectomy, provided that the partner is the sole sexual partner and has received medical verification of the surgical success\n   * Be of non-childbearing potential, defined as one of the following:\n\n     * Post-menopausal (12 consecutive months of amenorrhea), OR\n     * Permanently sterile following hysterectomy, bilateral salpingectomy, or bilateral oophorectomy (supporting evidence required)\n\nAdditional inclusion criteria for subjects in Cohort A\n\nFor subjects enrolled in Cohort A, these specific inclusion criteria, in addition to those above, apply:\n\n* Aged ≥18 to ≤70 years\n* Treatment with agalsidase beta or agalsidase alfa for at least the last 12 months prior to screening, with the dose stable (defined as having received at least 80% of the labelled dose) for at least the last 6 months\n* Diagnosis of kidney impairment, defined as a linear slope of eGFR more negative than or equal to -2 mL\u002Fmin\u002F1.73 m2\u002Fyear. The historical eGFR slope will be calculated based on at least 3 serum creatinine values obtained over the 9 to 24 months prior to screening, using the JPN-CKD-EPI Creatinine equation (2009). This criterion will be confirmed at screening by calculating the screening eGFR slope using historical and screening serum creatinine values. Both historical and screening eGFR slopes will be used for the diagnosis of kidney impairment.\n\nAdditional inclusion criterion for subjects in Cohort B\n\nFor subjects enrolled in Cohort B, this specific inclusion criterion, in addition to those above, applies:\n\n\\- Aged ≥18 to ≤70 years\n\nAdditional inclusion criteria for subjects in Cohort C\n\nFor subjects enrolled in Cohort C, these specific inclusion criteria, in addition to those above, apply:\n\n* Aged ≥13 to \\\u003C18 years\n* Subjects who have previously received or are currently receiving ERT treatment, must be negative for ADAs to PRX-102\n\nExclusion Criteria:\n\n1. Administration of ERT for Fabry disease within 14 days before baseline, substrate reduction therapy for Fabry disease within 3 days before baseline, or chaperone therapy for Fabry disease within 3 days before baseline\n2. History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or to any component of the study drug\n3. Cohort A only: eGFR value of \\>90 to ≤120 mL\u002Fmin\u002F1.73 m2 at screening and a historical eGFR value \\>120 mL\u002Fmin\u002F1.73 m2 in the 9 to 24 months before screening, indicating absence of renal impairment. eGFR to be calculated using the JPN-CKD-EPI creatinine equation (2009).\n4. Urine protein to creatinine ratio (UPCR) \\>0.5 g\u002Fg (0.5 mg\u002Fmg or 500 mg\u002Fg) if not treated with an ACE inhibitor or ARB\n5. Initiation of treatment, or a change in dose to ongoing treatment, with an angiotensin-converting-enzyme inhibitor (ACEI) or angiotensin II receptor blocker (ARB) in the 4 weeks prior to screening.\n6. Currently taking another investigational drug for any condition\n7. Carry only known non-pathogenic Fabry mutations\n8. History of renal dialysis or kidney transplantation\n9. History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and renal vasculitis); non-specific conditions (e.g., ischemia, toxic injury); or extrarenal pathology (e.g., prerenal azotaemia, and acute postrenal obstructive nephropathy\n10. History of (or current) malignancy requiring treatment; the one exception is a prior history of resected basal cell carcinoma\n11. Severe cardiomyopathy or significant unstable cardiac disease within 6 months prior to screening\n12. A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within 3 months prior to screening, using a validated molecular assay or validated antigen assay\n13. Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until 30 days after the last infusion received\n14. Presence of any medical, emotional, behaevioral, or psychological condition that in the judgment of the Investigator could interfere with the subject's compliance with the requirements of the study\n15. Previous treatment with cellular therapy or gene therapy for any condition","13 Years","70 Years",{"count":198,"type":20},16,[56,80],"The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease. It is planned of a total of approximately 16 male and female Fabry disease patients between the ages of 13 and 70 years to be part of the study. The study is conducted in Japan.",[155],"2026-03-16",{"date":204,"type":37},"2026-03-18",{"date":206,"type":37},"2023-08-01",{"date":208,"type":20},"2029-08",{"name":43,"class":44},{"id":211,"slug":4,"hasResults":10,"nctId":212,"briefTitle":213,"officialTitle":214,"acronym":4,"eligibilityCriteria":215,"healthyVolunteers":216,"sex":16,"minAge":17,"maxAge":217,"enrollmentInfo":218,"targetDuration":4,"studyType":21,"phases":220,"briefSummary":222,"conditions":223,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":224,"lastUpdatePostDateStruct":225,"startDateStruct":227,"completionDateStruct":229,"leadSponsor":231,"locationsCount":232},"100528844","NCT06166056","A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Inhaled CHF6333 After Single Doses in Healthy Volunteers and After Single and Repeated Doses in Subjects With Bronchiectasis","A Randomised, Double-blind, Placebo-controlled Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Inhaled CHF6333 After Single Ascending Doses in Healthy Volunteers and One Single Dose in Adult Subjects With Bronchiectasis, Followed by a Placebo-controlled, Repeated Dose, 3-way Cross-over in Adult Subjects With Bronchiectasis","HV Inclusion Criteria:\n\n1. Signed and dated informed consent obtained prior to any study-related procedure;\n2. Healthy male or female subject ≥18 and ≤60 years of age at screening;\n3. Ability to understand the study procedures and the risks involved, and to be trained to use inhalers correctly and to generate sufficient peak inspiratory flow (PIF) (at least 40 L\u002Fmin) using the In-Check Dial set as per \"GenuAir\" inhaler resistance, at screening;\n4. Body mass index (BMI) ≥18 and ≤35 kg\u002Fm2 at screening;\n5. Non-smokers or ex-smokers who smoked \\\u003C5 pack-years and stopped smoking \\>1 year prior to screening;\n6. Good physical and mental status, determined via assessment of medical history and clinical examination, at screening and prior to randomisation;\n7. Vital signs within normal limits at screening and prior to randomisation: diastolic blood pressure (DBP) ≥40 and ≤89 mmHg, and systolic blood pressure (SBP) ≥90 and ≤139 mmHg; body temperature \\\u003C37.5°C;\n8. Triplicate 12-lead electrocardiogram (ECG) considered as normal (40 ≤ heart rate ≤110 bpm, 120 ms ≤ PR ≤210 ms, QRS ≤120 ms, Fridericia corrected QT interval \\[QTcF\\] ≤450 ms for males and QTcF ≤470 ms for females) at screening and prior to randomisation;\n9. Lung function measurements within limits at screening and prior to randomisation: forced expiratory volume in 1 second (FEV1) \\>80% predicted and FEV1\u002Fforced vital capacity (FVC) ratio \\>0.70;\n10. Male subjects willing to use a male condom throughout the study if they have women of childbearing potential (WOCBP) partners; male subjects with non-WOCBP partners or who are sterile do not have contraception requirements;\n11. Female subjects (if WOCBP) and\u002For their partners (if fertile) must be willing to use a highly effective birth control method, preferably with low user dependency, throughout the study; female subjects who are non-WOCBP or who have non-fertile partners do not have contraception requirements.\n\nHV Exclusion Criteria:\n\n1. Participation in another clinical study where investigational drug was received and the last investigations were performed less than 3 months prior to randomisation;\n2. Clinically relevant and uncontrolled respiratory, cardiac, hepatic, gastrointestinal, renal, endocrine, hematologic, metabolic, neurological, or psychiatric disorders that may interfere with successful completion of this protocol, according to the investigator's judgment;\n3. Clinically relevant abnormal laboratory values at screening suggesting an unknown disease and requiring further clinical investigation or which may impact the safety of the subject or the evaluation of the results of the study, according to the investigator's judgment;\n4. History of respiratory diseases;\n5. Positive human immunodeficiency virus (HIV) 1 or HIV2 serology results at screening;\n6. Hepatitis serology results which indicate acute or chronic hepatitis B (HB) or hepatitis C virus (HCV) at screening;\n7. Documented coronavirus disease 2019 (COVID-19) diagnosis within 2 weeks prior to screening or prior to randomisation, or associated complications\u002Fsymptoms, which have not resolved within 2 weeks prior to screening;\n8. Blood donation or blood loss (equal or more than 450 mL) less than 2 months prior to screening or prior to randomisation;\n9. Abnormal liver enzymes at screening or prior to randomisation (alanine aminotransferase \\[ALT\\] or aspartate aminotransferase \\[AST\\] or bilirubin: \\>1.5× upper limit of normal \\[ULN\\]);\n10. Positive urine test for cotinine at screening or prior to randomisation;\n11. Documented history of alcohol abuse within 12 months prior to screening or a positive alcohol breath test at screening or prior to randomisation;\n12. Documented history of drug abuse within 12 months prior to screening or a positive urine drug screen at screening or prior to randomisation;\n13. Treatment with prohibited concomitant medications or if the subject is expected to take prohibited concomitant medications during the study;\n14. Presence of any current infection, or previous infection that resolved less than 7 days prior to screening or prior to randomisation;\n15. Known intolerance and\u002For hypersensitivity to any of the excipients contained in the formulation used in the study;\n16. Unsuitable veins for repeated venepuncture;\n17. Heavy caffeine drinker;\n18. For females only: pregnant or lactating women, where pregnancy is defined as the state of a female after conception and until termination of the gestation, confirmed by a positive serum human chorionic gonadotropin laboratory test. A serum pregnancy test is to be performed at screening and a urine pregnancy test is to be performed prior to randomisation;\n19. Use of any kind of electronic smoking devices within 6 months prior to screening.\n\nPart I BE subjects Inclusion Criteria:\n\n1. Signed and dated informed consent obtained prior to any study-related procedure;\n2. Male or female subject ≥18 and ≤80 years of age at screening;\n3. Clinical history consistent with BE (cough, chronic sputum production, and\u002For recurrent respiratory infections) that is confirmed by chest computed tomography (CT) demonstrating BE affecting 1 or more lobes (confirmation may be based on prior chest CT); subjects whose past chest radiographic image records or report are not available will undergo chest CT scan during screening;\n4. Post-bronchodilator FEV1 ≥50% of the predicted value at screening;\n5. If currently receiving treatment for BE, this treatment should be administered at a stable dose;\n6. Subjects in a stable clinical condition with no exacerbation of BE for at least 4 weeks prior to randomisation. A pulmonary exacerbation is defined as a deterioration in three or more of the key symptoms for at least 48 hours (cough, sputum volume and\u002For consistency, sputum purulence, breathlessness and\u002For exercise tolerance, fatigue and\u002For malaise, haemoptysis) AND a clinician-prescribed course of antibiotics;\n7. Vital signs within normal limits at screening and prior to randomisation: DBP ≥40 and ≤89 mmHg, and SBP ≥90 and ≤150 mmHg; body temperature \\\u003C37.5°C;\n8. Ability to understand the study procedures and the risks involved, and the ability to be trained to use the inhalers correctly and to generate sufficient PIF (at least 40 L\u002Fmin) using the In-Check Dial set as per \"GenuAir\" inhaler resistance, at screening;\n9. Male subjects willing to use a male condom throughout the study if they have WOCBP partners; male subjects with non-WOCBP partners or who are sterile do not have contraception requirements;\n10. Female subjects (if WOCBP) and\u002For their partners (if fertile) must be willing to use a highly effective birth control method, preferably with low user dependency, throughout the study; female subjects who are non-WOCBP or who have non-fertile partners do not have contraception requirements.\n\nPart I BE subjects Exclusion Criteria:\n\n1. History of a clinically meaningful unstable or uncontrolled chronic comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering the study drug to the subject;\n2. Acute symptomatic respiratory tract infection which constitutes an increase from the subject's baseline, per the investigator's judgment, within 4 weeks prior to randomisation;\n3. Abnormal and clinically significant 12-lead ECG at screening or prior to randomisation that results in an active medical problem, which may impact the safety of the subject, per the investigator's judgment. Male subjects with QTcF \\>450 ms and female subjects with QTcF \\>470 ms cannot be enrolled;\n4. Clinically significant abnormal laboratory values at screening that, in the opinion of the investigator, may put the subject at risk by participating in the study, or interfere with the subject's treatment or assessment, or influence the results of the study;\n5. Participation in another clinical study, where investigational drug was received less than 30 days or less than 5 half-lives of the previous administered product (whichever is longer) prior to screening; participation in another clinical study is also not allowed between screening and randomisation;\n6. Primary diagnosis of asthma, as determined by the investigator;\n7. Concomitant diagnosis of cystic fibrosis;\n8. Diagnosis of chronic obstructive pulmonary disease (COPD) made by a clinician, with airflow obstruction (post-bronchodilator FEV1\u002FFVC ratio \\\u003C0.7) and at least a 10 pack-year smoking history; subjects with documented COPD but without airflow obstruction or smoking history will be permitted;\n9. Current smokers; ex-smokers must have stopped for at least 1 year prior to screening (≥6 months for electronic smoking devices);\n10. Subjects with an active tuberculous mycobacteria (TM) or non-tuberculous mycobacteria (NTM) infection requiring or receiving antibiotic treatment, an active allergic bronchopulmonary aspergillosis requiring treatment with corticosteroids or anti-fungal therapy, and\u002For a connective tissue disease (CTD) receiving an immunosuppressive treatment equivalent to \\>10 mg prednisolone daily or systemic immunosuppression;\n11. Diagnosis of common variable immunodeficiency (CVID) or other immunodeficiencies requiring immunoglobulin treatment, based on subject history;\n12. Diagnosis of rheumatoid arthritis;\n13. Malignancy that has not been in complete remission for at least 1 year or any untreated localised carcinomas;\n14. History of solid organ\u002Fhaematological transplantation and receiving immunosuppressive therapy;\n15. Use of any antimicrobials (oral, inhaled, or intravenous) within 4 weeks prior to randomisation, except for subjects who are on stable treatment with macrolide antibiotics (for at least 3 months);\n16. Medical history of discontinuation of previous inhaled therapy due to bronchospasm or intolerance;\n17. Documented COVID-19 diagnosis within 4 weeks prior to randomisation, or associated complications\u002Fsymptoms;\n18. Diagnosis of alpha-1-antitrypsin (AAT) deficiency defined as an AAT serum level \\\u003C110 mg\u002FdL; a prior test result of AAT serum level to confirm the diagnosis will be acceptable. In the event that a prior result is not available, a new test will be performed during the screening period and the result considered for the eligibility at randomisation.\n19. Treatment with prohibited concomitant medications or if the subject is expected to take prohibited concomitant medications during the study;\n20. Subjects treated with monoclonal antibodies (mAbs) for any respiratory conditions;\n21. Subjects with traction BE;\n22. For females only: pregnant or lactating women, where pregnancy is defined as the state of a female after conception and until termination of the gestation, confirmed by a positive serum human chorionic gonadotropin laboratory test. A serum pregnancy test is to be performed at screening, and a urine pregnancy test is to be performed prior to randomisation;\n23. Positive HIV1 or HIV2 serology results at screening;\n24. Hepatitis serology results which indicate acute or chronic HB or HCV at screening;\n25. BMI ≤17 kg\u002Fm2 at screening.\n\nPart II BE subjects Inclusion Criteria: the subjects must meet all the inclusion criteria listed in Part I for subjects with BE (Cohort B), except the inclusion criterion below:\n\n4\\. Post-bronchodilator FEV1 ≥30% of the predicted value at screening.\n\nThe subjects must also meet the additional inclusion criteria listed below:\n\n11\\. Subjects who are regular daily sputum producers and who are able to provide at least one sputum sample at screening and two sputum samples prior to randomisation; 12. Subjects with active NE level in sputum sample at screening, defined by either a positive bacterial culture in a local laboratory (a positive result for any pathogen will be accepted for eligibility) or by a positive reading of the NEATstik® research use only test.\n\nPart II BE subjects Exclusion Criteria:\n\nSee the list of criteria in Part I for subjects with BE (Cohort B), except the exclusion criterion below:\n\n15\\. Use of oral or inhaled antibiotics \\\u003C 3 months prior to randomization as chronic treatment for BE. Patients on antibiotics as chronic treatment should have been on such treatment for ≥ 3 months prior to randomization while meeting all other inclusion and exclusion criteria.",true,"80 Years",{"count":219,"type":20},45,[221,56],"PHASE1","The aim of this clinical trial is to assess the safety of:\n\n* single doses of the study drug CHF6333 in Healthy Volunteers (HVs) and in subjects with Bronchiectasis (BE) - Part I\n* repeated doses of the study drug CHF6333 in subjects with BE - Part II",[83],"2026-03-04",{"date":226,"type":37},"2026-03-05",{"date":228,"type":37},"2023-11-29",{"date":230,"type":20},"2026-08",{"name":43,"class":44},9,{"id":234,"slug":4,"hasResults":10,"nctId":235,"briefTitle":236,"officialTitle":237,"acronym":238,"eligibilityCriteria":239,"healthyVolunteers":10,"sex":16,"minAge":240,"maxAge":4,"enrollmentInfo":241,"targetDuration":4,"studyType":21,"phases":243,"briefSummary":244,"conditions":245,"keywords":256,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":259,"lastUpdatePostDateStruct":260,"startDateStruct":262,"completionDateStruct":264,"leadSponsor":266,"locationsCount":242},"100586616","NCT06917690","A Study to Learn About the Safety and Efficacy of the Drug Oleogel-S10 in Japanese Patients With Epidermolysis Bullosa","Multi-centeR, Open-label Study to EValuate the Safety, Efficacy, and Pharmacokinetics of FIlsuVEz (Oleogel-S10) in Japanese Subjects With Epidermolysis Bullosa (REVIVE)","REVIVE","Inclusion Criteria:\n\n1. Male or female aged ≥ 21 days\n2. Confirmed diagnosis of either JEB or DEB\n3. Both biological parents and all 4 grandparents of Japanese descent\n4. At least 3 EB wounds that meet the following criteria at the time of enrollment:\n\n   * All are located outside of the anogenital region\n   * All are distinguishable from any other wounds on the subject's body (separated by a minimum of 5 cm)\n   * At least one has been present for a minimum of 21 days but less than 9 months AND is 10 cm2 to 50 cm2 in size\n   * At least two have been present for a minimum of 21 days but less than 9 months AND\u002FOR are 10 cm2 to 50 cm2 in size\n5. A female subject must meet one of the following criteria:\n\n   * If of childbearing potential, she must:\n\n     1. Have a negative pregnancy test result at Screening and Baseline Visits, AND\n     2. Agree to use one of the following highly reliable methods of contraception from the day of the informed consent signature until the day after the last Oleogel- S10 application. The following methods are acceptable:\n\n        * Placement of an intrauterine device (IUD) or intrauterine releasing system (IUS)\n        * Combined (both estrogen and progestogen) hormonal contraception (oral) associated with inhibition of ovulation, supplemented with a barrier method (preferably male condom)\n        * Bilateral tubal occlusion\n        * Sexual abstinence, defined as refraining from heterosexual intercourse during the entire study period\n        * Partner vasectomy, provided that the partner is the sole sexual partner and has received medical verification of the surgical success\n   * Be of non-childbearing potential, defined as one of the following:\n\n     * Pre-menarche, OR\n     * Post-menopausal (12 consecutive months of amenorrhea without an alternative medical cause), OR\n     * Permanently sterile following hysterectomy, bilateral salpingectomy, or bilateral oophorectomy (supporting evidence required)\n6. Subject and\u002For subject's legal representative has been informed about the study, has read and understood the information provided, and has given written informed consent\n7. Subject and\u002For subject's legal representative is able and willing to follow all study procedures and instructions\n\nExclusion Criteria:\n\n1. Hypersensitivity to Oleogel-S10 or any of its excipients\n2. Diagnosis of EB subtypes simplex or Kindler EB\n3. Receipt of systemic antibiotics for wound-related infections within 7 days prior to enrollment\n4. Receipt of systemic or topical (on EB wounds) steroids within 30 days prior to enrollment Exceptions: Inhaled, ophthalmic, or mucosal applications, such as budesonide suspension for esophageal strictures\n5. Receipt of immunosuppressive therapy or cytotoxic chemotherapy within 60 days prior to enrollment Note: Dupilumab to manage pruritis is allowed if the subject has been on a stable dose for more than 3 months prior to baseline\n6. Receipt of systemic gene therapy for the treatment of inherited EB\n7. Receipt of short-acting stem cell therapy (infusion of Muse cells, allogeneic adipocyte-derived MSC sheet (topically applied), TEMCELL (allogeneic MSCs, subcutaneous injection)) within 6 months prior to study enrollment\n8. Receipt of topical gene therapy for the treatment of inherited EB (e.g., beremagene geperpavec) within 3 months prior to enrollment\n9. Receipt of a JACE® skin graft on any of the target wounds\n10. Current and\u002For former malignancy, including skin malignancies such as basal cell carcinoma and squamous cell carcinoma\n11. Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until study participation ends\n12. Enrollment in any other interventional study or treated with any other investigational drug for any disease within 4 weeks prior to enrollment\n13. Presence of any factor in the subject and\u002For legal representative that could interfere with study compliance, such as the ability to attend the scheduled study visits or to properly manage the dressing changes at home","21 Days",{"count":242,"type":20},6,[80],"The goal of this clinical trial is to learn if Oleogel-S10 gel works to treat skin wounds from two types of inherited epidermolysis bullosa (EB): junctional EB (JEB) or dystrophic EB (DEB) in the Japanese population. Children and adults may participate. The trial also looks at the safety of Oleogel-S10 gel. The main questions it will answer are:\n\n* Does Oleogel-S10 gel close wounds from JEB or DEB within 45 days or reduce the size of the wounds?\n* Are there any medical problems when using Oleogel-S10 gel?\n* How much of the drug ends up in your blood?\n\nThe study has 2 parts. In Part 1, participants will:\n\n* Apply Oleogel-S10 gel to the EB wound dressings at least once every 4 days for 45 days.\n* Visit the clinic once every 2 weeks for checkups and tests. If participants complete Part 1, they may participate in Part 2 if they choose to. In Part 2, participants will continue to receive Oleogel-S10 gel until the product is available to buy in Japan or until the trial is stopped by the company.",[246,247,248,249,250,251,252,253,254,255],"Epidermolysis Bullosa","Junctional Epidermolysis Bullosa","Epidermolysis Bullosa, Dystrophic","Epidermolysis Bullosa, Junctional","Skin Abnormalities","Connective Tissue Disease","Collagen Diseases","Skin Diseases","Genetic Diseases, Inborn","Congenital Abnormalities",[257,258],"Filsuvez","Birch Bark extract","2025-12-18",{"date":261,"type":37},"2025-12-22",{"date":263,"type":37},"2025-04-18",{"date":265,"type":20},"2029-09",{"name":43,"class":44},{"id":268,"slug":4,"hasResults":10,"nctId":269,"briefTitle":270,"officialTitle":270,"acronym":271,"eligibilityCriteria":272,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":273,"targetDuration":4,"studyType":152,"phases":4,"briefSummary":275,"conditions":276,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":277,"lastUpdatePostDateStruct":278,"startDateStruct":280,"completionDateStruct":282,"leadSponsor":284,"locationsCount":151},"100567067","NCT06663358","A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients","MODERN","Inclusion Criteria:\n\n* Male or female aged \\> 18 years of age at the time of consent.\n* Genetically confirmed diagnosis of Fabry disease.\n* Either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease.\n* No contraindications for cardiac magnetic resonance imaging (cMRI)\n* Informed consent form (ICF) signed and dated indicating the individual has been informed of and agreed to all pertinent aspects of the study and is willing to comply with all study requirements, including completion of electronic patient reported outcomes (ePROs).\n* Cardiac Cohort:\n* Evidence of Fabry disease (FD)-related heart disease including one or more of the following:\n* Left ventricular hypertrophy (LVH) measured by left ventricular mass index (LVMI) (g\u002Fm2) elevation above age\u002Fsex specific reference ranges.\n* Posterior septum wall thickness (e.g., \\>=13mm) not explained by other factors (e.g., hypertension)\n* Low native T1 mapping on cMRI.\n* Typical Fabry-like scar on cMRI\n* Participants can receive cardiac magnetic resonance imaging (cMRI) with gadolinium enhancement as part of their SoC.\n* Estimated glomerular filtration rate (eGFR) \\>45 mL\u002Fmin\u002F1.73 m2, assessed within the prior 6 months.\n* Naïve Cohort:\n* Most recent eGFR\\>45 mL\u002Fmin\u002F1.73 m2, assessed within prior 6 months.\n* Male participants should have abnormal elevation in plasma lysoGb3 as assessed within 6 months prior to enrolment.\n* Long-Term Cohort:\n* Participants previously enrolled in the open label study CLI-06657AA1-04 (Previously PB-102-F60) (using pegunigalsidase alfa at a dose of 1mg\u002Fkg every 2 weeks) who have initiated or plan to initiate commercial pegunigalsidase alfa (Elfabrio®).\n\nExclusion Criteria:\n\n* Contraindication to magnetic resonance imaging (MRI) including known history of hypersensitivity to gadolinium contrast agent that is not managed by the use of premedication.\n* Pregnant at the time of enrolment.\n* Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study.\n* Active participation in any interventional study for Fabry disease\n* Treatment regimen at the time of enrolment in the study is different from the approved 1mg\u002Fkg every two weeks (note if regimen subsequently changes during the prospective part of the study, the participants can remain in the study)\n* Prior participation in a pegunigalsidase alfa trial using a dose of 2 mg\u002Fkg administered every 4 weeks.\n* Cardiac Cohort:\n* History of acute myocardial infarction or congestive heart failure with reduced left ventricular (LV) ejection fraction of less than 35%.\n* Cerebral vascular accident (CVA) in the prior 6 months.\n* Chronic liver cirrhosis.\n* FD-unrelated heart disease (e.g., scarring due to myocardial infarction, symptomatic occlusive coronary artery disease, moderate valvular heart disease not thought to be Fabry related).\n* The participant is or has been treated with any investigational drug for Fabry disease within 6 months of study start or investigational gene therapy for Fabry disease at any time point in the past.\n* Severe cardiac fibrosis defined as more than 3 segments that each have \\>50% fibrosis upon late gadolinium enhancement cMRI at any prior cMRI.\n* Naïve Cohort:\n* Prior exposure to a FD therapy (Replagal®, Fabrazyme®, and Galafold®) at any time point.\n* Severe cardiac fibrosis defined as more than 3 segments that each have \\>50% fibrosis upon late gadolinium enhancement cMRI on any prior cMRI",{"count":274,"type":20},100,"A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.",[155],"2025-11-13",{"date":279,"type":37},"2025-11-14",{"date":281,"type":37},"2024-11-06",{"date":283,"type":20},"2029-11",{"name":43,"class":44},{"id":286,"slug":4,"hasResults":10,"nctId":287,"briefTitle":288,"officialTitle":289,"acronym":4,"eligibilityCriteria":290,"healthyVolunteers":216,"sex":16,"minAge":17,"maxAge":291,"enrollmentInfo":292,"targetDuration":4,"studyType":21,"phases":294,"briefSummary":295,"conditions":296,"keywords":298,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":303,"lastUpdatePostDateStruct":304,"startDateStruct":306,"completionDateStruct":308,"leadSponsor":310,"locationsCount":311},"100605489","NCT07163182","Evaluation of Safety, Side Effects and How the Drug CHF6467 Administered Via Intranasal Route is Absorbed, Modified and Removed in Healthy Subjects","A Randomised, Double-blind, Placebo-controlled Study to Investigate the Safety, Tolerability and Pharmacokinetics of CHF6467 After Single and Repeated Ascending Doses by Intranasal Route in Healthy Adult Subjects","Inclusion Criteria:\n\n1. Subject's written informed consent obtained prior to any study-related procedures;\n2. Willingness and ability to understand the risks involved and to understand and comply with the study procedures;\n3. Healthy male and female subjects, aged 18-55 years inclusive at screening;\n4. Weight ≥ 50 kg and \\\u003C85 kg and Body Mass Index (BMI) between 18.0 and 30.0 kg\u002Fm² inclusive at screening and on Day -1;\n5. Non-smoker or ex-smoker who smoked less than 5 pack years (Pack years = the number of cigarette packs per day times the number of years) and stopped smoking at least 1 year prior to screening;\n6. Good physical condition and mental status determined by the Investigator, based on the subject's medical history and general clinical examination at screening and Day -1;\n7. Vital signs within normal limits at screening and at Day-1: 60 mmHg ≤ diastolic blood pressure (DBP) ≤ 89 mmHg, 90 mmHg ≤ systolic blood pressure (SBP) ≤ 139 mmHg (three measures performed after at least 5 minutes of resting; the mean value must be within the defined range). Axillary body temperature of 35.5-37.0º Celsius inclusive;\n8. Bedside 12-lead electrocardiogram (ECG) considered as normal (45 bpm ≤ heart rate \\[HR\\] ≤ 100 bpm, 120 ms ≤ PR interval \\[PR\\] ≤ 210 ms, QRS interval \\[QRS\\] ≤ 120 ms, QT interval \\[QT\\] corrected using Fridericia's formula \\[QTcF\\] ≤ 450 ms for males and ≤ 470 ms for females) at screening visit and Day -1; the mean value of three measurements must be within the range.\n9. Males fulfilling one of the following criteria:\n\n   1. Males with pregnant or non-pregnant women of childbearing potential (WOCBP) partners: they must be willing to use male condom from the signature of the informed consent and until the follow-up visit or\n   2. Non-fertile male subjects (contraception is not required in this case) or\n   3. Males with partner not of childbearing potential (contraception is not required in this case);\n10. Female subjects fulfilling one of the following criteria:\n\n    1. Women of non-childbearing potential (WOCBP) defined as physiologically incapable of becoming pregnant (i.e., post-menopausal or permanently sterile). Tubal ligation or partial surgical interventions are not acceptable. If indicated, as per Investigator's request, post-menopausal status may be confirmed by follicle-stimulating hormone (FSH) levels (according to local laboratory ranges);\n    2. WOCBP fulfilling one of the following criteria:\n\n    i. WOCBP with fertile male partners: they and\u002For their partner must be willing to use a highly effective birth control method preferably with low user dependency from the signature of the informed consent and until the follow-up visit; or ii. WOCBP with non-fertile male partners (contraception is not required in this case).\n\nExclusion Criteria:\n\n1. The subject has taken non-permitted concomitant medications in the predefined period prior to screening or prior to randomisation or is expected to take non-permitted concomitant medications during the study;\n2. Participation to investigational study: subjects who have received any investigational drug within the 30 days (60 days for biologics) or a more appropriate time as determined by the Investigator (e.g. approximately 5 half-lives of the investigational drug whatever is longer);\n3. Significant nasal congestion or signs of nasal damage, bleeding, excoriation or ulceration at physical examination at screening or Day -1;\n4. History of frequent nosebleeds;\n5. Any ongoing acute (e.g. non-allergic rhinitis) or chronic (e.g. chronic rhinosinusitis or chronic purulent postnasal drip) condition of the nasal cavity, or clinically significant physical finding (e.g. nasal polyps, nasal structural abnormalities, nasal trauma, severe nasal septal deviation) which, in the opinion of the Investigator, can interfere with the administration or absorption of the study medication. Subjects with recent upper respiratory tract infections will be allowed in the study only if their nasal symptoms have been completely resolved for more than 2 weeks prior to screening;\n6. Clinically significant abnormal 24-hour Holter at screening;\n7. Clinically relevant and uncontrolled respiratory, cardiac, hepatic, gastrointestinal, renal, endocrine, metabolic, neurologic or psychiatric disorder that may interfere with successful completion of this protocol, any known malignancies, or any condition that in Investigator's opinion may pose the subject at risk from participating in the study;\n8. Any clinically relevant abnormal laboratory value at screening or Day -1suggesting an undiagnosed condition that requires further clinical investigation or may impact the safety of the subject or the evaluation of the study results according to the Investigator's judgement; Note: In case of abnormal laboratory values that could indicate a temporary condition, the test can be repeated once before randomisation;\n9. For females only: pregnant or lactating women, where pregnancy is defined as the state of a female after conception and until termination of the gestation, confirmed by a positive serum human chorionic gonadotropin laboratory test. Serum pregnancy test to be performed at screening and urine pregnancy test to be performed at Day -1.","55 Years",{"count":293,"type":20},68,[221],"A clinical trial to investigate the safety and tolerability of single and multiple intranasal (through the nose) dosing with the study drug CHF6467 in 68 healthy adult subjects.\n\nThe study will investigate also how CHF6467 moves and behaves in the blood and in the fluid around the brain and spinal cord (cerebrospinal fluid) and if the drug CHF6467 causes an immune response by looking for specific molecules, called antibodies that may form against it.\n\nThe study will be divided into two parts - Part 1 (testing single ascending doses of the study drug, SAD, lasting 4 days) and Part 2 (testing repeated or multiple ascending doses of the study drug, MAD, lasting 11 days).\n\nEach part of the study consists of a screening period, when eligible healthy volunteers will be selected, a treatment period, during which the study drug administration will take place and a follow-up period.",[297],"Healthy Volunteers",[299,300,301,302],"Single Ascending Dose","Multiple Ascending Dose","Intranasal","Cerebrospinal fluid","2025-09-18",{"date":305,"type":37},"2025-09-23",{"date":307,"type":37},"2025-09-15",{"date":309,"type":20},"2026-08-11",{"name":43,"class":44},1,{"id":313,"slug":4,"hasResults":10,"nctId":314,"briefTitle":315,"officialTitle":316,"acronym":317,"eligibilityCriteria":318,"healthyVolunteers":10,"sex":16,"minAge":4,"maxAge":319,"enrollmentInfo":320,"targetDuration":4,"studyType":152,"phases":4,"briefSummary":321,"conditions":322,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":324,"lastUpdatePostDateStruct":325,"startDateStruct":327,"completionDateStruct":329,"leadSponsor":330,"locationsCount":117},"100530263","NCT06184503","Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3","A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age","LAMPO(0-3)","Inclusion Criteria:\n\n* Subjects with the provision of informed consent from their legal guardians (LAR)\n* Have a confirmed diagnosis of alpha mannosidosis\n* Have initiated treatment with velmanase alfa between birth to at least six weeks before turning 3 years of age\n* Have information on the disease marker GlcNAc(Man)2 obtained:\n\nbefore velmanase treatment initiation (ideally max 6 month before), and at least one post-treatment sample, collected following at least six weeks of treatment.\n\n\\- Participants treated with Lamzede, 1 mg\u002Fkg body weight, via weekly intravenous infusions.\n\nExclusion Criteria:\n\nParticipants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).","3 Years",{"count":166,"type":20},"The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis.\n\nThe main questions it aims to answer are:\n\n* study the effect of velmanase alfa on a marker of the disease called GlcNAc(Man)2 after one year of therapy\n* explore how the child's body reacts to velmanase alfa during the therapy The parents or legal guardians of participants will be asked to provide the results of analyses performed in the routine clinical setting related to the participant's general health and the administration of velmanase alfa.\n\nAdditional data will be extracted from other observational sponsored studies\u002Fregistries, compassionate use programs, investigator-initiated studies (IIS), and published case reports (presented in the literature) if existing.",[323],"Alpha-Mannosidosis","2025-05-15",{"date":326,"type":37},"2025-05-16",{"date":328,"type":37},"2025-02-19",{"date":265,"type":20},{"name":43,"class":44},{"id":332,"slug":4,"hasResults":10,"nctId":333,"briefTitle":334,"officialTitle":335,"acronym":336,"eligibilityCriteria":337,"healthyVolunteers":10,"sex":16,"minAge":125,"maxAge":4,"enrollmentInfo":338,"targetDuration":4,"studyType":152,"phases":4,"briefSummary":340,"conditions":341,"keywords":343,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":348,"lastUpdatePostDateStruct":349,"startDateStruct":351,"completionDateStruct":353,"leadSponsor":354,"locationsCount":311},"100489386","NCT05652439","PASS to Assess Cardiovascular and Cerebrovascular Events in COPD Patients Initiating Fixed Triple Therapy (DPI or pMDI)","Multinational Database Cohort Study to Assess Adverse Cardiovascular and Cerebrovascular Outcomes in Patients With COPD Initiating a Fixed Triple Therapy Containing BDP, FF and GB Administered Via DPI Compared to pMDI","TRIBE","Inclusion Criteria:\n\n* Patients diagnosed with COPD\n* 40 years or older\n* Availability of baseline information for a minimum of 12 months before the index date\n* New users of single inhaler triple therapy BDP\u002FFF\u002FGB via DPI or pMDI\n\nExclusion Criteria:\n\n* Single or multi-inhaler triple therapy in the previous 90 days before the index date\n* Prescription of BDP\u002FFF\u002FGP administered via pMDI or DPI or any other single inhaler triple therapy in addition to a study drug on the index date.\n* Hospitalization due to cardiovascular causes in the previous 30 days before the index date.",{"count":339,"type":20},25000,"The aim of this Post Authorisation Safety Study (PASS) is to assess the incidence of adverse cardiovascular and cerebrovascular events in COPD patients who are new to inhaled fixed triple therapy (dual bronchodilator plus corticosteroid) administered via Dry Powder Inhaler (DPI) compared to new users of pressurized Metered Dose Inhaler (pMDI).\n\nData from clinical practice from different European data sources will be collected. The baseline hypothesis is that the DPI is not associated with different risks of the primary and secondary outcomes, compared with pMDI.",[342],"Chronic Obstructive Pulmonary Disease",[344,345,346,347],"retrospective","cardiovascular","cerebrovascular","triple therapy","2025-04-30",{"date":350,"type":37},"2025-05-04",{"date":352,"type":37},"2022-12-02",{"date":230,"type":20},{"name":43,"class":44},{"id":356,"slug":4,"hasResults":10,"nctId":357,"briefTitle":358,"officialTitle":4,"acronym":4,"eligibilityCriteria":359,"healthyVolunteers":10,"sex":4,"minAge":4,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":360,"phases":4,"briefSummary":361,"conditions":362,"keywords":4,"overallStatus":363,"whyStopped":4,"lastUpdateSubmitDate":364,"lastUpdatePostDateStruct":365,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":367,"locationsCount":4},"100436131","NCT04959240","Expanded Access to Velmanase Alfa","Inclusion Criteria:\n\n\\-\n\nExclusion Criteria:\n\n\\-","EXPANDED_ACCESS","Individual patient expanded access requests may be considered for patients who have no other treatment options.",[323],"AVAILABLE","2023-09-22",{"date":366,"type":37},"2023-09-25",{"name":43,"class":44},""]