Adrenoleukodystrophy clinical trials
The registry lists 68 studies for Adrenoleukodystrophy across more than 10 countries. 19 are recruiting or about to start. 48 of them are mainly about Adrenoleukodystrophy (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-09.
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Who runs them
Masonic Cancer Center, University of Minnesota (8) · Minoryx Therapeutics, S.L. (4) · bluebird bio (3) · Pujol, Aurora, M.D. (3) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (3) · Genetix Biotherapeutics Inc. (2) · Children's Hospital of Philadelphia (2) · Joanne Kurtzberg, MD (2)
What is being tested here
Drugs under test in Adrenoleukodystrophy studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Alemtuzumab | 1 | 0 | 3 |
| Melphalan | 1 | 0 | 2 |
| Hydroxyurea | 1 | 0 | 1 |
| Fludarabine | 1 | 0 | 1 |
| Thiotepa | 1 | 0 | 1 |
| Cyclophosphamide | 0 | 1 | 2 |
| Busulfan | 0 | 1 | 2 |
| Cyclosporine | 0 | 0 | 2 |
| Mycophenolic Acid | 0 | 0 | 2 |
| Pramipexole | 0 | 0 | 1 |
Drug classes tested here Alkylating agents 1 open
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 5, phase 3 first
- May 2027A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy · Phase 3 · Minoryx Therapeutics, S.L.
- Nov 2026Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT · Paul Szabolcs
- Dec 2026Disease Progression in Women With X-linked Adrenoleukodystrophy · Minoryx Therapeutics, S.L.
- Jun 2027Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy · Third Military Medical University
- Oct 2027UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells · Joanne Kurtzberg, MD
Who these studies take
Of the 16 Adrenoleukodystrophy studies recruiting now, 7 (44%) set no upper age limit and 9 (56%) stop at 75 or younger. 11 (69%) take children under 18. 2 (13%) are for women only and 2 (13%) for men only; 4 (25%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 10 (63%) | 11 (69%) |
| 18 | 13 (81%) | 12 (75%) |
| 30 | 11 (69%) | 11 (69%) |
| 40 | 11 (69%) | 11 (69%) |
| 50 | 11 (69%) | 11 (69%) |
| 65 | 8 (50%) | 7 (44%) |
| 75 | 7 (44%) | 6 (38%) |
| 85 | 6 (38%) | 5 (31%) |
Across all 68 studies, recruiting or not: 66% stop at 75 or younger and 59% at 65; 63% take children; 7% are for women only and 35% for men only; 13% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 12, 2026.
Studies
Recruiting first, then most recently updated. Showing 50 of 68. Browse all 68 → or search them.
- Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)RecruitingNCT079201981 country
- Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.RecruitingNCT048803561 country
- IT and IV Lentiviral Gene Therapy for X-ALDNot yet recruitingNCT03727555Not applicable1 country
- 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
- 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
- A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN PatientsCompletedNCT03231878Phase 2/38 countries
- 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
- A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral AdrenoleukodystrophyActive, not recruitingNCT05819866Phase 38 countries
- Treating Leg Symptoms in Women With X-linked AdrenoleukodystrophyActive, not recruitingNCT05003648Phase 42 countries
- MT2022-01: MSCs for ALDSuspendedNCT06030648Phase 11 country
- 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 Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMNCompletedNCT04973657Phase 15 countries
- Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)WithdrawnNCT05146284Phase 2
- Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)WithdrawnNCT05200104Phase 2
- 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
- Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in ChildrenWithdrawnNCT036499191 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
- A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy VolunteersCompletedNCT03500627Phase 11 country