NCT05425745
Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies. treatment 3 completed NCT00001405
Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes No drug interventions Not Available Not Available recruiting NCT02916888
A Study Comparing the Quality of Life of Patients in the Treatment of Eczema by Pediatric Generalists and Specialists No drug interventions Not Available Not Available completed NCT06337864
Evaluating the Efficacy and Safety of Large Neutral Amino Acids in the Treatment of Classical Phenylketonuria No drug interventions treatment Not Available not_yet_recruiting NCT02921321
Pilot Study of Cardiac MR in Patients With Muscular Dystrophy No drug interventions Not Available Not Available active_not_recruiting NCT01826487
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) treatment 3 completed NCT03738098
NYCKidSeq: Incorporating Genomics Into Clinical Care of Diverse NYC Children No drug interventions diagnostic Not Available completed NCT03734588
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors No drug interventions treatment 1 / 2 completed NCT00694109
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 treatment 3 completed NCT05730842
Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers treatment 1 completed NCT02884063
Utilizing Free DNA in Embryo Culture for PGT No drug interventions Not Available Not Available completed NCT00607373
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia treatment 3 completed NCT00477594
Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia treatment 2 completed NCT04208529
A Long-term Follow-up Study in Participants Who Received CTX001 other 3 enrolling_by_invitation NCT03627767
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 treatment 3 completed NCT03876301
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants No drug interventions Not Available Not Available completed NCT05479981
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients treatment 2 active_not_recruiting NCT00458055
High-Density Lipoprotein (HDL) Treatment Study treatment Not Available completed NCT03587155
Study on the Mechanism of Neurodevelopment Dysplasia of Fetal Brain Caused by ASNS Gene Mutation No drug interventions Not Available Not Available unknown_status NCT01963650
Natural History Study of Children With Metachromatic Leukodystrophy No drug interventions Not Available Not Available terminated NCT02676479
Uterine Irrigation Method in Infertility Patients Who Require PGD No drug interventions diagnostic Not Available unknown_status NCT02429947
An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients No drug interventions Not Available Not Available completed NCT03655678
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia treatment 2 / 3 active_not_recruiting NCT02090959
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy No drug interventions treatment 3 terminated NCT03720470
Study Evaluating Efficacy and Safety of PF-04965842 and Dupilumab in Adult Subjects With Moderate to Severe Atopic Dermatitis on Background Topical Therapy No drug interventions treatment 3 completed NCT05070234
Genetic Diagnosis and Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature No drug interventions Not Available Not Available unknown_status NCT03718234
Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia No drug interventions treatment 1 completed NCT05126758
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy No drug interventions treatment 3 recruiting NCT03721458
Whole Genome Sequencing in the Neonatal Intensive Care Unit No drug interventions Not Available Not Available completed NCT05477563
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease No drug interventions treatment 3 recruiting NCT03563066
Effect of Benralizumab in Atopic Dermatitis No drug interventions treatment 2 completed NCT03179631
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy No drug interventions treatment 3 completed NCT05657860
Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome No drug interventions treatment 4 completed NCT03406780
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy No drug interventions treatment 2 completed NCT00362180
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration No drug interventions treatment 2 completed NCT05290701
Evaluating Prenatal Exome Sequencing Study No drug interventions Not Available Not Available enrolling_by_invitation NCT00288704
Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) No drug interventions treatment 3 completed NCT03342495
Evaluating Innovations in Transition From Pediatric to Adult Care - The Transition Navigator Trial No drug interventions health_services_research Not Available completed NCT05356195
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) No drug interventions treatment 3 recruiting