heart-defect Clinical Trials
A listing of heart-defect medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 118 clinical trials
A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives. Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching. The main aim of this study is to observe the long-term, …
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)
The key objectives of this low-intervention clinical study are to evaluate tolerability, long-term safety and long-term efficacy for patients with Alagille syndrome (ALGS) who are prescribed Livmarli.
Clinical Trial in Patients With Barth Syndrome- 4TAZPower
Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily SC injection of elamipretide in subjects with genetically confirmed BTHS for 72 weeks. The primary trial objective is to confirm the efficacy of elamipretide which is approved in the United States(FORZINITY™) …
A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)
This study is a database study in Japan for maralixibat (TAK-625) used to treat participants with Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). The main aim of the study is to evaluate the risk of liver disorder during the use of maralixibat in Japanese patients with ALGS or …
HeartR™ PDA Occluder Post-Market Follow-Up Study
The HeartR™ PDA Occluder Post-Market Clinical Follow-up (PMCF) Study is a multi-center, retrospective, single-arm study sponsored by Lifetech Scientific (Shenzhen) Co., Ltd., aiming to evaluate the long-term safety and performance of the Lifetech HeartR™ PDA Occluder in real-world clinical practice in Indonesia. A total of 140 subjects diagnosed with Patent …
Precision Dosing of Oral Ibuprofen for PDA, A Pilot RCT
Newborns born early are at risk for a serious health problem called patent ductus arteriosus (PDA). PDA is a passageway between heart and lung that can cause life-threatening complications such as bleeding in the brain or even death if it remains open and large. When closure of PDA is needed, …
Pediatric Evaluation and Registry for Liver Cholestasis in Canada
The purpose of this study is to create a national, multi-centre registry for children with Alagille syndrome (ALGS) and Genetic Intrahepatic Cholestasis (GIC) that follows participants long-term, ensuring standardized, high-quality data capture across all participating pediatric hepatology centres. Inclusion criteria: • Pediatric participants (\<18 years old) with genetically confirmed or …
Executive Function Intervention for Children With Complex Congenital Heart Disease
Children with complex congenital heart disease (cCHD) nowadays survive thanks to medical advances into their teenage years and adulthood. However, many of them have difficulties with so-called executive functions. These include skills such as paying attention, planning, self-control, and flexible thinking, which are very important for school, daily life, and …
Constitution of a Biological Collection to Study the Pathophysiology in Noonan Syndrome
The present study will establish a collection of biological samples from Noonan patients to be used for research purposes only, with due respect for confidentiality.
National Multicentre Study on Lipid Profile in Noonan Syndrome and Related Disorders: Trends by Age, Gender and Genotype
RASopathies, including Noonan syndrome, involve dysmorphisms, metabolic alterations, and an unfavorable lipid profile. This study investigates lipid and glucose metabolism to improve patient care.
