183 completed, 75 recruiting, 71 unknown, 26 terminated. 109 Not applicable, 74 Phase 2, 41 Phase 3, 36 Phase 1, 10 Phase 4.
Follow Leukoencephalopathies: new trials by email Literature Review of Leukoencephalopathies trials Research Report on Leukoencephalopathies
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 |
| Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia Investigational Combination Product - Periventricular Leukomalacia, White Matter Disease | Early Phase 1 | Withdrawn | MGAM LLC | 2027-01-01 | 2025-07-22 | |
| 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 |
| Speech-Based Artificial Intelligence for Detection of Dementia in Danish Patients (DetectAI) Mini-mental State Examination, Addenbrooke's Cognitive Examination, Speech Task - Picture Description - Dementia (Diagnosis), Alzheimer Dementia (AD) | Not yet recruiting | Zealand University Hospital | 440 | 2026-11-01 | 2026-09-02 | |
| Music-4-MS to Improve Cognition in People Living With Multiple Sclerosis: A Feasibility Study Music Training Intervention, Music Listening - Multiple Sclerosis, Pathologic Processes | Not applicable | Recruiting | University of Texas at Austin | 64 | 2026-08-10 | 2026-08-17 |
| IDP-023 g-NK Cells Plus Ocrelizumab in Patients With Progressive Multiple Sclerosis IDP-023, Ocrelizumab, Interleukin-2 - Multiple Sclerosis, Primary Progressive Multiple Sclerosis (PPMS) | Phase 1 | Suspended | Indapta Therapeutics, INC. | 34 | 2026-06-30 | 2025-04-30 |
| Intravenous Brincidofovir as an Antiviral for Treatment of Progressive Multifocal Leukoencephalopathy: A Pilot Study Brincidofovir - Progressive Multifocal Leukoencephalopathy | Phase 2 | Recruiting | National Institute of Neurological Disorders and Stroke (NINDS) | 24 | 2026-06-05 | 2026-07-22 |
| Risk of Progressive Multifocal Leukoencephalopathy Among Patients With Multiple Sclerosis Exposed to Zeposia Ozanimod - Multiple Sclerosis, Progressive Multifocal Leukoencephalopathy | Recruiting | Bristol-Myers Squibb | 15 | 2026-06-04 | 2026-07-29 | |
| OPM- MEG in Non-immune And iMmune-medIated Neurological Disease OPM- Magnetoencephalography - Autoimmune Encephalitis (AE), Acute Disseminated Encephalomyelitis | Not applicable | Recruiting | Aston University | 90 | 2026-05-15 | 2026-10-22 |
| The Aim of the Present Study is to Determine Outcome Predictors in Children Who Were Diagnosed as Acute Disseminated Encephalomyelitis (ADEM). Acute Disseminated Encephalomyelitis, Encephalomyelitis | Not yet recruiting | Assiut University | 25 | 2026-03-29 | 2026-04-20 | |
| T-cell Therapy in Patients With PML Application of T-lymphocytes - Progressive Multifocal Leucoencephalopathy (PML) | Phase 2 | Recruiting | Hannover Medical School | 23 | 2026-02-06 | 2026-10-15 |
| Single Patient Investigational Treatment for Cree Leukoencephalopathy Fosigotifator - Cree Leukoencephalopathy | Not applicable | Active not recruiting | McGill University Health Centre/Research Institute of the McGill University Health Centre | 1 | 2025-12-03 | 2026-01-16 |
| Making Connections Thru Music Volunteer Led Making Connections Thru Music, Staff Led Making Connections Thru Music - Dementia, Alzheimer Disease | Not applicable | Recruiting | Hopeful Aging | 196 | 2025-12-01 | 2026-09-15 |
| High-throughput Omic Technology for Identification of Biomarkers of Relapsing Acute Disseminated Encephalomyelitis in Immune Cell Network Blood test - Acute Disseminated Encephalomyelitis, Encephalitis Autoimmune | Not applicable | Recruiting | University Hospital, Angers | 20 | 2025-11-16 | 2026-04-27 |
| Music Effects on Dementia Music Therapy - Dementia, Music Therapy | Not applicable | Completed | Instituto Nacional de Geriatria, Mexico | 7 | 2025-11-10 | 2026-04-21 |
| Evaluation of Cerogrin for Auricular Vagus Nerve Stimulation in Vascular Dementia or Vascular Mild Cognitive Impairment Cerogrin, Sham Device - Vascular Dementia, Mild Cognitive Impairment | Not applicable | Active not recruiting | Neurogrin Inc. | 24 | 2025-10-15 | 2026-09-18 |
| Natural History and Genotype-Phenotype Correlations in Mitochondrial Leukoencephalopathies Mitochondrial, Leukoencephalopathies | Recruiting | Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta | 100 | 2025-09-15 | 2026-10-09 | |
| Inectolizumab With Steroid Optimization in Newly Treated NMOSD Inebilizumab + Rapid Steroid Tapering group, Inebilizumab + Standard Steroid Tapering group - Neuromyelitis Optica, Autoimmune Diseases | Not applicable | Not yet recruiting | First Affiliated Hospital of Wenzhou Medical University | 25 | 2025-09-01 | 2025-09-08 |
Showing 20 of 416. Sign in to see all of them.
Dementia, Vascular (181) · Alzheimer Disease (116) · Dementia (100) · Cognitive Dysfunction (66) · Adrenoleukodystrophy (62) · Lewy Body Disease (54) · Leukodystrophy, Metachromatic (49) · Mixed Dementias (41) · Multiple Sclerosis (36) · Leukodystrophy, Globoid Cell (31) · Leukoencephalopathy, Progressive Multifocal (29) · Demyelinating Diseases (26) · Frontotemporal Dementia (26) · Demyelinating Autoimmune Diseases, CNS (24)
Brain Diseases (410) · Central Nervous System Diseases (407) · Nervous System Diseases (397) · Cardiovascular Diseases (198) · Vascular Diseases (196) · Cerebrovascular Disorders (192) · Mental Disorders (191) · Intracranial Arterial Diseases (186) · Arterial Occlusive Diseases (182) · Arteriosclerosis (181) · Intracranial Arteriosclerosis (181) · Neurocognitive Disorders (178) · Demyelinating Diseases (175) · Nutritional and Metabolic Diseases (166)
Alemtuzumab (6) · Stem Cell Transplantation (6) · Blood Specimen Collection (5) · Donepezil (5) · Cyclophosphamide (4) · Exercise (4) · Galantamine (4) · Busulfan (3) · Hematologic Tests (3) · Lorenzo's oil (3) · Magnetic Resonance Imaging (3) · Melphalan (3) · Music Therapy (3) · Risperidone (3) · trierucate (3)
Masonic Cancer Center, University of Minnesota (8) · Shire (8) · Assistance Publique - Hôpitaux de Paris (7) · Hopeful Aging (6) · National Institute of Neurological Disorders and Stroke (NINDS) (6) · Biogen (5) · Dongzhimen Hospital, Beijing (5) · Genetix Biotherapeutics Inc. (5) · University of Pennsylvania (5) · Dunjin Chen (4) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (4) · Ionis Pharmaceuticals, Inc. (4) · Mayo Clinic (4) · National Taiwan University Hospital (4) · University Health Network, Toronto (4)
United States (183) · China (41) · France (40) · Germany (36) · United Kingdom (30) · Italy (28) · Netherlands (25) · Canada (23) · Spain (18) · Australia (14) · Denmark (10) · Brazil (9) · Japan (8) · Argentina (7) · Russia (7)
Paul Orchard, MD (6) · Joanne Kurtzberg, MD (5) · Irene CM Cortese, M.D. (4) · Michael Skrajner, MA (4) · Stephen Huhn, MD (4) · Patrick Aubourg, MD, PhD (3) · Adeline Vanderver, MD (2) · Aimee Spector, PhD, DClinPsych (2) · Caroline Sevin, MD, PhD (2) · Carolyn Phillips, PhD (2) · Dunjin Chen (2)
PMID 21586746, PMID 33332761, PMID 39383458, PMID 23867200, PMID 31566651, PMID 33189083, PMID 36196048, PMID 39383459, PMID 40049590, PMID 40267426, PMID 10084637, PMID 10356105, PMID 10496251, PMID 11001602, PMID 11231629, PMID 11399225, PMID 11965273, PMID 12040306, PMID 12064356, PMID 12633121, PMID 12973660, PMID 14505162, PMID 15364702, PMID 15784740, PMID 16002461, PMID 16194720, PMID 16831964, PMID 17186320, PMID 18294247, PMID 18674411, PMID 19347838, PMID 19500421, PMID 19833552, PMID 19891819, PMID 19892975, PMID 19933977, PMID 20204386, PMID 20386637, PMID 20656516, PMID 20880607, PMID 20930266, PMID 21118724, PMID 21259015, PMID 21474353, PMID 21717193, PMID 21738597, PMID 21908805, PMID 22067635, PMID 22123780, PMID 22133740
2023 (3), 2008 (2), 2015 (2), 2017 (2), 2018 (2), 2019 (2), 2025 (2), R01AG056715 (2), U54NS078059 (2), 1RF1AG074608-01 (1), 2005 (1), K23HD101667 (1), K23NS087151 (1), R01AG011378 (1), R01AG023116 (1)