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
Testing effectiveness (Phase 2)Study completedNCT01495260What this trial is testingA Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of AntioxidantsWho this might be right forAdrenomyeloneuropathy Pujol, Aurora, M.D. 13
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
Testing effectiveness (Phase 2)Ended earlyNCT00004418What this trial is testingEffect of Glycerol Trierucate on Clinical Course of AdrenoleukodystrophyWho this might be right forAdrenoleukodystrophy Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 126
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
Not applicableUnknownNCT06497829What this trial is testingGeranylgeraniol Supplementation in Patients With Mevalonate Kinase DeficiencyWho this might be right forMevalonate Kinase Deficiency University Hospital, Motol 6
Not applicableEnded earlyNCT00260299What this trial is testingDietary Cholesterol and Defects in Cholesterol Synthesis in Mevalonate Kinase DeficiencyWho this might be right forMevalonic AciduriaMevalonate Kinase DeficiencyImmune System Diseases+2 more Oregon Health and Science University 1
Not applicableNot Yet RecruitingNCT05911919What this trial is testingValidation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Assistance Publique - Hôpitaux de Paris 200
Not applicableLooking for participantsNCT06190626What this trial is testingLongitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum DisorderWho this might be right forZellweger Spectrum Disorder McGill University Health Centre/Research Institute of the McGill University Health Centre 30
Not applicableLooking for participantsNCT04569162What this trial is testingRhizomelic Chondrodysplasia Punctata RegistryWho this might be right forRCDP - Rhizomelic Chondrodysplasia PunctataRCDP1RCDP2+3 more Nemours Children's Clinic 100
Testing effectiveness (Phase 2)Ended earlyNCT01372228What this trial is testingPhase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersWho this might be right forHurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more Talaris Therapeutics Inc. 3
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 applicableStudy completedNCT01594853What this trial is testingExercise Study of Function and Pathology for Women With X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 31
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 participantsNCT05443906What this trial is testingHome Exercise for Individuals With Neurodegenerative DiseaseWho this might be right forNeurodegenerative DiseasesLeukodystrophyAtaxia+5 more Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 30
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
Not applicableLooking for participantsNCT06178120What this trial is testingDisease Progression in Women With X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Minoryx Therapeutics, S.L. 40
Large-scale testing (Phase 3)Study completedNCT02334748What this trial is testingCanakinumab in Patients With Systemic Juvenile Idiopathic Arthritis or Hereditary Periodic Fevers Who Participated in the CACZ885G2301E1, CACZ885G2306 or CACZ885N2301 StudiesWho this might be right forSystemic Juvenile Idiopathic ArthritisHereditary Periodic Fevers Novartis Pharmaceuticals 31
Testing effectiveness (Phase 2)UnknownNCT02559830What this trial is testingAutologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and AdrenoleukodystrophyWho this might be right forMetachromatic LeukodystrophyAdrenoleukodystrophy Shenzhen Second People's Hospital 50
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