Not applicableLooking for participantsNCT04776824What this trial is testingSwiss Cardiac Amyloidosis REgistry (Swiss-CARE)Who this might be right forAmyloid Cardiomyopathy Insel Gruppe AG, University Hospital Bern 300
Testing effectiveness (Phase 2)UnknownNCT02663024What this trial is testingStudy of Idursulfase-beta (GC1111) in Hunter SyndromeWho this might be right forMucopolysaccharidosis II Green Cross Corporation 20
Not applicableLooking for participantsNCT07435168What this trial is testingCHEST-CA: Study of Chest Pain and Hidden Cardiac AmyloidosisWho this might be right forCardiac AmyloidosisChest Pain Rigshospitalet, Denmark 300
Large-scale testing (Phase 3)Ended earlyNCT00545597What this trial is testingA Phase III Trial of Lorenzo's Oil in AdrenomyeloneuropathyWho this might be right forAdrenomyeloneuropathyAdrenoleukodystrophy Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 240
Not applicableUnknownNCT06260982What this trial is testingCognitive Disorders in Hereditary Spastic Paraplegia Type 4Who this might be right forSpastic Paraplegia Central Hospital, Nancy, France 30
Early research (Phase 1)Looking for participantsNCT07354724What this trial is testingEvaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe DiseaseWho this might be right forLate-onset Pompe Disease Denali Therapeutics Inc. 32
Not applicableStudy completedNCT03911089What this trial is testingA Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCDWho this might be right forUrea Cycle DisorderInborn Errors of Metabolism Nutricia Research 3
Not applicableLooking for participantsNCT06573723What this trial is testingInstitutional Registry of Rare DiseasesWho this might be right forRare DiseasesAmyloidosisSarcoidosis+23 more Hospital Italiano de Buenos Aires 380
Not applicableLooking for participantsNCT07766135What this trial is testingLiver and Coagulation Disorders in Cardiac Transthyretin AmyloidosisWho this might be right forTransthyretin (TTR) Amyloid CardiomyopathyCardiac AmyloidosisCardiomyopathies+2 more University of Messina 70
Not applicableStudy completedNCT02354651What this trial is testingResponse to Diaphragmatic Pacing in Subjects With Pompe DiseaseWho this might be right forPompe Disease University of Florida 9
Not applicableNot Yet RecruitingNCT07315126What this trial is testingReliability of Transcutaneous Bilirubin Measurement According to the Skin Colour of NewbornsWho this might be right forNeonatal Jaundice Assistance Publique - Hôpitaux de Paris 510
Not applicableStudy completedNCT04201418What this trial is testingObservational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A MutationWho this might be right forHereditary Transthyretin-mediated (ATTRv) AmyloidosisPolyneuropathy Alnylam Pharmaceuticals 67
Early research (Phase 1)Study completedNCT00692926What this trial is testingUnrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood CellsWho this might be right forMDSAnemia, AplasticInborn Errors of Metabolism+2 more Joanne Kurtzberg, MD 37
Not applicableLooking for participantsNCT06877715What this trial is testingAutistic Symptomatology and Sensory Profile in Children With Prader-Willi SyndromeWho this might be right forPrader-Willi Syndrome University Hospital, Toulouse 75
Not applicableWithdrawnNCT02462213What this trial is testingProspective Identification of Cardiac Amyloidosis by Cardiac Magnetic Resonance ImagingWho this might be right forAmyloidosisParaproteinemiasCardiomyopathies University of Cincinnati
Not applicableActive Not RecruitingNCT03090789What this trial is testingFA Clinical Outcome MeasuresWho this might be right forFriedreich AtaxiaNeuro-Degenerative Disease Friedreich's Ataxia Research Alliance 2,000
Not applicableStudy completedNCT02699190What this trial is testingLeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesWho this might be right forLeukodystrophyWhite Matter Disease4H Syndrome+61 more Children's Hospital of Philadelphia 236
Large-scale testing (Phase 3)Study completedNCT05099640What this trial is testingPTC923 in Participants With PhenylketonuriaWho this might be right forPhenylketonuria PTC Therapeutics 157
Not applicableStudy completedNCT06201832What this trial is testingCardiac Amyloidosis in HFpEF Tunisian PatientsWho this might be right forCardiac AmyloidosisHeart Failure With Preserved Ejection FractionEchocardiography+4 more University Tunis El Manar 87
Testing effectiveness (Phase 2)Looking for participantsNCT07371663What this trial is testingAn Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid TumorsWho this might be right forSolid CancersNSCLC (Advanced Non-small Cell Lung Cancer)Gastric (Stomach) Cancer+2 more Beijing Tide Pharmaceutical Co., Ltd 266