Leukoencephalopathies clinical trials
The registry lists 57 studies for Leukoencephalopathies across more than 10 countries. 13 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-09-24.
Status
- Completed
- 24
- Recruiting
- 11
- Terminated
- 9
- Status unknown
- 5
- Active, not recruiting
- 4
- Enrolling by invitation
- 1
Phase
- Phase 2
- 12
- Phase 1
- 5
- Early Phase 1
- 2
- Phase 3
- 2
- Phase 4
- 2
Where
- United States
- 33
- Canada
- 8
- Germany
- 7
- Netherlands
- 6
- France
- 5
- Italy
- 4
Who runs them
Biogen (5) · Fondation Ophtalmologique Adolphe de Rothschild (3) · McGill University Health Centre/Research Institute of the McGill University Health Centre (2) · Children's Hospital of Philadelphia (2) · Beth Israel Deaconess Medical Center (2) · National Institute of Allergy and Infectious Diseases (NIAID) (2) · SwanBio Therapeutics, Inc. (2) · Aspa Therapeutics (2)
Studies
Recruiting first, then most recently updated. Showing 50 of 57 — search for more.
- An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter DiseaseRecruitingNCT05757141Phase 1/23 countries
- Long-term Prospective Study of Korean CADASIL PatientsRecruitingNCT074978671 country
- A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)RecruitingNCT04998396Phase 1/21 country
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT033332001 country
- North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)RecruitingNCT016949402 countries
- A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell TransplantationEnrolling by invitationNCT045032131 country
- The Myelin Disorders Biorepository ProjectRecruitingNCT030473691 country
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordRecruitingNCT017931682 countries
- Natural History Study of Patients with HPDL MutationsRecruitingNCT058482711 country
- Natural History Study of Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation (LBSL)RecruitingNCT036243741 country
- The Fingerprinting of Inherited Leukoencephalopathies: A New Brain Imaging, Genetic and Clinical AssessmentRecruitingNCT063326251 country
- Pembrolizumab in Progressive Multifocal Leukoencephalopathy (PML) in Immunocompromised Patients Without HIV InfectionNot yet recruitingNCT06276504Phase 2/31 country
- Quantifying Disease Progression in LBSLRecruitingNCT057509791 country
- A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)TerminatedNCT05394064Phase 1/22 countries
- Natural History Study of Patients With Canavan Disease (CANinform Study)CompletedNCT041260052 countries
- Single Patient Investigational Treatment for Cree LeukoencephalopathyActive, not recruitingNCT07300397Not applicable1 country
- Expanded Access to FosigotifatorNo longer availableNCT06594016
- Assessment of the Impact of Gadolinium Injection on the Measurement of the QSM SignalCompletedNCT049069411 country
- Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter DiseaseActive, not recruitingNCT07272525Early Phase 11 country
- Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCTActive, not recruitingNCT05739643Phase 1/21 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
- Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented GliaTerminatedNCT050207436 countries
- Cell-Based Therapy for White Matter Repair in Periventricular LeukomalaciaWithdrawnNCT06985303Early Phase 1
- A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented GliaTerminatedNCT05677659Phase 25 countries
- TOPIK Study: A Study to Report Progressive Multifocal Leukoencephalopathy and Other Serious Opportunistic Infections in Natalizumab Treated ParticipantsCompletedNCT052367771 country
- Idiopathic Normal Pressure Hydrocephalus: Focus on Imaging and Clinical Symptoms.CompletedNCT04808076Not applicable1 country
- Amyloid and Glucose PET Imaging in Alzheimer and Vascular Cognitive Impairment Patients With Significant White Matter DiseaseCompletedNCT023305101 country
- Cerebrovascular Reserve and White Matter Disease in Patients with Chronic AnemiaCompletedNCT037159721 country
- Observational Study on Characteristics and Survival Correlates of Progressive Multifocal Leukoencephalopathy (PML) in ItalyCompletedNCT065946141 country
- Evaluation of Brain Waste Clearance Pathways Using Magnetic Resonance Imaging in Pediatric Patients With White Matter DiseasesActive, not recruitingNCT063350041 country
- Tysabri Observational Cohort Study - Multiple Sclerosis (MS) RegistriesCompletedNCT033999811 country
- Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry PatientsCompletedNCT045771701 country
- Brain Networks and Mobility Function: B-NETCompletedNCT034304271 country
- Role of Inflammation in Progressive Multifocal Leukoencephalopathy (PML)CompletedNCT011320531 country
- A Phase 2 Study Evaluating JCPyV-specific T Cell Therapy for PMLStatus unknownNCT05541549Phase 2
- Comparison of Anti HIV Drugs Used Alone or in Combination With Cytosine Arabinoside to Treat Progressive Multifocal Leukoencephalopathy (PML) in HIV-Infected PatientsCompletedNCT00001048Phase 21 country
- A Study to Evaluate the Use of Cidofovir (an Experimental Drug) for the Treatment of Progressive Multifocal Leukoencephalopathy (PML) in AIDS PatientsCompletedNCT00000945Not applicable1 country
- The Relationship Between Right-to-left Shunt and Brain White Matter Lesions in Patients With MigraineStatus unknownNCT034187661 country
- Treatment of PD-1 Inhibitor in AIDS-associated PMLStatus unknownNCT04091932Phase 21 country
- Do CCR5 Antagonists Improve the Overall Survival of Patients With AIDS-related Progressive Multifocal Leucoencephalopathy?CompletedNCT03969550
- The Nosology and Etiology of Leukodystrophies of Unknown CausesCompletedNCT008891741 country
- Therapeutic Effects of Maternal Melatonin Administration on Brain Injury and White Matter DiseaseTerminatedNCT02395783Phase 2/31 country
- Initial Efficacy Study of Supporting Play, Exploration, & Early Development InterventionCompletedNCT02153736Not applicable1 country
- Relation of Carotid Artery Plaque Inflammation, Covert Stroke and White Matter DiseaseTerminatedNCT01236508Phase 41 country
- Study of Neurological Complication After Radiotherapy for High Grade GlioblastomaStatus unknownNCT025441781 country
- Retrospective Study of the Survival of Patients Suffering From Hiv-related Progressive Multifocal LeukoencephalopathyCompletedNCT02895581
- Cognitive Changes and Rehabilitation in People With Transient Ischemic Attack, Stroke, or Stroke Risk FactorsStatus unknownNCT01951612Not applicable1 country
- JC Virus Reactivation in Multiple SclerosisCompletedNCT020044441 country
- Genetic Evaluation of Natalizumab-Treated Patients With Progressive Multifocal LeukoencephalopathyTerminatedNCT012116392 countries