A Study on the Safety of Ozanimod Exposure in Pregnant Women and Their Offspring

Short-Term Oral Mifepristone for Central Serous Chorioretinopathy

NCT02354170 ·Status: COMPLETED ·Phase: PHASE2

Pregnancy Exposure Registry for Vumerity (Diroximel Fumarate)

NCT05658497 ·Status: RECRUITING

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

NCT04252066 ·Status: RECRUITING

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

NCT05758922 ·Status: COMPLETED ·Phase: PHASE2

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome

NCT04977986 ·Status: COMPLETED ·Phase: PHASE3

An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome

NCT05066217 ·Status: RECRUITING ·Phase: PHASE3

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07082543 ·Status: RECRUITING ·Phase: PHASE3

Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU

NCT05356377 ·Status: COMPLETED

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT06567769 ·Status: RECRUITING ·Phase: PHASE1

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

NCT07082725 ·Status: RECRUITING ·Phase: PHASE3

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515 ·Status: RECRUITING ·Phase: PHASE3

A Study to Evaluate the Safety and Efficacy of Basmisanil Treatment in Children Aged 2-14 Years With Dup15q Syndrome

NCT05307679 ·Status: TERMINATED ·Phase: PHASE2

A Pharmacokinetic, Pharmacodynamic, Safety and Tolerability Study of CERC-006 in Adults With Complex Lymphatic Malformations

NCT04994002 ·Status: WITHDRAWN ·Phase: PHASE1

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)

NCT05025241 ·Status: COMPLETED ·Phase: PHASE2

Cerebrospinal Fluid Pharmacokinetics of Daptomycin

NCT01025271 ·Status: TERMINATED ·Phase: NA

Safety of 25 and 50 mg Proellex® in the Treatment of Women With Symptomatic Uterine Fibroids

NCT01069120 ·Status: TERMINATED ·Phase: PHASE3

Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas

NCT00053937 ·Status: COMPLETED ·Phase: PHASE1

Evaluating the Safety and Efficacy of Proellex® (CDB-4124) in Premenopausal Women With Symptomatic Uterine Fibroids

NCT00735553 ·Status: TERMINATED ·Phase: PHASE3

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

NCT01253629 ·Status: COMPLETED ·Phase: PHASE2

Natural History Studies of Mucopolysaccharidosis III

NCT02037880 ·Status: COMPLETED

A Phase II, Parallel Group, Randomized, Placebo-Controlled Study of the Safety and Efficacy of Thalidomide in Reducing Weight Loss in Adults With HIV Wasting Syndrome

NCT00002127 ·Status: COMPLETED ·Phase: PHASE2

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

NCT03802799 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE2/PHASE3

Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)

NCT07185256 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy

NCT02176733 ·Status: UNKNOWN ·Phase: PHASE2