Leukoencephalopathies clinical trials
The registry lists 39 studies for Leukoencephalopathies across more than 10 countries. 16 are recruiting or about to start. 21 of them are mainly about Leukoencephalopathies (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06. Counting its 13 narrower conditions too, such as Multiple Sclerosis: 4,184 studies, 859 open.
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Who runs them
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (2) · Aspa Therapeutics (2) · SwanBio Therapeutics, Inc. (2) · IRCCS Eugenio Medea (2) · Children's Hospital of Philadelphia (2) · McGill University Health Centre/Research Institute of the McGill University Health Centre (2) · Vigil Neuroscience, Inc. (2) · Fondation Ophtalmologique Adolphe de Rothschild (1)
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
- Dec 2026Natural History and Genotype-Phenotype Correlations in Mitochondrial Leukoencephalopathies · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
- Dec 2026North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) · Columbia University
- Dec 2026Natural History Study of Patients with HPDL Mutations · University of California, San Diego
- Nov 2027An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease · Calico Life Sciences LLC
- Nov 2027Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease · McGill University Health Centre/Research Institute of the McGill University Health Centre
Who these studies take
Of the 15 Leukoencephalopathies studies recruiting now, 11 (73%) set no upper age limit and 3 (20%) stop at 75 or younger. 11 (73%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 3 (20%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 9 (60%) | 9 (60%) |
| 18 | 12 (80%) | 12 (80%) |
| 30 | 13 (87%) | 13 (87%) |
| 40 | 13 (87%) | 13 (87%) |
| 50 | 13 (87%) | 13 (87%) |
| 65 | 13 (87%) | 13 (87%) |
| 75 | 13 (87%) | 13 (87%) |
| 85 | 11 (73%) | 11 (73%) |
Across all 39 studies, recruiting or not: 23% stop at 75 or younger and 15% at 65; 56% take children; 0% are for women only and 5% for men only; 23% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 39 of 39. search them.
- Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.RecruitingNCT048803561 country
- An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter DiseaseRecruitingNCT05757141Phase 1/23 countries
- Natural History and Genotype-Phenotype Correlations in Mitochondrial LeukoencephalopathiesRecruitingNCT078687961 country
- 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
- Diffusion Magnetic Resonance Imaging (dMRI) in the Early Evaluation of Brain White Matter DiseasesRecruitingNCT071087121 country
- 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
- Imaging - Clinical Evaluation of Altered Nervous System DrainageRecruitingNCT064320231 country
- The Fingerprinting of Inherited Leukoencephalopathies: A New Brain Imaging, Genetic and Clinical AssessmentRecruitingNCT063326251 country
- Quantifying Disease Progression in LBSLRecruitingNCT057509791 country
- INflammation and Small Vessel Disease StudyRecruitingNCT057462212 countries
- Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCTActive, not recruitingNCT05739643Phase 1/21 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
- 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
- Idiopathic Normal Pressure Hydrocephalus: Focus on Imaging and Clinical Symptoms.CompletedNCT04808076Not applicable1 country
- Evaluation of Brain Waste Clearance Pathways Using Magnetic Resonance Imaging in Pediatric Patients With White Matter DiseasesActive, not recruitingNCT063350041 country
- Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry PatientsCompletedNCT045771701 country
- Brain Networks and Mobility Function: B-NETCompletedNCT034304271 country
- Remote Preconditioning Over Time To Empower Cerebral TissueTerminatedNCT02169739Not applicable1 country
- The Relationship Between Right-to-left Shunt and Brain White Matter Lesions in Patients With MigraineStatus unknownNCT034187661 country
- The Nosology and Etiology of Leukodystrophies of Unknown CausesCompletedNCT008891741 country
- Study of Neurological Complication After Radiotherapy for High Grade GlioblastomaStatus unknownNCT025441781 country
- COL4A1 Gene Related Cerebra-retinal AngiopathyCompletedNCT010975641 country
- Hyperbaric Oxygen Treatment in Patients With White Matter HyperintensitiesCompletedNCT004974321 country
- Research Study on Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy (CADASIL)CompletedNCT011148151 country