Not applicableStudy completedNCT00004306What this trial is testingClinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)Who this might be right forHereditary Ataxia Office of Rare Diseases (ORD) 18
Not applicableNot Yet RecruitingNCT07432035What this trial is testingWalking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort StudyWho this might be right forCharcot Marie Tooth Disease (CMT)Pes CavovarusSurgery+3 more Peking University Third Hospital 200
Testing effectiveness (Phase 2)Enrolling By InvitationNCT06253507What this trial is testingPCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)Who this might be right forp47Autosomal RecessiveChronic Granulomatous Disease National Institute of Allergy and Infectious Diseases (NIAID) 10
Not applicableLooking for participantsNCT05129605What this trial is testingProstate Cancer Genetic Risk Evaluation and Screening StudyWho this might be right forProstatic NeoplasmProstate CancerBRCA2 Mutation+5 more Massachusetts General Hospital 400
Not applicableStudy completedNCT00849823What this trial is testingHIV Prevention Program for African American Teen MalesWho this might be right forHIV InfectionsSexually Transmitted Infections Richard Crosby 840
Not applicableStudy completedNCT03982446What this trial is testingGermline Mutations in Pancreatic AdenocarcinomaWho this might be right forGermline Mutation AbnormalityPancreatic CancerPredisposition, Genetic+1 more Hellenic Cooperative Oncology Group 549
Early research (Phase 1)Study completedNCT00004294What this trial is testingPhase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher DiseaseWho this might be right forGaucher's Disease National Center for Research Resources (NCRR) 15
Not applicableStudy completedNCT02826694What this trial is testingNorth Carolina Newborn Exome Sequencing for Universal ScreeningWho this might be right forMetabolism, Inborn ErrorsHearing LossHereditary Disease University of North Carolina, Chapel Hill 106
Large-scale testing (Phase 3)Study completedNCT02544750What this trial is testingAn Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)Who this might be right forTuberous Sclerosis ComplexSeizures Jazz Pharmaceuticals 199
Not applicableStudy completedNCT00446862What this trial is testingThe Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) StudyWho this might be right forFabry DiseaseProteinuria University of Alabama at Birmingham 36
Not applicableStudy completedNCT04226586What this trial is testingNutritional Stimulation of Growth in Children With Short StatureWho this might be right forNutritional StuntingGrowth Failure Arkansas Children's Hospital Research Institute 24
Testing effectiveness (Phase 2)Study completedNCT02428296What this trial is testingStudy of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K ActivationWho this might be right forPIK3CA-Related Overgrowth Spectrum (PROS)Growth DisorderGenetics National Human Genome Research Institute (NHGRI) 39
Not applicableStudy completedNCT00174954What this trial is testingMagnetic Resonance Imaging in Subjects With Gouty TophiWho this might be right forHyperuricemiaGout Takeda 32
Not applicableLooking for participantsNCT06325488What this trial is testingFibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY DiseaseWho this might be right forFabry DiseaseChronic Kidney Diseases Caroline Michaela Kistorp 60
Not applicableLooking for participantsNCT07461896What this trial is testingStudying Nerve Function and Structure in Charcot-Marie-Tooth Disease, Anti-MAG Neuropathy and CIDPWho this might be right forCharcot-Marie-ToothCMT1AChronic Inflammatory Demyelinating Polyneuropathy (CIDP)+2 more Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 39
Not applicableStudy completedNCT01070173What this trial is testingGhrelin Levels in Children With Poor GrowthWho this might be right forIdiopathic Short StatureFailure to Thrive Tripler Army Medical Center 52
Testing effectiveness (Phase 2)Study completedNCT00002673What this trial is testingImmunotherapy in Treating Patients Who Are Undergoing Bone Marrow or Peripheral Stem Cell TransplantationWho this might be right forCancer Fred Hutchinson Cancer Center 30
Not applicableStudy completedNCT00004753What this trial is testingLong-Term Study of Cerebral Glucose Metabolism in Huntington's DiseaseWho this might be right forHuntington's Disease National Center for Research Resources (NCRR) 90
Post-approval studies (Phase 4)Study completedNCT02500641What this trial is testingIntensive Urate Lowering Therapy of Febuxostat Compared to Allopurinol on Cardiovascular Risk in Patients With GoutWho this might be right forGout Menarini International Operations Luxembourg SA 196
Not applicableStudy completedNCT04925622What this trial is testingComplex Eye Movements in Parkinson's Disease and Related Movement DisordersWho this might be right forParkinson DiseaseParkinson's Disease and ParkinsonismProgressive Supranuclear Palsy+7 more Saccadous, Inc. 90