Testing effectiveness (Phase 2)Study completedNCT01303380What this trial is testingCanakinumab in Patients With Active Hyper-IgD SyndromeWho this might be right forMevalonate Kinase Deficiency Novartis Pharmaceuticals 9
Not applicableLooking for participantsNCT02435940What this trial is testingInherited Retinal Degenerative Disease RegistryWho this might be right forEye Diseases HereditaryRetinal DiseaseAchromatopsia+25 more Foundation Fighting Blindness 20,000
Not applicableStudy completedNCT01626092What this trial is testingReduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersWho this might be right forLysosomal Storage DiseasePeroxisomal Disorder Masonic Cancer Center, University of Minnesota 3
Not applicableUnknownNCT03278899What this trial is testingProspectively Assess Disease Progression in Male Children With X-ALDWho this might be right forX-Linked Adrenoleukodystrophy NeuroVia, Inc. 100
Not applicableStudy completedNCT03440905What this trial is testingProxy-Reported Symptoms and Quality of Life Survey in Zellweger Spectrum DisordersWho this might be right forZellweger Spectrum University of South Florida 92
Large-scale testing (Phase 3)Study completedNCT01838941What this trial is testingBetaine and Peroxisome Biogenesis DisordersWho this might be right forPeroxisome Biogenesis Disorders McGill University Health Centre/Research Institute of the McGill University Health Centre 12
Not applicableActive Not RecruitingNCT02698579What this trial is testingLong-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug ProductWho this might be right forCerebral Adrenoleukodystrophy (CALD)Adrenoleukodystrophy (ALD)X-Linked Adrenoleukodystrophy (X-ALD) Genetix Biotherapeutics Inc. 64
Not applicableUnknownNCT00005900What this trial is testingStudy of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationWho this might be right forI Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more Fairview University Medical Center 10
Not applicableNo Longer AvailableNCT02233257What this trial is testingExpanded Access for Lorenzo's Oil (GTO/GTE) in AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy University of Minnesota
Not applicableWithdrawnNCT03649919What this trial is testingMulti-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in ChildrenWho this might be right forXlsmaDMDX-ALD+1 more Children's Hospital of Fudan University
Not applicableLooking for participantsNCT04675749What this trial is testingQuality of Life in Women with X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Leipzig University Medical Center 200
Not applicableLooking for participantsNCT06224413What this trial is testingParticipants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene AutotemcelWho this might be right forCerebral Adrenoleukodystrophy (CALD) Genetix Biotherapeutics Inc. 120
Not applicableLooking for participantsNCT06796920What this trial is testingEpidural Spinal Cord Stimulation for Lower-limb Impairment in AdrenomyeloneuropathyWho this might be right forAdrenomyeloneuropathy Without Cerebral Involvement Third Military Medical University 10
Not applicableEnded earlyNCT00004442What this trial is testingStudy of Bile Acids in Patients With Peroxisomal DisordersWho this might be right forInfantile Refsum's DiseaseZellweger SyndromeBifunctional Enzyme Deficiency+1 more University of Cincinnati 25
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
Not applicableEnded earlyNCT02204904What this trial is testingObservational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)Who this might be right forX-Linked Adrenoleukodystrophy (X-ALD)Cerebral Adrenoleukodystrophy (CALD)Adrenoleukodystrophy (ALD) Genetix Biotherapeutics Inc. 59
Early research (Phase 1)Study completedNCT02595489What this trial is testingA Pilot Study of Vitamin D in Boys With X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Stanford University 21
Large-scale testing (Phase 3)Study completedNCT00007020What this trial is testingCompassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidWho this might be right forInfantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more Mirum Pharmaceuticals, Inc. 85
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 applicableWithdrawnNCT02948062What this trial is testingEarly Diagnosis Of Childhood Cerebral ALDWho this might be right forAdrenoleukodystrophy Masonic Cancer Center, University of Minnesota