Not applicableStudy completedNCT04126005What this trial is testingNatural History Study of Patients With Canavan Disease (CANinform Study)Who this might be right forCanavan Disease Aspa Therapeutics 67
Testing effectiveness (Phase 2)Ended earlyNCT00668564What this trial is testingHematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismWho this might be right forHurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more Masonic Cancer Center, University of Minnesota 18
Testing effectiveness (Phase 2)Study completedNCT00730314What this trial is testingUnrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood CellsWho this might be right forSickle Cell DiseaseThalassemiaAnemia+9 more Children's Hospital Los Angeles 25
Not applicableStudy completedNCT05687474What this trial is testingBaby Detect : Genomic Newborn ScreeningWho this might be right forCongenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more Centre Hospitalier Universitaire de Liege 6,824
Testing effectiveness (Phase 2)Looking for participantsNCT04998396What this trial is testingAAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)Who this might be right forCanavan Disease Aspa Therapeutics 26
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Testing effectiveness (Phase 2)Enrolling By InvitationNCT04833907What this trial is testingRAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan DiseaseWho this might be right forCanavan Disease Myrtelle Inc. 24
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104What this trial is testingMT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisWho this might be right forMucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more Masonic Cancer Center, University of Minnesota 149
Not applicableLooking for participantsNCT01793168What this trial is testingRare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordWho this might be right forRare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more Sanford Health 20,000