Genetic Phenomena

34 trials 15 recruiting

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A study to find how well VES001 works and how safe it is in participants with GRN -frontotemporal dementia (GRN-FTD) showing no symptoms Ended

2024-513258-31-00 Frontotemporal dementia (FTD) 3 2024-12-11
A Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Infigratinib in Children with Achondroplasia Ended

2023-506130-67-00 Achondroplasia 5 2024-08-20
An open label, balanced, randomized, single dose, two treatment, two period, two sequence, crossover, bioequivalence study comparing Ivacaft… Ended

2023-508115-23-00 Cystic Fibrosis 2 2024-02-20
A Phase 2 Study to Evaluate Efficacy, Safety, and Pharmacokinetics of PBF-999 in the Treatment of Patients with Prader-Willi Syndrome. Ended

2022-501462-22-00 Prader-Willi Syndrome 4 2023-02-22
A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with Familial Chylomicronemia Syndrome Ended

2024-514336-24-00 Familial Chylomicronemia Syndrome (FCS) 5 2022-03-24
This is a randomized, double-blinded, Placebo controlled trial that will evaluate the potential of tildacerfont to reduce glucocorticoid use… Ended

2023-503771-13-00 Classic Congenital Adrenal Hyperplasia 4 2021-01-28
This is a randomized, double-blinded, Placebo controlled trial with a 3-part treatment period that will evaluate the efficacy and safety of… Ended

2023-503770-21-00 Classic Congenital Adrenal Hyperplasia 4 2021-01-05
ISIS 721744-CS3: An Open-Label Extension Study of ISIS 721744 in Patients with Hereditary Angioedema Ended

2024-517249-15-00 Hereditary Angioedema 4 2020-10-22
Gene therapy study using a frozen formulation of OTL-200 in patients with Metachromatic Leukodystrophy (MLD) Ended

2024-511970-66-00 Metachromatic Leukodystrophy (MLD) 5 2017-12-13
Hematopoietic stem cell gene therapy study Ended

2024-515253-25-00 Metachromatic Leukodystrophy (MLD) 9 2010-04-08