Clinical trials

Title: Effect of Pediatric Lung Ultrasound on Antibiotic Prescriptions in Hospitalized Children and Adolescents with Lower Respiratory Tract Infections (PLUS-AP Trial) Purpose of the Study: This study is being done to find out if a type of imaging called lung ultrasound (LUS) can help doctors decide when to stop antibiotics in children and teens who are in the hospital with lung infections, like pneumonia. Antibiotics are often given to these patients, but sometimes they are used for longer than necessary, which can be harmful. The investigators want to see if using LUS helps doctors make decisions that reduce unnecessary antibiotic use, which can help prevent antibiotic resistance and other complications. What is Lung Ultrasound (LUS)? Lung ultrasound is a safe, non-invasive, and painless test that uses sound waves to look at the lungs. It doesn't involve radiation like chest X-rays. It is already being used to help doctors understand lung conditions, and this study will test if it can also help in deciding when to stop antibiotics. How the Study Works: Participants: Children and adolescents (ages 3 months-18 years) who are admitted to the hospital with a lung infection. Groups: LUS Group: These patients will have a lung ultrasound within 24 hours of enrollment. The results will help guide decisions about when to stop antibiotics. Standard Care Group: These patients will receive regular hospital care, which may include chest X-rays and other tests to guide their treatment. What Will Be Measured? Primary Goal: The study will measure how long children and teens stay on antibiotics. The investigators are testing whether LUS can help shorten this time without affecting their recovery. Secondary Goals: Investigators will also look at how long patients stay in the hospital, whether they need further treatment or care after leaving. Why is This Important? If this study shows that LUS can safely reduce the use of antibiotics, it could change how doctors treat lung infections in children. Reducing unnecessary antibiotic use can help fight antibiotic resistance and protect children from side effects of unnecessary treatments.

A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: * Participants receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit from continuing treatment with luspatercept * Participants in the follow-up phase previously treated with luspatercept or placebo in the parent protocol will continue into long-term post-treatment follow-up in the rollover study until the follow-up commitments are met * The study design is divided into the Transition Phase, Treatment Phase and Follow-up Phase. Participants will enter transition phase and depending on their background will enter either the treatment phase or the Long-term Post-treatment Follow-up (LTPTFU) phase * Transition Phase is defined as one Enrollment visit * Treatment Phase: For participants in luspatercept treatment the dose and schedule of luspatercept in this study will be the same as the last dose and schedule in the parent luspatercept study. This does not apply to participants that are in long-term follow-up from the parent protocol * Follow-up Phase includes: \- 42 Day Safety Follow-up Visit * During the Safety Follow up, the participants will be followed for 42 days after the last dose of luspatercept, for the assessment of safety-related parameters and adverse event (AE) reporting \- Long-term Post-treatment Follow-up (LTPTFU) Phase * Participants will be followed for overall survival every 6 months for at least 5 years from first dose of luspatercept in the parent protocol, or 3 years of post-treatment from last dose, whichever occurs later, or until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. Participants will also be monitored for progression to AML or any malignancies/pre-malignancies. New anticancer or disease related therapies should be collected at the same time schedule Participants transitioning from a parent luspatercept study in post-treatment follow-up (safety or LTPTFU) will continue from the same equivalent point in this rollover study. The ACE-536-LTFU-001 rollover study will be terminated, and relevant participants will discontinue from the study when all participants fulfill 5 years on the study, including treatment and follow-up.

The primary goal of this randomized clinical trial (RCT) is to evaluate whether Kinesio Taping versus McConnell Taping, when integrated into a standardized hip-knee strengthening and neuromuscular control program, produces significant effects in individuals aged 18-50 with patellofemoral pain syndrome (PFPS). The main question it aims to answer is: Does Kinesio Taping compared with McConnell Taping when both combined with a standardized hip-knee exercise program, produce different effects on quadriceps neuromuscular activation (VMO-VL sEMG), pain, functional knee pain, and dynamic knee valgus over a 6 week period intervention? Participants will be divided into three groups for comparison: the control group will receive a structured hip-knee physical therapy exercise program without any taping intervention, Kinesio Taping group with a standardized exercise program, and a McConnell Taping group with a standardized exercise program.

This randomized controlled clinical trial aims to evaluate a minimally invasive technique for peri-implant soft tissue augmentation using microneedling combined with injectable platelet-rich fibrin (i-PRF) compared with the current gold-standard connective tissue graft (CTG). Patients with thin peri-implant soft tissue requiring implant second-stage surgery in the esthetic zone will be randomly assigned to receive either CTG harvested from the palate or a series of microneedling and i-PRF treatments. Clinical outcomes including mucosal thickness, keratinized tissue width, peri-implant health parameters, volumetric soft tissue changes, esthetic outcomes, and patient-reported measures of pain and satisfaction will be evaluated during follow-up. The study aims to determine whether microneedling with i-PRF can provide comparable soft tissue augmentation while reducing patient morbidity associated with connective tissue graft harvesting.

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes. The objectives of the Registry are: * To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Pompe disease population. * To evaluate the long-term effectiveness of alglucosidase alfa.

Roxadustat is a licensed medicine to treat anemia in adults with chronic kidney disease (CKD). Anemia is a low level of red blood cells. Current treatment for anemia is to have injections of medicines called erythropoietin stimulating agents (also known as ESAs) to help the bone marrow make more red blood cells. These are often given together with iron. This treatment is also available to children and teenagers with CKD. However, there are some safety concerns with ESAs. Also, as roxadustat is taken orally, this may be another option for treating anemia in children and teenagers with CKD. In this study, children and teenagers with CKD and anemia will take roxadustat for up to 52 weeks to treat their anemia. The main aim of the study is to learn how roxadustat affects anemia in children and teenagers with CKD. This is an open-label study which means the children and teenagers in the study and the clinic staff know they will be taking roxadustat. In this study, the children and teenagers with CKD who need treatment for anemia can take part. Those currently being treated with an ESA will be switched to roxadustat. Those who have not been treated with an ESA can start on roxadustat straight away. All children and teenagers in the study will take roxadustat 3 times a week for up to 52 weeks (1 year). They will start on a fixed dose of roxadustat for 4 weeks. Blood samples will be taken regularly to check hemoglobin levels. The roxadustat dose may be changed if the blood levels of hemoglobin are too high, too low, or change too quickly. After 4 weeks the dose may be changed, if needed, to keep blood levels of hemoglobin in the blood to just below the normal range. Firstly, teenagers will take roxadustat. 10 teenagers will take their fixed dose of roxadustat for 4 weeks. They will give blood samples to help the researchers work out the most suitable dose for the rest of the teenagers in the study. When the rest of the teenagers start taking roxadustat at the most suitable dose for teenagers, 10 children will take roxadustat for 4 weeks. These 10 children will give blood samples to help the researchers work out the most suitable dose for the rest of the children in the study. Then, the rest of the children will take roxadustat at the most suitable dose for children. There will be many clinic visits during the study. Overnight hospital stays are not expected. There will be 1 visit every 2 weeks for the first 4 weeks of taking roxadustat, then every 4 weeks until the end of treatment. Finally there is 1 visit 4 weeks after treatment has finished. During most visits, the children and teenagers will have their vital signs checked (blood pressure, body temperature and heart rate). Fluid status (how much water is in the body) will also be checked for those who need dialysis. The children and teenagers will also have blood tests and the study doctors will check for any medical problems. The children and teenagers will have a medical examination before their first dose of roxadustat and again at about 24-week (6-month) and 52-week (13-month) visits. They will have an electrocardiogram (ECG) before their first dose of roxadustat and again at the 12-week, 24-week, 36-week, and 52-week visit. They will also have urine tests at the 4-week, 24-week and 52-week visits. At the 52-week visit, the children and teenagers will also have blood tests for hemoglobin and iron levels. The study doctors will also check for any medical problems.

This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years.

The tobacco use burden in Lebanon is exceptionally high: 35% of adults are current cigarette smokers and 39% are current waterpipe smokers. Although the World Health Organization endorses evidence-based interventions for population-level tobacco dependence treatment, recommended treatments are not integrated as a routine part of primary care in Lebanon, as is the case in other low-resource settings. The objective of this proposal is to evaluate the comparative effectiveness of promising multi-component interventions for implementing evidence-based cessation treatment in Lebanon's national system of primary health care centers.

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

This is a long term safety study for patients who have completed a Novartis sponsored asciminib study and are judged by the investigator to benefit from continued treatment

This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator.

The goal of this clinical trial is to learn if the erector spinae block can reduce the incidence of chronic pain compared to the paravertebral block in adult patients undergoing total mastectomy. The study includes patients aged 18 to 80 years scheduled for mastectomy, with or without axillary dissection. The main questions it aims to answer are: * Does erector spinae block reduce the incidence of chronic pain at 3 months after mastectomy compared to paravertebral block? * Does erector spinae block affect postoperative outcomes such as opioid consumption at 48 hours, pain scores (in PACU, 24 and 48 hours), block performance time, and the incidence of complications, anxiety or depression, and pain intensity at 3 months? Researchers will compare patients receiving erector spinae block to those receiving paravertebral block to determine if erector spinae block provides equivalent or improved outcomes in terms of chronic pain and perioperative measures. Participants will: * Be randomly assigned to receive either erector spinae block or paravertebral block prior to surgery * Undergo total mastectomy (with or without axillary dissection) * Have their pain assessed in the PACU and at 24 and 48 hours postoperatively * Have opioid consumption measured during the first 48 hours after surgery * Be followed up at 3 months to assess chronic pain, pain intensity, and psychological outcomes (anxiety or depression)

Impedance can have an impact on nerve block during loco-regional anesthesia, particularly when using techniques such as nerve stimulation or ultrasound guidance to locate and block specific nerves. Impedance refers to the resistance to electrical current flow within tissue, and it can affect the ability to stimulate nerves or visualize them using ultrasound. The aim of this research is to assess the impedance across different tissue type during an axillary peripheral nerve block (skin, fat, fascia, muscle, nerve proximity). The results of this study would help clinicians performing nerve block to enhance the precision of needle placement, thus increase the success rate of nerve block and reduce adverse events such as intraneural or intravascular injections.

The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH who have completed the ORION-16 (CKJX839C12301), ORION-13 (CKJX839C12302), ORION-20 (CKJX839C12303) or ORION-19 (CKJX839C12304) studies.

The goal of this randomized controlled trial is to evaluate the efficacy of the Epley maneuver versus a sham maneuver in treating patients presenting to the emergency department (ED) with BPPV. The main questions that the study aims to answer are: * What is the mean difference in Dizziness Handicap Inventory (DHI) scores between patients receiving the Epley maneuver and those receiving the sham maneuver 3 days after enrollment? * What is the immediate response (using a 0-10-point Likert scale) of patients in each arm of the study? * What is the proportion of patients who, despite agreeing to participate, were unable to complete the full Epley maneuver due to symptom severity? Researchers will compare the Epley maneuver arm to the sham maneuver arm to see if the Epley maneuver is more effective in reducing BPPV symptoms. Participants will: * Undergo either the Epley maneuver or a sham maneuver. * Complete the Dizziness Handicap Inventory (DHI) questionnaire at baseline and 3 days post-discharge. * Patients with insufficient improvement in the sham maneuver group will receive a rescue Epley maneuver. * Have their vertigo severity assessed using a 0-10 point Likert scale at baseline, immediately and post-maneuver.

This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat. Eligible participants who enter the study will start a 96-week treatment period. At the end of the 96 weeks, participants will have an end of study visit that occurs 4 weeks later. The participants will receive etavopivat every day throughout the treatment period.

Advancements in systemic antineoplastic therapies have led to improved overall survival rates for many solid tumors. However, metastatic disease remains a significant challenge and remains the leading cause of mortality for these patients. Additionally, there is a high attrition rate after first-line standard treatment across various tumor types, with studies indicating that 20-70% of patients may be unable to undergo second-line therapy, depending on the tumor type. This highlights an urgent need to enhance outcomes from the first line of treatment. Although first-line therapy often represents the best available option, most patients experience relapse and disease progression despite an initial tumor response. This is attributed to both intrinsic and acquired resistance arising from the heterogeneity of primary tumors and metastases. To address this issue, metastasis-directed therapy (MDT) has been explored as a way to reduce tumor burden and mitigate the risk of resistance due to therapeutic selective pressure. MDT offers a promising opportunity to improve first-line treatment outcomes, but more precise patient selection criteria are needed to maximize therapeutic benefit and minimize the potential toxicity of ablative therapies. Indeed, despites its efficacy, fatal complication may occur so do grade 3 to 4 toxicities. Toxicity depends of the local ablative therapy (LAT) planned but as it will never be none, oncologists have to propose invasive treatment to patient that may benefit the most. Based on the published data, the investigators propose a pragmatic, selective approach centered on sensitivity to systemic therapy. The investigators aim to evaluate the benefit of local ablative therapy (LAT) in patients who demonstrate non-progressive disease after three months of first-line standard of care. Given the importance of this question across cancer subtypes, the investigators will employ a prospective database to enroll patients with various solid tumor type, excluding the ones for which the impact of LAT has already been explored or may be difficult to achieve. Outcomes of this strategy will be evaluated compared to outcomes from pivotal studies defining optimal standard first line therapy (OST) . Several analyses will be performed to better characterize the population for whom a multimodal approach may significantly improve their survival. The first one will aim to compare the median duration of response (mDOR) of the population treated with LAT compared to the mDOR reported by the pivotal study(ies) of each OST. This study will serve as a proof of concept, supporting chemosensitivity as a viable selection factor for multimodal treatment in a broad range of cancer types. Primary objective: Improvement of the duration of response (DOR) after completion of LAT compared to DOR reported in pivotal study that evaluated first line OST. Secondary objectives: * Evaluation of the safety of the addition of LAT. * Evaluation of overall progression free survival (PFS) and PFS at 1 year. * Evaluation of overall survival from OST start and from the time of LAT completion. * Documentation of acceptance and compliance to LAT decision by the institutional expert committee

The goal of this prospective, randomized, single-blinded is to learn if there is an ideal sedation protocol in cataract surgery in adults. The main questions it aims to answer are: * Does the combination of Dexmedetomidine and Propofol affect significantly Ramsay sedation scale, compared to Dexmedetomidine and compared to Propofol? * How does each sedation protocol affect hemodynamics? (Heart rate and blood pressure) * Are respiratory events more common in a certain group? * Is the surgeon's satisfaction similar among groups? * Are adverse effects (bradycardia, hypotension, nausea) more common in a certain group? Researchers will compare 3 sedation protocols : Dexmedetomidine versus Propofol versus the combination of these 2 drugs and to see if one protocol is overall superior to the others. Fentanyl will also be used in all 3 sedation protocols. Participants will : * Receive one of these three protocols * Be operated for one or both eyes * Monitored during the whole surgery and in the recovery room * Be evaluated by the Ramsay sedation scale by a trained Anesthesiologist or CRNA during surgery and in the recovery room Surgeons will be asked about how much they were satisfied.

Benign prostatic hyperplasia (BPH) is one of the most common performed surgical procedures in urology. Over the past few decades there have been an increasing development of newer surgical treatment options. Additionally, the outcome parameters for BPH treatments have been standardized. While data are available for the initial pivotal studies, post-market release data are lacking. Under the umbrella of uCARE, we have started a prospective, ongoing international registry for recording demographics and outcomes for patients undergoing surgical treatments for BPH.

The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The objectives of the Registry are: * To enhance understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Gaucher medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Gaucher disease population. * To evaluate the long-term effectiveness of imiglucerase and of eliglustat. Gaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.

Maxillary posterior area is the most challenging site for dental implant placement. Indeed, complex remodeling processes, resulting in changes of the maxillary sinus and shape of the alveolar ridge will cause difficulties in rehabilitating the posterior maxillae sites. Various techniques have been proposed in order to achieve the necessary bone volume for the placement of long term successful implants. However, unbalanced crown to implant ratio remains a risk factor after successful dental implants installation following sinus graft. No study on humans assessed the effect of apical positioning of the implants in posterior grafted sites (sinus graft or sinus graft + vertical GBR) on the following clinical parameters: The peri-implant bone remodeling (vertical bone loss (mm) and probing depth (mm) around the implants, crown bulkiness, emergence profile and crown height, and Patients satisfaction, food impaction (PROM)). This study will elucidate these points and based on the parameter's results, suggest a treatment based classification facilitating the treatment decision-making of the clinicians in case of an atrophic posterior maxilla; aiming to ensure a long-term successful supra-implant rehabilitation which will maintain its bio-mechanically and biological success.

This research aims to investigate the influence of alveolar ridge preservation, after extraction of maxillary posterior teeth, on the potential need of sinus floor augmentation procedures (that include lateral or crestal sinus augmentations). From peri-apical radiographs, maxillary posterior teeth will be divided into 4 groups according to the relationship of their apexes with the sinus. Then, the patients will be divided into 2 sub-groups: unassisted socket healing (only extraction) and ARP group (extraction + ARP) and their CBCT scans before implant placement will be collected. The CBCT scans should be at least 4 months post-extraction and ARP. Depending on the residual bone height, patients will be divided into 3 categories, according to the ABC classification: 1. In need of lateral sinus floor augmentation 2. In need of crestal sinus floor augmentation 3. No need for sinus floor augmentation, therefore, implant placement. These divisions will be conducted, according to: \- The residual bone height (RBH) which is the height in mm from the alveolar ridge crest to the lowest point of the maxillary sinus floor. The results of the proposed hypothesis are verified and interpreted after statistical analysis.

This randomized clinical trial aims to compare two behavior guidance techniques used to reduce anxiety in children during local anesthesia injections in dental treatment. The study evaluates the traditional Tell-Show-Do (TSD) technique versus an artificial intelligence (AI)-driven, avatar-based video method called the "Mini Dentist." Children aged 5 to 7 years who require local anesthesia for routine dental treatment will be randomly assigned to one of two groups. The control group will receive the conventional Tell-Show-Do explanation from the pediatric dentist. The experimental group will watch a short animated video featuring an AI-generated avatar that explains and demonstrates the procedure in a child-friendly manner before the injection. Anxiety and behavior will be assessed using heart rate monitoring, behavioral observation (FLACC scale), and a self-reported pain scale (Wong-Baker FACES). The goal is to determine whether the AI-based method improves cooperation and reduces anxiety compared to the traditional approach. Participation is voluntary, and all procedures follow ethical research standards.

This study will explore how intermittent fasting (specifically the 16/8 method) affects the mental health of women who are in the perimenopause stage. This life stage often brings hormonal changes that can impact emotional and physical well-being. The study will include 98 women aged 45-55, who will be randomly divided into two groups. The study will compare the mental health outcomes of those who follow intermittent fasting with those who do not, using independent t-tests to analyze the results. The goal is to better understand if intermittent fasting can help improve mental health during perimenopause.

The goal of this clinical trial is to learn if using sodium hypochlorite/amino acid gel, with or without cross-linked hyaluronic acid, as an addition to standard non-surgical periodontal therapy works to treat periodontitis in adults. It will also learn about the safety of these treatments. The main questions it aims to answer are: Does the adjunctive treatment reduce bleeding on probing, plaque index, and periodontal pocket depth? What medical or dental problems do participants have when receiving these treatments? Researchers will compare: Standard non-surgical periodontal therapy alone Standard therapy plus sodium hypochlorite/amino acid gel Standard therapy plus sodium hypochlorite/amino acid gel and cross-linked hyaluronic acid ("Clean and Seal" protocol) Participants will: Receive their assigned treatment during one clinical session per the study protocol Have clinical measurements taken before treatment and at three months after treatment Be monitored for any dental or medical adverse events related to the procedures