Genetic Diseases, Inborn Clinical Trials

40 clinical trials for Genetic Diseases, Inborn across international registries, including 5 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Long-term Follow-up Study in Participants Who Received CTX001 — RECRUITING · PHASE3
• Health Economic Evaluation of Non-Invasive Prenatal Exclusion Diagnosis — RECRUITING
• Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease — RECRUITING · PHASE3
• A Study to Learn About the Safety and Efficacy of the Drug Oleogel-S10 in Japanese Patients With Epidermolysis Bullosa — RECRUITING · PHASE3
• Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria — RECRUITING · NA
• Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping — OTHER
• Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) — ACTIVE_NOT_RECRUITING · PHASE3
• Prenatal Transplantation for Fetuses With Fanconi Anemia — NOT_YET_RECRUITING · PHASE1
• A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy — ACTIVE_NOT_RECRUITING · PHASE3
• Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy — COMPLETED · PHASE3
• A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia — COMPLETED · PHASE2
• Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients — COMPLETED · PHASE2
• Effect of Benralizumab in Atopic Dermatitis — COMPLETED · PHASE2
• Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia — COMPLETED · PHASE1
• A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy — COMPLETED · PHASE2
• Evaluating Prenatal Exome Sequencing Study — ACTIVE_NOT_RECRUITING
• Pilot Study of Cardiac MR in Patients With Muscular Dystrophy — COMPLETED
• Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome — COMPLETED · PHASE4
• Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors — COMPLETED · PHASE1
• NYCKidSeq: Incorporating Genomics Into Clinical Care of Diverse NYC Children — COMPLETED · NA
• Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants — COMPLETED
• Evaluating Innovations in Transition From Pediatric to Adult Care - The Transition Navigator Trial — COMPLETED · NA
• Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers — COMPLETED · PHASE1
• Genetic Diagnosis and Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature — OTHER
• Study to Investigate Efficacy and Safety of PF-04965842 in Subjects Aged 12 Years and Over With Moderate to Severe Atopic Dermatitis With the Option of Rescue Treatment in Flaring Subjects — COMPLETED · PHASE3
• Utilizing Free DNA in Embryo Culture for PGT — COMPLETED
• Natural History Study of Children With Metachromatic Leukodystrophy — TERMINATED
• Whole Genome Sequencing in the Neonatal Intensive Care Unit — COMPLETED
• Study Evaluating Efficacy and Safety of PF-04965842 and Dupilumab in Adult Subjects With Moderate to Severe Atopic Dermatitis on Background Topical Therapy — COMPLETED · PHASE3
• An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy — TERMINATED · PHASE3
• Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) — COMPLETED · PHASE3
• Study on the Mechanism of Neurodevelopment Dysplasia of Fetal Brain Caused by ASNS Gene Mutation — OTHER
• An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients — COMPLETED
• Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration — COMPLETED · PHASE2
• An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia — COMPLETED · PHASE3
• Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia — COMPLETED · PHASE2
• Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia — COMPLETED · PHASE3
• Uterine Irrigation Method in Infertility Patients Who Require PGD — OTHER · NA
• Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) — COMPLETED · PHASE3
• High-Density Lipoprotein (HDL) Treatment Study — COMPLETED · NA

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