Adrenoleukodystrophy clinical trials
The registry lists 61 studies for Adrenoleukodystrophy across more than 10 countries. 17 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-09-16.
Status
- Completed
- 23
- Recruiting
- 14
- Terminated
- 8
- Withdrawn
- 6
- Status unknown
- 4
- Active, not recruiting
- 2
Phase
- Phase 2
- 20
- Phase 1
- 11
- Phase 3
- 8
- Phase 4
- 1
Where
- United States
- 35
- Germany
- 11
- France
- 10
- Netherlands
- 8
- United Kingdom
- 7
- Italy
- 6
Who runs them
Masonic Cancer Center, University of Minnesota (7) · Pujol, Aurora, M.D. (3) · Minoryx Therapeutics, S.L. (3) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (3) · bluebird bio (3) · Genetix Biotherapeutics Inc. (2) · NeuroVia, Inc. (2) · Assistance Publique - Hôpitaux de Paris (2)
Studies
Recruiting first, then most recently updated. Showing 50 of 61 — search for more.
- Home Exercise for Individuals With Neurodegenerative DiseaseRecruitingNCT05443906Not applicable1 country
- UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsRecruitingNCT02254863Phase 11 country
- IT and IV Lentiviral Gene Therapy for X-ALDNot yet recruitingNCT03727555Not applicable1 country
- Quality of Life in Women With X-linked AdrenoleukodystrophyRecruitingNCT046757491 country
- Adrenoleukodystrophy National Registry StudyRecruitingNCT037897211 country
- Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICRecruitingNCT045283551 country
- Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 21 country
- Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood TransplantsAvailableNCT018813341 country
- The Myelin Disorders Biorepository ProjectRecruitingNCT030473691 country
- Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented GliaRecruitingNCT049253491 country
- A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene AutotemcelRecruitingNCT062244131 country
- Epidural Spinal Cord Stimulation for Lower-limb Impairment in AdrenomyeloneuropathyRecruitingNCT06796920Not applicable1 country
- Disease Progression in Women With X-linked AdrenoleukodystrophyRecruitingNCT061781201 country
- Precision Exercise in Children With Malignant HemopathiesRecruitingNCT04090268Not applicable1 country
- Registry of X-linked AdrenoleukodystrophyRecruitingNCT059392321 country
- Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked AdrenoleukodystrophyNot yet recruitingNCT05911919
- Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and AdrenoleukodystrophyRecruitingNCT02559830Phase 1/21 country
- Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug ProductActive, not recruitingNCT026985799 countries
- A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)TerminatedNCT05394064Phase 1/22 countries
- Treating Leg Symptoms in Women With X-linked AdrenoleukodystrophyActive, not recruitingNCT05003648Phase 42 countries
- LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesCompletedNCT026991901 country
- Study of Disease Progression in Adults With Inherited Forms of Spastic ParaplegiaTerminatedNCT050088743 countries
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.CompletedNCT03231878Phase 2/38 countries
- A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMNCompletedNCT04973657Phase 15 countries
- Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)WithdrawnNCT05200104Phase 2
- Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)WithdrawnNCT05146284Phase 2
- Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant DiseasesTerminatedNCT03367546Phase 21 country
- A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)CompletedNCT03852498Phase 36 countries
- SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)CompletedNCT04687007Not applicable1 country
- Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidCompletedNCT00007020Phase 31 country
- Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationCompletedNCT03513328Phase 1/21 country
- A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)Status unknownNCT04528706Phase 24 countries
- Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersTerminatedNCT01372228Phase 1/21 country
- Human Placental-Derived Stem Cell TransplantationCompletedNCT01586455Phase 11 country
- Plasma Exchange With Albumin in AMN PatientsCompletedNCT04303416Phase 2/31 country
- A Pilot Study of Vitamin D in Boys With X-linked AdrenoleukodystrophyCompletedNCT02595489Phase 11 country
- A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)CompletedNCT01896102Phase 2/36 countries
- Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and AdrenomyeloneuropathyCompletedNCT03627416Not applicable1 country
- Effect of Glycerol Trierucate on Clinical Course of AdrenoleukodystrophyTerminatedNCT00004418Phase 21 country
- Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)TerminatedNCT022049047 countries
- Effect of Pioglitazone Administered to Patients With AdrenomyeloneuropathyCompletedNCT03864523Phase 21 country
- HSCT for High Risk Inherited Inborn ErrorsCompletedNCT00383448Phase 21 country
- A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of AntioxidantsCompletedNCT01495260Phase 21 country
- Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With AdrenoleukodystrophyWithdrawnNCT03196765Phase 1/28 countries
- Exercise Study of Function and Pathology for Women With X-linked AdrenoleukodystrophyCompletedNCT01594853Not applicable1 country
- A Study to Prospectively Assess Disease Progression in Male Children With X-ALDStatus unknownNCT032788991 country
- Newborn Screening for AdrenoleukodystrophyCompletedNCT029524821 country
- Early Diagnosis Of Childhood Cerebral ALDWithdrawnNCT029480621 country
- Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersCompletedNCT01043640Phase 21 country