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

Retreatment and Its Efficiency of Thalidomide for Vascular Malformation Patients With Failure of First Course Treatment

NCT02301949 ·Status: WITHDRAWN ·Phase: PHASE2

A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy

NCT03570931 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2/PHASE3

Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome

NCT03790865 ·Status: TERMINATED ·Phase: PHASE2/PHASE3

Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in Adult Patients With Untreated LCH

NCT07187193 ·Status: RECRUITING ·Phase: PHASE2

A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)

NCT05543174 ·Status: COMPLETED ·Phase: PHASE3

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

NCT03692312 ·Status: COMPLETED ·Phase: PHASE2/PHASE3

Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)

NCT01525901 ·Status: COMPLETED ·Phase: PHASE2

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

NCT00053937 ·Status: COMPLETED ·Phase: PHASE1

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

NCT02858908 ·Status: COMPLETED ·Phase: PHASE2

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

NCT05025241 ·Status: COMPLETED ·Phase: PHASE2

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

NCT04977986 ·Status: COMPLETED ·Phase: PHASE3

A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

NCT00515398 ·Status: COMPLETED

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

NCT05004129 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

NCT00304512 ·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

A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

NCT05139680 ·Status: COMPLETED

An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

NCT07222371 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

NCT07281079 ·Status: RECRUITING ·Phase: PHASE3

Safety and Durability of Sirolimus for Treatment of LAM

NCT02432560 ·Status: RECRUITING

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

NCT03425539 ·Status: COMPLETED ·Phase: PHASE3

Effect of Rimonabant on Weight Gain and Body Composition in Adults With Prader Willi Syndrome

NCT00603109 ·Status: TERMINATED ·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

An Open-Label Study to Assess the Safety & Efficacy of Leniolisib in Japanese Patients With APDS

NCT06249997 ·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 (NPC)

NCT07082725 ·Status: RECRUITING ·Phase: PHASE3

A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age

NCT02484703 ·Status: TERMINATED ·Phase: PHASE2