Not applicableStudy completedNCT02952482What this trial is testingNewborn Screening for AdrenoleukodystrophyWho this might be right forAdrenoleukodystrophy National Taiwan University Hospital 45,796
Testing effectiveness (Phase 2)Study completedNCT01043640What this trial is testingAllogeneic Bone Marrow Transplant for Inherited Metabolic DisordersWho this might be right forMucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more Masonic Cancer Center, University of Minnesota 46
Not applicableStudy completedNCT01165060What this trial is testingThe Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)Who this might be right forX-linked AdrenoleukodystrophyAdrenomyeloneuropathy Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) 10
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
Post-approval studies (Phase 4)Active Not RecruitingNCT05003648What this trial is testingTreating Leg Symptoms in Women With X-linked AdrenoleukodystrophyWho this might be right forAdrenoleukodystrophyRestless Legs Syndrome Massachusetts General Hospital 24
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
Early research (Phase 1)WithdrawnNCT01787578What this trial is testingSafety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)Who this might be right forX-Linked AdrenoleukodystrophyAdrenomyeloneuropathy Thomas S. Scanlan
Not applicableUnknownNCT04090268What this trial is testingPrecision Exercise in Children With Malignant HemopathiesWho this might be right forLeukemia, Myeloid, AcuteLeukemia, B-cellLeukemia, T Cell+5 more University of Milano Bicocca 380
Not applicableWithdrawnNCT02948062What this trial is testingEarly Diagnosis Of Childhood Cerebral ALDWho this might be right forAdrenoleukodystrophy Masonic Cancer Center, University of Minnesota
Not applicableLooking for participantsNCT03789721What this trial is testingAdrenoleukodystrophy National Registry StudyWho this might be right forALD (Adrenoleukodystrophy)AdrenoleukodystrophyCerebral Adrenoleukodystrophy Masonic Cancer Center, University of Minnesota 1,000
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 applicableLooking for participantsNCT04528355What this trial is testingData Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICWho this might be right forPrimary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more Paul Szabolcs 50
Large-scale testing (Phase 3)Ended earlyNCT00654433What this trial is testingALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesWho this might be right forInherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more Aldagen 40
Testing effectiveness (Phase 2)Looking for participantsNCT01962415What this trial is testingReduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTWho this might be right forPrimary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more Paul Szabolcs 100
Testing effectiveness (Phase 2)WithdrawnNCT05200104What this trial is testingStudy to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)Who this might be right forALD (Adrenoleukodystrophy) Poxel SA
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
Early research (Phase 1)Study completedNCT04973657What this trial is testingAssess the Pharmacodynamics of VK0214 in Male Subjects With AMNWho this might be right forAdrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD) Viking Therapeutics, Inc. 24
Testing effectiveness (Phase 2)Study completedNCT00383448What this trial is testingHSCT for High Risk Inherited Inborn ErrorsWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more Masonic Cancer Center, University of Minnesota 38
Early research (Phase 1)WithdrawnNCT02410239What this trial is testingMT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)Who this might be right forCerebral Adrenoleukodystrophy Masonic Cancer Center, University of Minnesota
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