24 completed, 14 recruiting, 8 terminated, 6 withdrawn. 18 Phase 2, 12 Not applicable, 10 Phase 1, 9 Phase 3, 1 Phase 4.
Follow Adrenoleukodystrophy: new trials by email Literature Review of Adrenoleukodystrophy trials Research Report on Adrenoleukodystrophy
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| IT and IV Lentiviral Gene Therapy for X-ALD Intrathecal and intravenous LV gene therapy - X-linked Adrenoleukodystrophy | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 30 | 2027-06-01 | 2026-11-02 |
| 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) Cushing s Syndrome, Cushing Disease | Recruiting | Azienda Ospedaliero-Universitaria di Modena | 2000 | 2026-07-09 | 2026-11-09 | |
| Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy Spinal cord stimulation - Adrenomyeloneuropathy Without Cerebral Involvement | Not applicable | Recruiting | Third Military Medical University | 10 | 2025-02-10 | 2025-01-28 |
| A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel No Intervention - Cerebral Adrenoleukodystrophy (CALD) | Recruiting | Genetix Biotherapeutics Inc. | 120 | 2024-03-27 | 2025-07-02 | |
| Disease Progression in Women With X-linked Adrenoleukodystrophy X-linked Adrenoleukodystrophy | Recruiting | Minoryx Therapeutics, S.L. | 40 | 2024-01-02 | 2024-01-31 | |
| Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) PXL065 - ALD (Adrenoleukodystrophy) | Phase 2 | Withdrawn | Poxel SA | 2023-09-01 | 2024-12-16 | |
| Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) PXL770 - Adrenomyeloneuropathy | Phase 2 | Withdrawn | Poxel SA | 2023-09-01 | 2024-12-16 | |
| Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked Adrenoleukodystrophy brainQuant module of brainTale-care platform - X-linked Adrenoleukodystrophy | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 200 | 2023-09-01 | 2023-07-03 | |
| Registry of X-linked Adrenoleukodystrophy X-linked Adrenoleukodystrophy | Recruiting | Beijing Tiantan Hospital | 200 | 2023-07-20 | 2023-07-11 | |
| Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy Pramipexole, Placebo - Adrenoleukodystrophy, Restless Legs Syndrome | Phase 4 | Active not recruiting | Massachusetts General Hospital | 24 | 2023-04-01 | 2026-07-01 |
| Home Exercise for Individuals With Neurodegenerative Disease Home exercise - Neurodegenerative Diseases, Leukodystrophy | Not applicable | Recruiting | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | 30 | 2023-02-13 | 2026-09-16 |
| A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) SBT101 Treatment - AMN, AMN Gene Mutation | Phase 1 / Phase 2 | Terminated | SwanBio Therapeutics, Inc. | 9 | 2022-11-17 | 2026-08-26 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD) SMART-ALD, Waiting list - X-linked Adrenoleukodystrophy | Not applicable | Completed | Leipzig University Medical Center | 68 | 2022-05-20 | 2024-03-13 |
| Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children Xlsma, DMD | Withdrawn | Children's Hospital of Fudan University | 2021-09-30 | 2021-08-11 | ||
| Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia Blood sample collection - Adrenoleukodystrophy, Adrenomyeloneuropathy | Recruiting | Assistance Publique - Hôpitaux de Paris | 100 | 2021-08-30 | 2025-09-24 | |
| Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia Natural History Observation - AMN, AMN Gene Mutation | Terminated | SwanBio Therapeutics, Inc. | 65 | 2021-06-21 | 2025-10-30 | |
| Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases. collection of data - Inherited Disease, Rare Diseases | Recruiting | Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta | 100 | 2021-03-01 | 2026-11-06 | |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC data collection - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Recruiting | Paul Szabolcs | 50 | 2020-08-20 | 2026-01-13 | |
| Plasma Exchange With Albumin in AMN Patients Albumin solution - Adrenomyeloneuropathy, Adrenoleukodystrophy | Phase 2 / Phase 3 | Completed | Pujol, Aurora, M.D. | 5 | 2020-03-09 | 2022-09-22 |
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Leukodystrophy, Metachromatic (17) · Leukodystrophy, Globoid Cell (14) · Peroxisomal Disorders (10) · Metabolism, Inborn Errors (9) · Mucopolysaccharidosis I (9) · Gaucher Disease (6) · Mucopolysaccharidoses (6) · Mucopolysaccharidosis II (6) · Rare Diseases (6) · Sandhoff Disease (6) · Sphingolipidoses (6) · Tay-Sachs Disease (6) · Wolman Disease (6) · alpha-Mannosidosis (6)
Brain Diseases (63) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (63) · Endocrine System Diseases (63) · Intellectual Disability (63) · Neurobehavioral Manifestations (63) · Neurologic Manifestations (63) · Nutritional and Metabolic Diseases (63) · X-Linked Intellectual Disability (63) · Adrenal Gland Diseases (62) · Brain Diseases, Metabolic (61) · Genetic Diseases, X-Linked (61) · Hereditary Central Nervous System Demyelinating Diseases (61) · Heredodegenerative Disorders, Nervous System (61) · Nervous System Diseases (61) · Adrenal Insufficiency (60)
Alemtuzumab (4) · Lorenzo's oil (3) · Melphalan (3) · Stem Cell Transplantation (3) · trierucate (3) · Busulfan (2) · Cholic Acid (2) · Clofarabine (2) · Cyclophosphamide (2) · Cyclosporine (2) · GC 1 compound (2) · Hematopoietic Stem Cell Transplantation (2) · Hydroxyurea (2) · Mycophenolic Acid (2) · Triolein (2)
Masonic Cancer Center, University of Minnesota (6) · Genetix Biotherapeutics Inc. (5) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (3) · Pujol, Aurora, M.D. (3) · Assistance Publique - Hôpitaux de Paris (2) · Children's Hospital of Philadelphia (2) · Joanne Kurtzberg, MD (2) · Leipzig University Medical Center (2) · Minoryx Therapeutics, S.L. (2) · NeuroVia, Inc. (2) · Paul Szabolcs (2) · Poxel SA (2) · SwanBio Therapeutics, Inc. (2) · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) (1) · Aldagen (1)
United States (36) · Germany (9) · France (8) · Netherlands (8) · Italy (7) · United Kingdom (6) · China (5) · Spain (5) · Argentina (4) · Australia (3) · Taiwan (2) · Belgium (1) · Brazil (1) · Canada (1) · Chile (1)
Paul Orchard, MD (5) · Joanne Kurtzberg, MD (3) · Adeline Vanderver, MD (2) · Gerald V Raymond, M.D. (2) · John Henderson, MD (2) · Paul Szabolcs, MD (2) · Adrian Thrasher, MD, PhD (1) · Andrea Biondi, MD, PhD (1) · Biljana Horn, MD (1) · Christine Duncan, MD (1) · David Koeller, MD (1) · David Williams, MD (1)
PMID 39383458, PMID 21586746, PMID 39383459, PMID 11231629, PMID 11399225, PMID 19833552, PMID 19892975, PMID 22911730, PMID 23845948, PMID 23966770, PMID 26121453, PMID 27479926, PMID 28644367, PMID 28976817, PMID 31077039, PMID 31214256, PMID 32266384, PMID 32342562, PMID 32634238, PMID 32749716, PMID 32750127, PMID 34781360, PMID 35778568, PMID 36681445, PMID 37318239, PMID 37782421, PMID 38151399, PMID 41030609, PMID 41667276
2015 (1), 2018 (1), K23NS087151 (1), U01NS106845 (1), U54NS115052 (1)