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 applicableLooking for participantsNCT04880356What this trial is testingLongitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.Who this might be right forInherited DiseaseRare DiseasesMetabolic Disease+3 more Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
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
Not applicableStudy completedNCT05350982What this trial is testingNeonatal, Infant and Toddler Salivary Study (NITS Study)Who this might be right forAdrenal Insufficiency Sheffield Children's NHS Foundation Trust 30
Not applicableLooking for participantsNCT05939232What this trial is testingRegistry of X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Beijing Tiantan Hospital 200
Large-scale testing (Phase 3)Study completedNCT03852498What this trial is testingA Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)Who this might be right forCerebral Adrenoleukodystrophy (CALD) Genetix Biotherapeutics Inc. 35
Not applicableLooking for participantsNCT04885179What this trial is testingSPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)Who this might be right forSphingolipidosesEnzyme Deficiency University of California, San Francisco 120
Large-scale testing (Phase 3)Study completedNCT02961803What this trial is testingMD1003-AMN MD1003 in AdrenomyeloneuropathyWho this might be right forAdrenomyeloneuropathyAdrenoleukodystrophyAMN MedDay Pharmaceuticals SA 67
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
Large-scale testing (Phase 3)Study completedNCT00176904What this trial is testingStem Cell Transplant for Inborn Errors of MetabolismWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more Masonic Cancer Center, University of Minnesota 135
Testing effectiveness (Phase 2)WithdrawnNCT03196765What this trial is testingSafety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With AdrenoleukodystrophyWho this might be right forX-Linked Adrenoleukodystrophy NeuroVia, Inc.
Post-approval studies (Phase 4)Looking for participantsNCT05836610What this trial is testingHydrocortisone Therapy Optimization During Hypothermia Treatment in Asphyxiated NeonatesWho this might be right forHypoxic-Ischemic EncephalopathyAsphyxiaHypotension+1 more Semmelweis University 50
Not applicableNot Yet RecruitingNCT04252001What this trial is testingGrowing up With the Young Endocrine Support System (YESS!)Who this might be right forCongenital Adrenal HyperplasiaHypogonadotropic HypogonadismGrowth Hormone Deficiency+6 more dr. Laura C. G. de Graaff-Herder 160
Not applicableUnknownNCT00278044What this trial is testingClinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese ChildrenWho this might be right forAdrenoleukodystrophy Far Eastern Memorial Hospital 20
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
Testing effectiveness (Phase 2)Study completedNCT02096510What this trial is testingUltradian Subcutaneous Hydrocortisone Infusion in Addison Disease and Congenital Adrenal HyperplasiaWho this might be right forAddison DiseaseAdrenal Hyperplasia Congenital Haukeland University Hospital 8
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
Not applicableStudy completedNCT00974337What this trial is testingRelative Adrenal Insufficiency in Preterm Very Low Birth Weight Infants With ShockWho this might be right forAdrenal Insufficiency All India Institute of Medical Sciences 39
Not applicableStudy completedNCT02956707What this trial is testingImpact of Pre-operative Steroids on Adrenal InsufficiencyWho this might be right forAdrenal Insufficiency Neonatal University of Alabama at Birmingham 50
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