Genetic Diseases Clinical Trials

38 clinical trials for Genetic Diseases across international registries, including 17 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• ECUR-506 in Neonatal OTC Deficiency Phase I/II — RECRUITING · PHASE2
• Observational long-term follow- up study for patients previously treated with ex vivo gene therapy — RECRUITING
• A Phase IIIb/IV, randomized, double-blind, parallel-group, placebo-controlled, trial to evaluate the efficacy and safety of daily subcutaneous injections of elamipretide in patients with genetically confirmed Barth syndrome — RECRUITING
• A 2-part, randomized, double-blind, placebo-controlled study in participants with Duchenne muscular dystrophy amenable to exon 44 skipping to evaluate the safety and efficacy of ENTR-601-44 (ELEVATE-44) — RECRUITING · PHASE1_PHASE2
• A clinical trial testing a new treatment called mRNA-4194 for people with Lynch syndrome — RECRUITING · PHASE2
• Study to test the safety and effectiveness of sutacimig for people with a rare bleeding condition called congenital factor VII deficiency — RECRUITING · PHASE2
• The Generation Study — RECRUITING
• Study of EN-374 gene therapy in participants with X-linked chronic granulomatous disease — RECRUITING · PHASE1_PHASE2
• Exploring the hidden burden of living with Glanzmann thrombasthenia — RECRUITING
• Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics — RECRUITING
• A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping — RECRUITING · PHASE3
• Genetic and Metabolic Disease in Children — RECRUITING
• PKP2-ACM Natural History Study — RECRUITING
• Genotype-phenotype Characterization Study on Genetic Diseases With Immune and Neurological Dysfunctions — RECRUITING
• Impaired Type I IFN Immunity Due to Autoantibodies or a Genetic Defect: a Prospective National Cohort — RECRUITING
• Rady Children's Institute Genomic Biorepository — RECRUITING · NA
• Rapid Whole Genome Sequencing Study — RECRUITING · NA
• A phase 3 study to evaluate the efficacy and safety of ARO-APOC3 in adults with familial chylomicronemia syndrome — ACTIVE_NOT_RECRUITING · PHASE3
• The effect of parental group sleep education in young children with Down syndrome: the REST-Ed study — TERMINATED
• The Scandcleft project — ACTIVE_NOT_RECRUITING
• Gene therapy in children with mucopolysaccharidosis II (MPSII) consented below the age of 22 months — ACTIVE_NOT_RECRUITING · PHASE1_PHASE2
• Effectiveness and safety of burosumab in an Early Access Program: a study of UK adults with X-linked hypophosphataemia — ACTIVE_NOT_RECRUITING
• Impact of Symprove in Ehlers-Danlos Syndrome — ACTIVE_NOT_RECRUITING
• Testing new medicine for adults with Cystic Fibrosis who do not have access to the currently available CFTR-modulating drugs due to genetic mutations — ACTIVE_NOT_RECRUITING · PHASE2
• A Phase II, open-label long-term extension study in participants with Duchenne muscular dystrophy amenable to exon skipping to assess the long-term safety, tolerability, pharmacokinetics, and efficacy of endosomal escape vehicle phosphorodiamidate morpholino oligomer platform products (ELEVATE-LTE) — NOT_YET_RECRUITING · PHASE2
• The Mayo Clinic Rare and Undiagnosed Disease Hackathon — NOT_YET_RECRUITING
• BAG3-DCM Natural History Study — NOT_YET_RECRUITING
• The Alfred Step Test Exercise Protocol (A-STEP), for Adults With Cystic Fibrosis. — COMPLETED · NA
• Perinatal Precision Medicine — OTHER · NA
• Decoding Developmental Disorders in Humams — COMPLETED
• The Pilot Study of High-throughput Sequencing in Neonatal Birth Defects — COMPLETED
• Safety, Tolerability, Pharmacokinetics of ELX-02 in Healthy Adult Volunteers — TERMINATED · PHASE1
• Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome — COMPLETED · PHASE4
• Prenatal Microarray Follow-Up Study — COMPLETED
• Mutation Exploration in Non-acquired, Genetic Disorders and Its Impact on Health Economy and Life Quality — COMPLETED
• Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells — TERMINATED · PHASE2
• Time-Lapse Incubation for Embryo Culture - Morphokinetics and Environmental Stability — OTHER · NA
• Prenatal Cytogenetic Diagnosis by Array-Based Copy Number Analysis — COMPLETED

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