46 completed, 21 recruiting, 13 terminated, 13 withdrawn. 37 Phase 2, 25 Phase 1, 20 Not applicable, 11 Phase 3, 1 Phase 4.
Follow Hereditary Central Nervous System Demyelinating Diseases: new trials by email Literature Review of Hereditary Central Nervous System Demyelinating Diseases trials Research Report on Hereditary Central Nervous System Demyelinating Diseases
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 |
|---|---|---|---|---|---|---|
| Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD Lentiviral TYF-ARSA correction of patient's autologous HSCs - Metachromatic Leukodystrophy (MLD) | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-06-01 | 2026-09-01 |
| 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 | 2026-09-01 |
| Direct Lentiviral Injection Gene Therapy for MLD Intrathecal and intravenous LV gene therapy - Metachromatic Leukodystrophy (MLD) | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-05-31 | 2026-09-01 |
| Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) PLX-200 - Lysosomal Storage Disorders, Sandhoff Disease | Phase 2 | Not yet recruiting | Polaryx Therapeutics, Inc. | 24 | 2026-12-01 | 2026-07-31 |
| 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 |
| Mesh-Free Versus Mesh-Based Surgery for Female Stress Urinary Incontinence: A Prospective Comparison of Pubo-Urethral Ligament Plication and Transobturator Tape Pubo-Urethral Ligament Plication (PLP), Transobturator Tape (TOT) - Stress Urinary Incontinence (SUI) | Not applicable | Recruiting | Mehmet Incebıyik | 147 | 2025-01-13 | 2025-12-18 |
| Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients FBX-101 - Krabbe Disease | Terminated | Forge Biologics, Inc | 2 | 2024-08-06 | 2025-09-05 | |
| Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD) ION356 - Pelizaeus-Merzbacher Disease | Phase 1 | Recruiting | Ionis Pharmaceuticals, Inc. | 24 | 2024-04-10 | 2025-12-12 |
| 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 | |
| A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain No Intervention - Metachromatic Leukodystrophy (MLD) | Withdrawn | Takeda | 2023-11-30 | 2024-01-12 | ||
| 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 |
| Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT FBX-101 - Krabbe Disease | Phase 1 / Phase 2 | Active not recruiting | Forge Biologics, Inc | 9 | 2023-02-03 | 2025-11-20 |
| 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 |
| Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease Pelizaeus-Merzbacher Disease | Recruiting | Ionis Pharmaceuticals, Inc. | 32 | 2022-10-03 | 2026-10-02 |
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Adrenoleukodystrophy (62) · Leukodystrophy, Metachromatic (49) · Leukodystrophy, Globoid Cell (31) · Mucopolysaccharidosis I (17) · Metabolism, Inborn Errors (16) · Canavan Disease (13) · Lysosomal Storage Diseases (13) · Peroxisomal Disorders (13) · Rare Diseases (13) · Sphingolipidoses (12) · Pelizaeus-Merzbacher Disease (11) · Mucopolysaccharidosis II (10) · Mucopolysaccharidosis VI (10) · Leukoencephalopathies (9) · Tay-Sachs Disease (9)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (124) · Nutritional and Metabolic Diseases (124) · Brain Diseases (122) · Brain Diseases, Metabolic (121) · Nervous System Diseases (120) · Central Nervous System Diseases (119) · Brain Diseases, Metabolic, Inborn (118) · Genetic Diseases, Inborn (118) · Demyelinating Diseases (117) · Metabolic Diseases (117) · Leukoencephalopathies (115) · Metabolism, Inborn Errors (108) · Heredodegenerative Disorders, Nervous System (80) · Genetic Diseases, X-Linked (72)
Stem Cell Transplantation (6) · Alemtuzumab (5) · Busulfan (3) · Cyclophosphamide (3) · Lorenzo's oil (3) · Melphalan (3) · trierucate (3) · Bone Marrow Transplantation (2) · Cholic Acid (2) · Clofarabine (2) · Cyclosporine (2) · Exercise (2) · GC 1 compound (2) · Hematopoietic Stem Cell Transplantation (2) · Hydroxyurea (2)
Masonic Cancer Center, University of Minnesota (8) · Shire (8) · Assistance Publique - Hôpitaux de Paris (5) · Genetix Biotherapeutics Inc. (5) · Ionis Pharmaceuticals, Inc. (4) · Children's Hospital of Philadelphia (3) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (3) · Joanne Kurtzberg, MD (3) · Orchard Therapeutics (3) · Pujol, Aurora, M.D. (3) · Shenzhen Geno-Immune Medical Institute (3) · Talaris Therapeutics Inc. (3) · University of Pittsburgh (3) · Aspa Therapeutics (2) · Forge Biologics, Inc (2)
United States (72) · Germany (18) · France (17) · Netherlands (13) · Italy (12) · United Kingdom (10) · Australia (7) · China (7) · Spain (7) · Argentina (6) · Canada (6) · Denmark (6) · Israel (6) · Japan (6) · Brazil (5)
Paul Orchard, MD (6) · Joanne Kurtzberg, MD (5) · Patrick Aubourg, MD, PhD (3) · Stephen Huhn, MD (3) · Adeline Vanderver, MD (2) · Caroline Sevin, MD, PhD (2) · Florian Eichler, MD (2) · Gerald V Raymond, M.D. (2) · John Henderson, MD (2) · Paul Szabolcs, MD (2) · Suzanne T Ildstad, MD (2) · Adrian Thrasher, MD, PhD (1)
PMID 21586746, PMID 33332761, PMID 39383458, PMID 36196048, PMID 39383459, PMID 40267426, PMID 11231629, PMID 11399225, PMID 12064356, PMID 15784740, PMID 16002461, PMID 16194720, PMID 19833552, PMID 19892975, PMID 21474353, PMID 22911730, PMID 23845948, PMID 23966770, PMID 25487283, PMID 25758611, PMID 26000324, PMID 27289174, PMID 28644367, PMID 28976817, PMID 31077039, PMID 31214256, PMID 31315600, PMID 32342562, PMID 32634238, PMID 32749716, PMID 32750127, PMID 32792226, PMID 34781360, PMID 35065785, PMID 35142618, PMID 35333110, PMID 35778568, PMID 36681445, PMID 37782421, PMID 37920915, PMID 39499628, PMID 40957959, PMID 41030609, PMID 41667276
2018 (2), 2008 (1), 2015 (1), 2017 (1), 2019 (1), K23NS087151 (1), R01HD073292 (1), R01HD104814 (1), R01NS061965 (1), U01NS106845 (1), U54NS065768 (1), U54NS115052 (1)