Testing effectiveness (Phase 2)UnknownNCT02412709What this trial is testingLong QT Syndrome Screening in NewbornsWho this might be right forLong QT Syndrome QT Medical, Inc. 4,000
Not applicableUnknownNCT03341416What this trial is testingEffects of Deep Brain Stimulation of the Dentate Nucleus on Cerebellar AtaxiaWho this might be right forCerebellar Ataxia University of Sao Paulo General Hospital 10
Not applicableStudy completedNCT03624556What this trial is testingPediatric Exploratory Research Study of EGCG Use and Safety (PERSEUS)Who this might be right forDown SyndromeFragile X Syndrome Parc de Salut Mar 76
Not applicableWithdrawnNCT06227429What this trial is testingA Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 PatientsWho this might be right forHereditary Tyrosinemia, Type I Swedish Orphan Biovitrum
Not applicableActive Not RecruitingNCT07250321What this trial is testingEfficacy of tDCS in Degenerative AtaxiaWho this might be right forAtaxia, CerebellarAtaxia - Genetic Diagnosis - UnknownAtaxia, Spinocerebellar+1 more University of Cagliari 16
Early research (Phase 1)UnknownNCT00004307What this trial is testingStudy of Treatment and Metabolism in Patients With Urea Cycle DisordersWho this might be right forAmino Acid Metabolism, Inborn Errors National Center for Research Resources (NCRR) 66
Not applicableStudy completedNCT02450604What this trial is testingPrevalence of Fabry's Disease in a Population of Patients With Chronic PainWho this might be right forFabry's DiseaseChronic Pain University Hospital, Bordeaux 776
Testing effectiveness (Phase 2)Ended earlyNCT00968864What this trial is testingT-cell Depleted Alternative Donor TransplantationWho this might be right forAcute Lymphoblastic LeukemiaAcute Myeloid LeukemiaChronic Myeloid Leukemia+6 more Wake Forest University Health Sciences 53
Not applicableStudy completedNCT01938014What this trial is testingLysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenWho this might be right forMucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more University of Chicago 19
Not applicableStudy completedNCT05425888What this trial is testingFunctional and Proprioceptive Status in Patients With Hemophilic Ankle ArthropathyWho this might be right forHaemophilia Investigación en Hemofilia y Fisioterapia 32
Testing effectiveness (Phase 2)Study completedNCT00004688What this trial is testingPhase II Study of Carmustine, Streptozocin, and Mercaptopurine for Refractory or Recurrent Brain NeoplasmsWho this might be right forAnaplastic AstrocytomaAnaplastic OligodendrogliomaMixed Gliomas+2 more Emory University 35
Large-scale testing (Phase 3)Study completedNCT00814073What this trial is testingMasitinib in Severe Indolent or Smoldering Systemic MastocytosisWho this might be right forIndolent Systemic Mastocytosis AB Science 135
Large-scale testing (Phase 3)Study completedNCT04966741What this trial is testingSetmelanotide in Pediatric Participants With Rare Genetic Diseases of ObesityWho this might be right forBardet-Biedl SyndromePOMC Deficiency ObesityPCSK1 Deficiency Obesity+1 more Rhythm Pharmaceuticals, Inc. 12
Not applicableStudy completedNCT03911531What this trial is testingWhole Exome Sequencing and Whole Genome Sequencing for Nonimmune Fetal/Neonatal HydropsWho this might be right forNonimmune Fetal HydropsNonimmune Hydrops in NeonateGenetic Disorders Thomas Jefferson University 55
Not applicableNot Yet RecruitingNCT07657702What this trial is testingEffects of Anisodamine on Sublingual Microcirculation and Vascular Waterfall Phenomenon in Patients With Septic ShockWho this might be right forSeptic ShockSepsisCritical Illness First Affiliated Hospital of Wannan Medical College 20
Testing effectiveness (Phase 2)UnknownNCT01906541What this trial is testingGene Therapy for X-CGDWho this might be right forX-linked Chronic Granulomatous Disease Hubert Serve, Prof., MD 5
Not applicableNot Yet RecruitingNCT07326839What this trial is testingOrlistat and Weight Management for Uric Acid Control in Obese Gout: A RCTWho this might be right forGoutObesity XueMei Guo 120
Large-scale testing (Phase 3)Ended earlyNCT04684511What this trial is testingRofecoxib Efficacy and Safety Evaluation Trial in Hemophilic ArthropathyWho this might be right forHemophilic Arthropathy Tremeau Pharmceuticals, Inc. 2
Not applicableStudy completedNCT03782909What this trial is testingEvaluation of a Behavioural Intervention Using App Technology in CFWho this might be right forCystic Fibrosis Sheffield Teaching Hospitals NHS Foundation Trust 5
Testing effectiveness (Phase 2)Looking for participantsNCT06325709What this trial is testingBase Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous DiseaseWho this might be right forChronic Granulomatous Disease (CGD)X-Linked Chronic Granulomatous Disease National Institute of Allergy and Infectious Diseases (NIAID) 15