[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Arrowhead Pharmaceuticals\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":313},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,14,0,[8,40,52,75,95,115,135,156,178,200,234,255,273,295],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":29,"startDateStruct":32,"completionDateStruct":34,"leadSponsor":36,"locationsCount":39},"100054176",false,"NCT07223658","Study of ARO-DIMERPA in Adult Participants With Mixed Hyperlipidemia","Phase 1\u002F2a, Double-blind, Placebo-controlled, Single and Multiple Dose-escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of ARO-DIMERPA in Adult Subjects With Mixed Hyperlipidemia","Inclusion Criteria:\n\n* Willing to follow diet counseling as per Investigator judgment based on local standard of care\n* Specific fasting TG, LDL-C, and non-high density lipoprotein cholesterol levels at Screening\n* Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later; participants must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later\n\nExclusion Criteria:\n\n* Current use or use within last 365 days or within 5-half-lives before Day 1 based on plasma PK, whichever is longer, of any hepatocyte-targeted siRNA\n* Current use or use within last 90 days or within 5-half-lives before Day 1 based on plasma PK, whichever is longer, of any antisense oligonucleotide therapy\n* Current use or use within last 60 days from Day 1 of any PCSK9 inhibitor monoclonal antibodies (eg, evolocumab or alirocumab)\n* Uncontrolled hypertension\n* History of bleeding diathesis or coagulopathy\n* Current diagnosis of nephrotic syndrome\n\nNote: Additional inclusion\u002Fexclusion criteria may apply per protocol.","ALL","18 Years",{"count":18,"type":19},78,"ESTIMATED","INTERVENTIONAL",[22,23],"PHASE1","PHASE2","Study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD), and effects on low-density lipoprotein cholesterol (LDL-C) and triglycerides (TGs) of single-dose ARO-DIMERPA and multiple doses of ARO-DIMERPA in adult participants with mixed hyperlipidemia.",[26],"Hyperlipidemia; Mixed","RECRUITING","2026-07-10",{"date":30,"type":31},"2026-07-13","ACTUAL",{"date":33,"type":31},"2025-12-22",{"date":35,"type":19},"2027-07",{"name":37,"class":38},"Arrowhead Pharmaceuticals","INDUSTRY",15,{"id":41,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":42,"targetDuration":4,"studyType":20,"phases":43,"briefSummary":24,"conditions":44,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":45,"lastUpdatePostDateStruct":46,"startDateStruct":48,"completionDateStruct":49,"leadSponsor":50,"locationsCount":51},"100610136",{"count":18,"type":19},[22,23],[26],"2026-06-29",{"date":47,"type":31},"2026-06-30",{"date":33,"type":31},{"date":35,"type":19},{"name":37,"class":38},11,{"id":53,"slug":4,"hasResults":10,"nctId":54,"briefTitle":55,"officialTitle":56,"acronym":57,"eligibilityCriteria":58,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":59,"targetDuration":4,"studyType":20,"phases":61,"briefSummary":63,"conditions":64,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":67,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":74},"100583779","NCT06880770","Study of Plozasiran in Adults With Severe Hypertriglyceridemia at Risk of Acute Pancreatitis","Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults With Severe Hypertriglyceridemia at High Risk of Acute Pancreatitis (SHASTA-5 Study)","SHASTA-5","Inclusion Criteria:\n\n* Males, or nonpregnant (who do not plan to become pregnant) nonlactating females\n* Established diagnosis of SHTG and prior documented evidence of fasting TG levels of ≥ 880 mg\u002FdL (≥ 10 mmol\u002FL)\n* Documented evidence of at least 1 prior AP event not attributed to other etiologies occurring within the last 60 months prior to Screening.\n* Fasting low-density lipoprotein cholesterol (LDL-C) ≤ 130 mg\u002FdL (≤ 3.37 mmol\u002FL) at Screening\n* Screening hemoglobin A1c (HbA1c) ≤ 9.5%\n* Willing to follow diet counseling and maintain a stable low-fat diet\n* Must be on standard of care lipid and TG-lowering medications per local guidelines (unless documented as intolerant, or a treatment failure as determined by the Investigator)\n\nExclusion Criteria:\n\n* Use of any hepatocyte-targeted small interfering ribonucleic acid (siRNA) that targets lipids and\u002For triglycerides within 365 days before Day 1, except inclisiran.\n* Use of any other hepatocyte targeted siRNA or antisense oligonucleotide molecule within 60 days or within 5 half-lives lives before day 1. Whichever is longer.\n* AP ≤ 4 weeks prior to Randomization\u002FDay 1\n* Body mass index (BMI) \\> 45 kg\u002Fm\\^2\n* Any planned bariatric surgery or similar procedures to induce weight lost starting at consent through End of Study (EOS)\n* Planned coronary intervention (e.g. stent placement or heart bypass) during the study\n* History of arterial revascularization within 16 weeks of Screening\n* History of acute coronary syndrome event within 24 weeks of Screening\n* Recent atherosclerotic cardiovascular disease (ASCVD) event within 24 weeks of Screening\n* Recent unstable or symptomatic cardiac arrhythmia (including any associated medication changes) within 90 days of Screening. Individuals with stable well-controlled atrial arrhythmia will be allowed to participate in the study\n* History of pacemaker or automatic implantable cardioverter defibrillators implant within 30 days before Screening\n* New York Heart Association Class III-IV heart failure or last known ejection fraction of \\\u003C 30%\n* Current diagnosis of nephrotic syndrome\n* Chronic kidney disease, defined by an estimated glomerular filtration rate (eGFR) \\\u003C 20 mL\u002Fmin\u002F1.73 m\\^2\n* Liver disease defined as cirrhosis or Child-Pugh Class B and C, or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>2.5× Upper Limit of Normal (ULN) at Screening\n\nNote: Additional Inclusion\u002FExclusion Criteria may apply per protocol",{"count":60,"type":19},288,[62],"PHASE3","This study will evaluate the efficacy and safety of plozasiran in approximately 288 adult participants with severe hypertriglyceridemia (SHTG) and history of at least two prior acute pancreatitis (AP) events not attributed to other etiologies, with at least one occurring within the last 12 months prior to screening. Eligible participants will be randomly assigned in a double-blind manner to either receive plozasiran 25 mg by subcutaneous (SC) injection every three months (Q3M) or matching placebo. Enrolled participants will be counseled to remain on the specified low-fat diet and background medications throughout the study. Following completion of the double-blind treatment period, or if the participant has a positively adjudicated AP event (whichever occurs first), participants will transition to the 12-month Open-Label Extension (OLE) treatment period receiving plozasiran 25 mg by SC injection Q3M.",[65],"Severe Hypertriglyceridemia","2026-06-25",{"date":68,"type":31},"2026-06-26",{"date":70,"type":31},"2025-04-24",{"date":72,"type":19},"2029-06",{"name":37,"class":38},95,{"id":76,"slug":4,"hasResults":10,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":4,"eligibilityCriteria":80,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":81,"enrollmentInfo":82,"targetDuration":4,"studyType":20,"phases":84,"briefSummary":85,"conditions":86,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":88,"startDateStruct":89,"completionDateStruct":91,"leadSponsor":93,"locationsCount":94},"100569922","NCT06700538","A First-In-Human Study of ARO-INHBE in Adults With Obesity With and Without Type 2 Diabetes Mellitus","A Phase 1\u002F2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-INHBE in Adult Volunteers With Obesity With and Without Diabetes Mellitus","Inclusion Criteria:\n\n* Obesity, defined as Body Mass Index (BMI) between 30 to 50 kilograms (kg)\u002Fsquare meter (m\\^2) at Screening\n* At least one self-reported, unsuccessful attempt at weight loss with lifestyle modification\n* Type 2 diabetes mellitus for at least 6 months prior to Screening, with glycated hemoglobin (HgbA1c) between 6.0% (42 millimole \\[mmol\\]\u002Fmole \\[mol\\]) and 9.5% (80 mmol\u002Fmol) at Screening, on a stable diabetes medication regimen for at least 3 months (select Part 2 and Part 3 cohorts only)\n* Willing, able and motivated to comply with all study assessments and adhere to the protocol schedule, including adherence to a stable diet and exercise routine for the duration of the study\n* No abnormal finding of clinical relevance at Screening that, in the opinion of investigator, could adversely impact participant safety or adversely impact study results\n* Participants of childbearing potential must agree to use highly effective contraception during the study and for at least 90 days following the end of the study or last dose of study medication, whichever is later. Participants must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study medication, whichever is later.\n\nExclusion Criteria:\n\n* Self-reported (or documented) weight gain or loss \\>5% within 3 months prior to Screening\n* Use of glucagon-like protein 1 receptor (GLP1R) agonists (liraglutide, semaglutide, etc.) for any indication within 6 months prior to Screening\n* Use of non-GLP1R medications for weight loss within 3 months prior to Screening, including but not limited to naltrexone\u002Fbupropion, orlistat, phentermine\u002Ftopiramate, and other prescription or over-the-counter medication or supplements taken for weight loss\n* Obesity attributable, in the investigator's opinion, to medication use, monogenic, or endocrinologic disorders (other than polycystic ovary syndrome)\n* History or prior surgical or device-based therapy for obesity\n* Use of medications strongly associated with weight gain within 3 months prior to Screening\n* Type 1 diabetes mellitus\n* History of hyperthyroidism or thyroid-stimulating hormone (TSH) levels \\\u003C0.4 or \\>6.0 milli-international units (mIU)\u002Fliter (L) at Screening\n* Evidence of clinically significant end-organ disease\n\nNote: Other Inclusion\u002FExclusion criteria may apply per protocol","65 Years",{"count":83,"type":19},180,[22,23],"This is a Phase 1\u002F2a double-blind dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of ARO-INHBE in adult participants with obesity (in Part 1), the safety, tolerability, PK, and PD of multiple doses of ARO-INHBE either as monotherapy, or in combination with tirzepatide, in adult participants with obesity with and without type 2 diabetes mellitus (in Part 2 and Part 3).",[87],"Obesity",{"date":68,"type":31},{"date":90,"type":31},"2024-12-04",{"date":92,"type":19},"2028-01-17",{"name":37,"class":38},5,{"id":96,"slug":4,"hasResults":10,"nctId":97,"briefTitle":98,"officialTitle":99,"acronym":4,"eligibilityCriteria":100,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":81,"enrollmentInfo":101,"targetDuration":4,"studyType":20,"phases":103,"briefSummary":104,"conditions":105,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":107,"lastUpdatePostDateStruct":108,"startDateStruct":110,"completionDateStruct":112,"leadSponsor":114,"locationsCount":94},"100611512","NCT07241546","Study of Inhaled ARO-RAGE in Allergen-induced Mild Asthma","A Randomized, Double-blind, Placebo-controlled Phase 2a Study of ARO-RAGE Inhalation Solution to Assess Efficacy on Small Airway Dysfunction in Allergen-induced Mild Asthma","Inclusion Criteria:\n\n* Clinically stable, mild atopic asthma (FEV1 ≥70% predicted)\n* Established allergy confirmed by positive skin prick test at screening\n* Willing and able to perform lung function tests and other study-related procedures\n* Participants of childbearing potential must consent to use a method of highly-effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last investigational product administration, whichever is later\n\nExclusion Criteria:\n\n* Concomitant diagnosis of a clinically important pulmonary disease other than asthma\n* Use of corticosteroids, immunosuppressives, or anti-inflammatory medications that interfere with inhaled challenges or inflammation, or chronic use of any other medication for treatment of allergic asthma\n* History or current medical condition contraindicating methacholine challenge\n\nNote: Additional inclusion\u002Fexclusion criteria may apply per protocol.",{"count":102,"type":19},36,[23],"The purpose of this study is to evaluate the impact of inhaled ARO-RAGE on the late asthmatic response (LAR) following an inhaled allergen challenge in participants with mild atopic asthma.",[106],"Asthma","2026-06-22",{"date":109,"type":31},"2026-06-23",{"date":111,"type":31},"2026-03-30",{"date":113,"type":19},"2028-04",{"name":37,"class":38},{"id":116,"slug":4,"hasResults":10,"nctId":117,"briefTitle":118,"officialTitle":119,"acronym":4,"eligibilityCriteria":120,"healthyVolunteers":121,"sex":15,"minAge":16,"maxAge":81,"enrollmentInfo":122,"targetDuration":4,"studyType":20,"phases":124,"briefSummary":125,"conditions":126,"keywords":4,"overallStatus":128,"whyStopped":4,"lastUpdateSubmitDate":129,"lastUpdatePostDateStruct":130,"startDateStruct":131,"completionDateStruct":132,"leadSponsor":134,"locationsCount":4},"100644354","NCT07662096","A First-In-Human Study of ARO-033 in Adult Participants","A First-In-Human Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of ARO-033 in Adult Participants","Key Inclusion Criteria:\n\n* Adults who are not pregnant, not breastfeeding, and do not plan to become pregnant (or impregnate their partners) during the study and for at least 90 days following the end of the study.\n* Body mass index (BMI) between 18.0 and 35.0 kilograms (kg)\u002Fsquare meter (m\\^2), inclusive.\n* No abnormal finding of clinical relevance at the Screening evaluation that in the opinion of the Investigator could adversely impact participant's safety during the study or adversely impact study results.\n\nKey Exclusion Criteria:\n\n* Human immunodeficiency virus (HIV) infection, as shown by the presence of anti-HIV antibody (seropositive).\n* Seropositive for hepatitis B virus (HBV) (hepatitis B surface antigen positive at screening) or hepatitis C virus (HCV) (HCV antibody positive with reflex confirmation using HCV RNA amplification at Screening). Cured HCV (positive antibody test without detectable HCV RNA) is permitted if HCV RNA has been negative for at least 2 years.\n* Uncontrolled hypertension (resting systolic blood pressure ≥160 millimeters of mercury \\[mmHg\\] or diastolic blood pressure ≥95 mmHg, confirmed by repeat measurement, at Screening).\n* Evidence of clinically significant immunocompromising condition (for example, primary immunodeficiency syndrome, aplastic anemia, known or suspected complement factor deficiency, or any other condition resulting in significantly impaired immune response as evidenced by recurrent infections), or recent\u002Fongoing treatment with immunosuppressive agents.\n* History of major surgery within 90 days of Screening.\n* Use of an investigational agent or device within 30 days or 5 half-lives (whichever is longer) prior to dosing or current participation in an investigational study. Participants recently participating in studies involving investigational agents with prolonged therapeutic effect (such as ribonucleic acid interference \\[RNAi\\] therapeutics, cell or gene therapies) should be discussed with the Medical Monitor.\n* Any medical condition or clinically significant laboratory abnormality at Screening that in the opinion of the Investigator should exclude the participant from participation, preclude safe and successful completion of the study, or confound study results.\n\nNote: Other protocol-defined inclusion and exclusion criteria may apply.",true,{"count":123,"type":19},42,[22],"This study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of ARO-033 compared to placebo in adult normal healthy volunteers (NHVs).",[127],"Healthy Participants","NOT_YET_RECRUITING","2026-06-18",{"date":109,"type":31},{"date":107,"type":19},{"date":133,"type":19},"2027-07-18",{"name":37,"class":38},{"id":136,"slug":4,"hasResults":10,"nctId":137,"briefTitle":138,"officialTitle":139,"acronym":4,"eligibilityCriteria":140,"healthyVolunteers":121,"sex":15,"minAge":16,"maxAge":141,"enrollmentInfo":142,"targetDuration":4,"studyType":20,"phases":144,"briefSummary":145,"conditions":146,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":129,"lastUpdatePostDateStruct":149,"startDateStruct":150,"completionDateStruct":152,"leadSponsor":154,"locationsCount":155},"100609958","NCT07221344","Study of ARO-MAPT-SC in Healthy Participants and Participants With Early Alzheimer's Disease","A Phase 1\u002F2a Placebo-Controlled Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-MAPT-SC in Healthy Subjects and Subjects With Early Alzheimer's Disease","Inclusion Criteria (All Participants):\n\n* Body mass index between 18.0 and 35.0 kilograms (kg)\u002Fsquare meter (m\\^2) at Screening\n* Not pregnant or breast-feeding\n* Able and willing to provide written informed consent prior to the performance of any study specific procedures\n* Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later; participants must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later\n\nInclusion Criteria (Alzheimer's Disease):\n\n* Adults aged 50 to 80 years of age with a clinical diagnosis of early AD and plasma, CSF, or imaging biomarkers consistent with the diagnosis\n* If participant is on AD medications, the doses must be stable for ≥weeks prior to Screening.\n\n  a. Participants with early AD are not required to be on AD medications.\n* Have a reliable and competent caregiver or trial partner who is ≥18 years of age, able and willing to accompany the participant to study visits involving informant-based assessments, to be available to site staff by telephone as needed, and in the opinion of the Investigator, be sufficiently familiar with the participant throughout the study in order to provide accurate and reliable information relevant to study outcome measures\n\nExclusion Criteria (All Participants):\n\n* Blood pressure outside of specified range in the protocol\n* Human immunodeficiency virus (HIV) infection (seropositive at Screening)\n* Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening\n* Intellectual disability or significant behavioral neuropsychiatric manifestation\n* Clinically significant cardiac, liver, or renal disease\n* Any contraindications to lumbar puncture\n* Known allergy or possible allergy to either ARO-MAPT-SC or to its excipients\n\nNote: Additional inclusion\u002Fexclusion criteria may apply per protocol.","80 Years",{"count":143,"type":19},112,[22,23],"Study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of ARO-MAPT-SC compared to placebo in adult healthy volunteers and in participants with early Alzheimer's disease (AD), defined as mild cognitive impairment due to AD and mild AD dementia.",[147,148],"Alzheimer Disease","Alzheimer Disease, Early Onset",{"date":109,"type":31},{"date":151,"type":31},"2025-11-18",{"date":153,"type":19},"2027-06",{"name":37,"class":38},3,{"id":157,"slug":4,"hasResults":10,"nctId":158,"briefTitle":159,"officialTitle":160,"acronym":4,"eligibilityCriteria":161,"healthyVolunteers":10,"sex":15,"minAge":162,"maxAge":4,"enrollmentInfo":163,"targetDuration":4,"studyType":20,"phases":165,"briefSummary":166,"conditions":167,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":169,"lastUpdatePostDateStruct":170,"startDateStruct":172,"completionDateStruct":174,"leadSponsor":176,"locationsCount":177},"100595847","NCT07037771","A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)","Phase 3 Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)","Inclusion Criteria:\n\n* Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study\n* Body weight ≥35 kg at Screening as patients could theoretically be \\\u003C35 kg as the study continues.\n* HoFH based on a supportive genetic test or a clinical diagnosis (total cholesterol \\>500 mg\u002FdL\\[13 mmol\u002FL\\] OR treated LDL-C concentration of ≥300 mg\u002FdL \\[≥8 mmol\u002FL\\] either accompanied by TGs \\\u003C300 mg\u002FdL \\[3.4 mmol\u002FL\\] AND both parents with documented total cholesterol \\>250 mg\u002FdL \\[6.5 mmol\u002FL\\] OR cutaneous or tendinous xanthoma before 10 years of age)\n* LDL-C ≥70 mg\u002FdL (1.8 mmol\u002FL). For adolescents 12 to \\\u003C18 years of age, screening LDL-C ≥116 mg\u002FdL (3 mmol\u002FL).\n* Hemoglobin A1c (HbA1c) ≤9.5%\n* Total bilirubin \\\u003C2xULN, unless in previously confirmed cases of Gilbert's syndrome\n* Alanine aminotransferase or aspartate aminotransferase \\\u003C3×ULN\n* On standard of care, maximally tolerated lipid-lowering therapy to include a maximally tolerated statin, ezetimibe, and a PCSK9 inhibitor\n\nExclusion Criteria:\n\n* Use of a hepatocyte-targeted siRNA within 365 days before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)\n* Use of an antisense oligonucleotide molecule within 3 months before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)\n* Use of evinacumab within 3 months before Day 1. Evinacumab use is prohibited during the study.\n* Non-response to evinacumab, defined as LDL-C reduction \\\u003C15% from baseline after 2 doses\n* Use of any other investigational agent or device within 30 days or 5 half-lives (whichever is longer) before Day 1\n* Use of systemic corticosteroids (unless used as replacement therapy for pituitary\u002Fadrenal disease with a stable regimen)\n* Estimated glomerular filtration rate \\\u003C30 mL\u002Fmin\n\nNOTE: Additional Inclusion\u002Fexclusion criteria may apply per protocol","12 Years",{"count":164,"type":19},60,[62],"This multicenter, randomized, placebo-controlled study will evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous familial hypercholesterolemia (HoFH). After completion of the double blind (DB) treatment period subjects will be eligible to continue in the optional open-label extension (OLE) period of the study. All placebo subjects who opt to continue will transition to active drug during the OLE Period.",[168],"Homozygous Familial Hypercholesterolemia","2026-06-09",{"date":171,"type":31},"2026-06-10",{"date":173,"type":31},"2025-06-17",{"date":175,"type":19},"2027-08-20",{"name":37,"class":38},43,{"id":179,"slug":4,"hasResults":10,"nctId":180,"briefTitle":181,"officialTitle":182,"acronym":183,"eligibilityCriteria":184,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":185,"targetDuration":4,"studyType":20,"phases":187,"briefSummary":188,"conditions":189,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":191,"lastUpdatePostDateStruct":192,"startDateStruct":194,"completionDateStruct":196,"leadSponsor":198,"locationsCount":199},"100579322","NCT06822790","Long-Term Safety and Efficacy of Plozasiran in Adults With Hypertriglyceridemia","A Phase 3 Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Plozasiran in Adults With Hypertriglyceridemia (SHASTA-10 Study)","SHASTA-10","Inclusion Criteria:\n\n* Adult males, or nonpregnant (who do not plan to become pregnant), nonlactating adult females, who are able and willing to provide written informed consent prior to the performance of any study-specific procedures\n* Completed all required study visits per protocol in the parent study\n* Female subjects of childbearing potential must agree to use a highly effective method of contraception during the study and for at least 90 days after the End of Study (EOS) or the last dose of plozasiran, whichever is later. Male subjects must agree to use a condom during the study and for at least 90 days after the EOS or last dose of plozasiran whichever is later. Subjects must not donate sperm or eggs during the study and for at least 90 days after the EOS or last dose of plozasiran, whichever is later. Female subjects of childbearing potential on hormonal contraceptives must be stable on medication for \\>1 menstrual cycle prior to Day 1.\n* Subjects must be on standard of care lipid and TG-lowering medications per local guidelines (unless documented as intolerant as determined by the Investigator, including an inability to safely administer or re-administer a specific drug because of fear, preference, genetic, clinical, or metabolic considerations, or due to a previous adverse reaction associated with, attributed to, or caused by specific drug)\n* If the subject has a medical history of clinical atherosclerotic cardiovascular disease (ASCVD) or elevated 10-year ASCVD risk (eg, ≥7.5% per American Heart Association\u002FAmerican College of Cardiology \\[AHA\u002FACC\\] risk calculator for subjects ≥40 years of age or Framingham risk score calculator for subjects under the age of 40), the subject must be on appropriate lipid-lowering therapy as per local standard of care (ie, including moderate-to-high intensity statin, as indicated).\n\nIf the subject has diabetes:\n\n1. Subject must be on optimized antidiabetic regimen as defined by the local standards, Investigator, and institutional practices\n2. Subject must have no events of diabetic ketoacidosis, diabetic decompensation\u002F hyperosmolar hyperglycemic nonketotic coma, diabetes complications, recurrent infections, or hospitalization related to poor glycemic control within 24 weeks of the Day 1 visit - Willing to follow diet counseling and maintain a stable low-fat diet\n\nSubjects in the USA and Canada who completed protocol AROAPOC3-2003 meeting all eligibility criteria (with the exception of inclusion criteria #9 which is not applicable to these subjects) who also meet the following additional criteria may enroll in this trial:\n\n* HbA1c ≤10% within 30 days prior to Day 1\n* Completed AROAPOC3-2001 prior to entry into AROAPOC3-2003 AND either (c) or (d) below:\n* Baseline fasting TG level of ≥500 mg\u002FdL and prior history of acute pancreatitis at the time of enrollment into AROAPOC3-2001\n* Baseline fasting TG level of ≥1000 mg\u002FdL at the time of enrollment into AROAPOC3-2001\n\n  * Subjects who previously met all eligibility requirements for AROAPOC3-3003, or AROAPOC3-3004 and were not permitted to proceed to randomization per Sponsor's direction in order to prevent excessive over-enrollment may also be enrolled in this trial. These subjects must meet all eligibility criteria prior to enrollment (with the exception of inclusion criteria #2 and #8 which are not applicable to these subjects) and have an HbA1c ≤10% within 30 days of Day 1.\n\nExclusion Criteria:\n\n* Subject was permanently discontinued from receiving plozasiran in the parent study due to elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) or due to HbA1c elevation that did not respond to antidiabetic regimen\n* Subject withdrew consent for continued study treatment in the parent study\n* Known hypersensitivity to the active substance or to any of the excipients of plozasiran\n* Known hypersensitivity to the active substance or to any of the excipients of plozasiran\n* Any new condition or worsening of existing condition or any other situation that in the Investigator's judgment, would make the subject unsuitable for enrollment, could interfere with the subject participating in or completing the study, would make it difficult to comply with protocol requirements, or put the subject at an additional safety risk\n* Unwilling to limit alcohol consumption to within moderate limits for the duration of the study.\n* Poorly controlled glycemia (ie, HbA1c \\>10%) based upon the most recent HbA1c level reported in the parent trial prior to Day 1\n* Acute pancreatitis within 4 weeks prior to Day 1\n* Use of any hepatocyte-targeted siRNA that targets lipids and\u002For triglycerides within 365 days before Day 1 (except plozasiran or inclisiran, which are permitted). Administration of inclisiran must be separated from administration of plozasiran by at least 4 weeks throughout the treatment period\n* Use of any other hepatocyte targeted siRNA or antisense oligonucleotide molecule within 60 days or within 5 half-lives before Day 1 based on plasma PK, whichever is longer.\n* Use of an investigational agent (other than plozasiran) or device within 30 days or within 5 half-lives, based on plasma PK, whichever is longer, prior to Day 1 (V1) or current participation in an interventional investigational study.\n* Recent unstable or symptomatic cardiac arrhythmia (including any associated medication changes) within 90 days prior to Day 1. Individuals with stable well-controlled atrial arrhythmias will be allowed to participate in the study.\n* Uncontrolled hypertension (ie, seated systolic blood pressure \\>160 mmHg and\u002For diastolic blood pressure \\>100 mmHg) at Day 1; subject may be re-evaluated when hypertension is controlled.\n\nNote: Other inclusion\u002Fexclusion criteria may apply per protocol",{"count":186,"type":19},869,[62],"This is an open-label study to be conducted in adults with hypertriglyceridemia (HTG) and severe hypertriglyceridemia (SHTG). Each participant must have completed all required visits per protocol in the parent study AROAPOC3-2003 (USA and Canada participants only; NCT# 05413135), AROAPOC3-3001(Canada and Japan participants only; NCT05089084), AROAPOC3-3003 (NCT06347003), AROAPOC3-3004 (NCT06347016) or AROAPOC3-3009 (Argentina, Italy, South Africa, and Spain; NCT06347133).\n\nSubjects who previously met all eligibility requirements for AROAPOC3-3003 or AROAPOC3-3004 and were not permitted to proceed to randomization per the Sponsor's direction in order to prevent excessive over-enrollment may also be enrolled in this trial. The subjects must meet all other applicable eligibility criteria prior to enrollment and have an HbA1c results of \\\u003C=10% within 30 days prior to Day 1.\n\nSubjects entering this OLE from AROAPOC3-2003 must meet the following additional criteria to be considered for enrollment in addition to applicable eligibility criteria:\n\n1. HbA1c ≤10% within 30 days prior to Day 1\n2. Completed AROAPOC3-2001 prior to entry into AROAPOC3-2003 AND fulfill either (c) or (d)\n3. Baseline fasting TG level of ≥500 mg\u002FdL and prior history of acute pancreatitis at the time of enrollment into AROAPOC3-2001\n4. Baseline fasting TG level of ≥1000 mg\u002FdL at the time of enrollment into AROAPOC3-2001\n\nAll eligible participants will receive plozasiran administered subcutaneously (SC) approximately every 3 months for 24 months. Participants will be counseled to remain on the specified low-fat diet throughout the study in accordance with local standard of care.",[190],"Hypertriglyceridemia","2026-05-30",{"date":193,"type":31},"2026-06-02",{"date":195,"type":31},"2025-04-09",{"date":197,"type":19},"2028-07",{"name":37,"class":38},275,{"id":201,"slug":4,"hasResults":10,"nctId":202,"briefTitle":203,"officialTitle":204,"acronym":205,"eligibilityCriteria":206,"healthyVolunteers":10,"sex":15,"minAge":162,"maxAge":207,"enrollmentInfo":208,"targetDuration":4,"studyType":20,"phases":210,"briefSummary":211,"conditions":212,"keywords":213,"overallStatus":128,"whyStopped":4,"lastUpdateSubmitDate":226,"lastUpdatePostDateStruct":227,"startDateStruct":229,"completionDateStruct":231,"leadSponsor":233,"locationsCount":4},"100629374","NCT07473843","Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia","Phase 3 Single-Arm Open-Label Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent Participants (Age 12 to \u003C18 Years) With Homozygous Familial Hypercholesterolemia (SPRUCE)","SPRUCE","Inclusion Criteria:\n\n* Adolescents 12 to \\\u003C18 years of age who are nonpregnant, nonlactating, and do not plan to become pregnant during the study\n* Body weight ≥35 kilograms (kg) at screening\n* HoFH based on a supportive genetic test (from a source-verifiable medical record or based on screening genotype) or clinical diagnosis\n* Screening LDL-C ≥116 mg\u002FdL (3 mmol\u002FL)\n* Screening hemoglobin A1c (HbA1c) ≤9.5%\n* Total bilirubin \\\u003C2×upper limit of normal (ULN), unless in previously confirmed cases of Gilbert's syndrome\n* Alanine aminotransferase or aspartate aminotransferase \\\u003C3×ULN\n\nExclusion Criteria:\n\n* Use of a hepatocyte-targeted siRNA within 365 days before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)\n* Use of an antisense oligonucleotide molecule within 3 months before Day 1\n* Use of evinacumab within 3 months before Day 1\n* Non-response to evinacumab, defined as LDL-C reduction \\\u003C15% from baseline after 2 doses\n* Use of systemic corticosteroids (unless used as replacement therapy for pituitary\u002Fadrenal disease with a stable regimen)\n\nNOTE: Additional inclusion\u002Fexclusion criteria may apply per protocol","17 Years",{"count":209,"type":19},12,[62],"This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to \\\u003C18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and low-density lipoprotein cholesterol (LDL-C) ≥116 milligrams per deciliter (mg\u002FdL) on maximally tolerated lipid-lowering therapy.",[168],[214,215,216,217,218,219,220,221,222,223,224,168,225],"Hyperlipoproteinemia Type II","Lipid Metabolism, Inborn Errors","Metabolism, Inborn Errors","Genetic Diseases, Inborn","Congenital, Hereditary, and Neonatal Diseases and Abnormalities","Hyperlipoproteinemias","Hyperlipidemias","Dyslipidemias","Lipid Metabolism Disorders","Metabolic Diseases","Nutritional and Metabolic Diseases","Zodasiran","2026-05-27",{"date":228,"type":31},"2026-05-29",{"date":230,"type":19},"2026-08",{"date":232,"type":19},"2028-08",{"name":37,"class":38},{"id":235,"slug":4,"hasResults":10,"nctId":236,"briefTitle":237,"officialTitle":238,"acronym":4,"eligibilityCriteria":239,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":81,"enrollmentInfo":240,"targetDuration":4,"studyType":20,"phases":242,"briefSummary":243,"conditions":244,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":246,"lastUpdatePostDateStruct":247,"startDateStruct":249,"completionDateStruct":251,"leadSponsor":253,"locationsCount":254},"100588116","NCT06937203","A First-In-Human Study of ARO-ALK7 in Adults With Obesity With and Without Type 2 Diabetes Mellitus","A Phase 1\u002F2A Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ARO-ALK7 in Adult Volunteers With Obesity With and Without Type 2 Diabetes Mellitus","Inclusion Criteria:\n\n* Obesity, defined as BMI between 30-50 kg\u002Fm2, inclusive, with weight at Screening not to exceed 159 kg (350 lbs)\n* At least one self-reported, unsuccessful attempt at weight loss with lifestyle modification\n* No abnormal finding of clinical relevance at Screening that could adversely impact participant safety during the study or adversely impact study results\n* Participants of childbearing potential must agree to use highly effective contraception during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study for at least 90 days following the end of the study or last dose of study drug, whichever is later\n\nExclusion Criteria:\n\n* Self-reported (or documented) weight gain or loss \\>5% within 3 months prior to Screening\n* Use of GLP-1RAs (liraglutide, semaglutide, etc.) for any indication within 6 months prior to Screening\n* Use of non-GLP-1R medications for weight loss within 3 months prior to Screening including but not limited to naltrexone\u002Fbupropion, orlistat, phentermine\u002Ftopiramate and other prescription or over-the-counter medication or supplement taken for weight loss purposes\n* Obesity attributable primarily in the Investigator's opinion to medication use, monogenic or endocrinologic disorders (other than polycystic ovary syndrome)\n* History of prior surgical or device-based therapy for obesity (including endoscopic bariatric procedures)\n* Use of medications or therapies strongly associated with weight gain, alterations in body composition, or increase in muscle mass, within 3 months prior to Screening\n* Type 1 diabetes mellitus\n\nNote: Additional inclusion\u002Fexclusion criteria may apply per protocol",{"count":241,"type":19},138,[22,23],"This is a Phase 1\u002F2a double-blind dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple doses of ARO-ALK7 in adult participants with obesity without Type 2 Diabetes Mellitus (T2DM) (Part 1), and the safety, tolerability and PD of multiple doses of ARO-ALK7 in adult participants with obesity with and without T2DM, either as monotherapy or in combination with tirzepatide (Part 2).",[87,245],"Diabetes Mellitus, Type 2","2026-04-07",{"date":248,"type":31},"2026-04-13",{"date":250,"type":31},"2025-05-09",{"date":252,"type":19},"2026-10",{"name":37,"class":38},8,{"id":256,"slug":4,"hasResults":10,"nctId":257,"briefTitle":258,"officialTitle":259,"acronym":4,"eligibilityCriteria":260,"healthyVolunteers":10,"sex":15,"minAge":261,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":262,"phases":4,"briefSummary":263,"conditions":264,"keywords":4,"overallStatus":267,"whyStopped":4,"lastUpdateSubmitDate":268,"lastUpdatePostDateStruct":269,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":271,"locationsCount":272},"100577294","NCT06796426","Treatment Protocol of Plozasiran in Adults With High-Risk Severe Hypertriglyceridemia (SHTG) and in Adults and Adolescents With FCS","A Treatment Protocol for the Use of Plozasiran in Adults With Familial Chylomicronemia Syndrome (FCS). A Single Patient Protocol for the Use of Plozasiran In Adolescents With Familial Chylomicronemia Syndrome (FCS). An Expanded Access Treatment Protocol for Use of Plozasiran In Adults With High-Risk Severe Hypertriglyceridemia (SHTG).","FCS Inclusion Criteria:\n\n* ≥ 15 years of age\n* Fasting TG levels ≥ 880 mg\u002FdL that are not sufficiently controlled on standard lipid-lowering therapy\n* Established diagnosis of FCS based on documented history of fasting TG levels in excess of 1000 mg\u002FdL on repeated testing (for at least 3 prior occasions), and at least 1 of the following: a supportive genetic test, documented history of recurrent episodes of acute pancreatitis not caused by alcohol or cholelithiasis, documented history of recurrent hospitalizations for severe abdominal pain without other explainable cause, documented history of childhood pancreatitis, family history of hypertriglyceridemia-induced pancreatitis\n* Willing to follow dietary counseling based on local standard of care, consistent with an intake of ≤ 20 g of fat per day\n* If on medications for management of type 2 diabetes the dosing regimen must be stable.\n* Participants of childbearing potential must agree to use a highly effective form of contraception in addition to a male condom during the program and for at least 90 days after the last dose of plozasiran\n\nSHTG Inclusion Criteria:\n\n* Established diagnosis of high risk SHTG and documented evidence (medical history) of either: fasting triglycerides (TG) ≥ 880 mg\u002FdL OR fasting TG levels of ≥ 500 mg\u002FdL AND history of recurrent episodes of acute pancreatitis not caused by alcohol or cholelithiasis\n* Hemoglobin A1c (HbA1c) ≤ 9.0% at Day 1\n* Fasting low density lipoprotein cholesterol (LDL-C) ≤130 mg\u002FdL (≤ 3.37 mmol\u002FL) at Day 1\n* Willing to follow dietary counseling and maintain stable, low-fat diet Participant must be on standard of care lipid and TG-lowering medications per local guidelines,\n* If the participant has a medical history of clinical atherosclerotic cardiovascular disease (ASCVD) or an elevated 10-year ASCVD risk, the participant must be on appropriate lipid-lowering therapy as per local standard of care prior to collection of qualifying TG levels\n* Females of childbearing potential must agree to use a highly effective form of contraception and males must agree to use a condom during the program and for at least 90 days after the last dose of plozasiran\n\nFCS Exclusion Criteria:\n\n* Diabetes mellitus with any of the following at Day 1: newly diagnosed within the past 24 weeks, HbA1c ≥ 9.0% within the past 4 weeks, meaningful medical events relating to poor glycemic control, changes in basal insulin regimen of more than =\u002F- 10 units within 12 weeks if insulin-dependent\n* Clinical evidence of primary hypothyroidism, primary subclinical hypothyroidism, or secondary hypothyroidism\n* History of bleeding diathesis or coagulopathy\n* Current diagnosis of nephrotic syndrome\n* Eligible to receive any commercially available FDA-approved therapeutic for treatment of FCS unless proven to be ineffective or judged inappropriate by the treating physician\n* History of acute coronary syndrome events (adults with FCS only)\n* New York Heart Association Class III or IV heart failure or last known ejection fraction of \\\u003C30% (adults with FCS only)\n* History of stroke, transient ischemic attack, or peripheral artery disease within 24 weeks of first dose (adults with FCS only)\n\nSHTG Exclusion Criteria:\n\n* Eligible for approved Redemplo®.\n* Untreated or inadequately treated hypothyroidism or hypothyroidism\n* Eligible for any current study of plozasiran\n* History of acute coronary syndrome events\n* New York Heart Association Class IV heart failure or last known ejection fraction of \\\u003C 30%\n* Known clinically significant blood dyscrasia\n\nNote: Additional inclusion\u002Fexclusion criteria may apply per protocol","15 Years","EXPANDED_ACCESS","This is a treatment program for the use of plozasiran in adults (AROAPOC3-EAP-002) and adolescents (AROAPOC3-EAP-003) with familial chylomicronemia syndrome (FCS) as well as in adults (AROAPOC3-EAP-004) with high risk severe hypertriglyceridemia (SHTG).\n\nThe program will enroll eligible patients ≥ 15 years of age, with fasting triglycerides (TGs) ≥ 880 mg\u002FdL (≥ 10 mmol\u002FL) that is not adequately controlled with standard lipid-lowering therapy, and with a diagnosis of FCS. Patients will receive 25 mg of plozasiran by subcutaneous (sc) injection on Day 1 and every 3 months for a total of 5 injections. The duration of the program is 15 months.\n\nThe program will also enroll eligible patients ≥18 years of age, with fasting TGs \\> 880 mg\u002FdL (\\> 9.94 mmol\u002FL), or fasting TGs \\> 500 mg\u002FdL plus a history of acute pancreatitis, that are not adequately controlled with standard lipid-lowering therapy, and with a diagnosis of high risk SHTG. SHTG patients will receive 25 mg of plozasiran by sc injection on Day 1 and every 3 months for a total of 7 injections. The duration of the program is 21 months.",[265,266],"Familial Chylomicronemia","High Risk Severe Hypertriglyceridemia (SHTG)","AVAILABLE","2026-03-02",{"date":270,"type":31},"2026-03-03",{"name":37,"class":38},1,{"id":274,"slug":4,"hasResults":10,"nctId":275,"briefTitle":276,"officialTitle":277,"acronym":4,"eligibilityCriteria":278,"healthyVolunteers":10,"sex":15,"minAge":279,"maxAge":280,"enrollmentInfo":281,"targetDuration":4,"studyType":20,"phases":282,"briefSummary":283,"conditions":284,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":286,"lastUpdatePostDateStruct":287,"startDateStruct":289,"completionDateStruct":291,"leadSponsor":293,"locationsCount":294},"100526226","NCT06131983","Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1","A Phase1\u002F2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1","Inclusion Criteria:\n\n* Genetically confirmed FSHD1 based on Screening evaluation or source verifiable medical record\n* Clinical severity score between 3 and 8 (scale, 0 to 10)\n* Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader\n* A 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety in the study\n* Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 12 weeks following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 12 weeks following the end of study or last dose of study medication, whichever is later.\n\nExclusion Criteria:\n\n* Human Immunodeficiency Virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at Screening\n* Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening\n* Uncontrolled hypertension\n* Severe cardiovascular disease\n* History of thrombolic events\n* Platelet count less that the lower limit of normal at Screening\n* History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.\n* Any contraindication to muscle biopsy or MRI\n\nNote: additional inclusion\u002Fexclusion criteria may apply per protocol","16 Years","70 Years",{"count":164,"type":19},[22,23],"The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 4 doses of ARO-DUX4 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose MRI-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.",[285],"Facio-Scapulo-Humeral Dystrophy","2026-02-04",{"date":288,"type":31},"2026-02-06",{"date":290,"type":31},"2024-02-22",{"date":292,"type":19},"2026-12",{"name":37,"class":38},17,{"id":296,"slug":4,"hasResults":10,"nctId":297,"briefTitle":298,"officialTitle":299,"acronym":4,"eligibilityCriteria":300,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":81,"enrollmentInfo":301,"targetDuration":4,"studyType":20,"phases":302,"briefSummary":303,"conditions":304,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":306,"lastUpdatePostDateStruct":307,"startDateStruct":309,"completionDateStruct":311,"leadSponsor":312,"locationsCount":51},"100526745","NCT06138743","Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy","A Phase 1\u002F2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years","Inclusion Criteria:\n\n* Genetically confirmed diagnosis of DM1\n* Clinician-assessed signs of DM1 including clinically apparent myotonia\n* Onset of DM1 symptoms occurred after the age of 12 years\n* Walk for at least 10 meters independently at Screening\n* Subjects of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Subjects must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later.\n\nExclusion Criteria:\n\n* Inadequately controlled diabetes\n* Confirmed diagnosis of congenital DM1\n* Uncontrolled hypertension\n* History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period\n* Clinically significant cardiac, liver or renal disease\n* HIV infection (seropositive) at Screening\n* Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at screening\n* Untreated or poorly controlled epilepsy\n* Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to Screening.\n* Abnormal coagulation parameters at Screening including platelet count, international normalized ratio (INR), prothrombin time, and activated partial thromboplastin time (APTT)\n\nNote: Additional inclusion\u002Fexclusion criteria may apply per protocol",{"count":18,"type":19},[22,23],"This is a phase 1\u002F2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple ascending doses of ARO-DM1 compared to placebo in male and female subjects with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of ARO-DM1 or placebo.",[305],"Myotonic Dystrophy 1","2025-11-06",{"date":308,"type":31},"2025-11-10",{"date":310,"type":31},"2024-03-04",{"date":292,"type":19},{"name":37,"class":38},""]