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ClinicalTrials.gov public records Last synced Jul 23, 2026, 2:05 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Hereditary Central Nervous System Demyelinating Diseases Clear all
Available No phase listed Expanded access

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

NCT01881334
View directory record Official record
Conditions
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions
CliniMACS CD34 Reagent System
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 65 Years
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced Jul 23, 2026, 2:05 PM EDT
Recruiting Not applicable Interventional

ECoG BMI for Motor and Speech Control

NCT03698149
View directory record Official record
Conditions
ALS, SCI - Spinal Cord Injury, Stroke, Multiple Sclerosis, Muscular Dystrophies
Interventions
PMT/Blackrock Combination Device
Device
Lead sponsor
Karunesh Ganguly
Other
Eligibility
21 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Jul 23, 2026, 2:05 PM EDT
Completed No phase listed Observational

The Natural History of Infantile Globoid Cell Leukodystrophy

NCT00983879
View directory record Official record
Conditions
Infantile Globoid Cell Leukodystrophy
Interventions
Not listed
Lead sponsor
Zymenex A/S
Industry
Eligibility
Up to 2 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 7, 2018 · Synced Jul 23, 2026, 2:05 PM EDT
Terminated Phase 3 Interventional

A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

NCT00545597
View directory record Official record
Conditions
Adrenomyeloneuropathy, Adrenoleukodystrophy
Interventions
Lorenzo's oil
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 1, 2012 · Synced Jul 23, 2026, 2:05 PM EDT
Terminated No phase listed Observational

The Natural History of Mucolipidosis Type IV

NCT01067742
View directory record Official record
Conditions
Mucolipidosis Type IV
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
1 Year to 64 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2021
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Jul 23, 2026, 2:05 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

NCT01594853
View directory record Official record
Conditions
X-linked Adrenoleukodystrophy
Interventions
exercise training
Behavioral
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
21 Years to 70 Years · Female only
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced Jul 23, 2026, 2:05 PM EDT
Recruiting No phase listed Observational

Krabbe Disease Global Patient Registry

NCT02993796
View directory record Official record
Conditions
Krabbe Disease
Interventions
Not listed
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 23, 2026, 2:05 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Retinal Imaging in Neurodegenerative Disease

NCT03233646
View directory record Official record
Conditions
Alzheimer's Disease, Mild Cognitive Impairment, Parkinson's Disease, Multiple Sclerosis, Huntington Disease, Lewy Body Dementia, Frontotemporal Dementia, Amyotrophic Lateral Sclerosis (ALS), APOE-4 Positive, Traumatic Brain Injury, Concussion, Post-Traumatic Stress Disorder, Down Syndrome, Neuro-Degenerative Disease, Normal Cognition
Interventions
Retinal and Choroidal Imaging
Device
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced Jul 23, 2026, 2:05 PM EDT
Completed Phase 2 Interventional Results available

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

NCT03406962
View directory record Official record
Conditions
Inherited Metabolic Disorders (IMD)
Interventions
MGTA-456
Drug
Lead sponsor
Magenta Therapeutics, Inc.
Industry
Eligibility
0 Years to 17 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
2
States / cities
Minneapolis, Minnesota • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 2, 2021 · Synced Jul 23, 2026, 2:05 PM EDT
Terminated No phase listed Observational

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia

NCT05008874
View directory record Official record
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
Natural History Observation
Other
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
4
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Jul 23, 2026, 2:05 PM EDT
Completed Not applicable Interventional

A Wheelchair Propulsion Training Program

NCT04009187
View directory record Official record
Conditions
Spinal Cord Injuries, Multiple Sclerosis, Amputation, Spina Bifida
Interventions
In-person wheelchair propulsion training program, 30-minute education session
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 27, 2025 · Synced Jul 23, 2026, 2:05 PM EDT
No Longer Available No phase listed Expanded access

Single Patient Expanded Access Protocol: Metabolic Boost

NCT02021266
View directory record Official record
Conditions
Metachromatic Leukodystrophy
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Female only
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 7, 2020 · Synced Jul 23, 2026, 2:05 PM EDT
Recruiting No phase listed Observational

Evaluation of Outcome Metrics in Alexander Disease

NCT02714764
View directory record Official record
Conditions
Alexander Disease
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced Jul 23, 2026, 2:05 PM EDT
Terminated Phase 2 Interventional Results available

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

NCT00004418
View directory record Official record
Conditions
Adrenoleukodystrophy
Interventions
glyceryl trierucate/glyceryl trioleate
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
18 Months to 8 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2014
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 22, 2020 · Synced Jul 23, 2026, 2:05 PM EDT
Completed Not applicable Interventional

Laughter Therapy Effects on Mood, Stress and Self-efficacy in People With Neurological Diseases.

NCT02750982
View directory record Official record
Conditions
Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Brain Injury, Huntington's Disease, Multiple Sclerosis, Parkinson's Disease, Stroke, Spinal Cord Injury
Interventions
Laughter Therapy
Other
Lead sponsor
Brown, Theodore R., M.D., MPH
Individual
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Kirkland, Washington
Source: ClinicalTrials.gov public record
Updated Sep 25, 2018 · Synced Jul 23, 2026, 2:05 PM EDT
Completed Phase 2Phase 3 Interventional

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

NCT03231878
View directory record Official record
Conditions
Adrenoleukodystrophy
Interventions
MIN-102, Placebos
Drug
Lead sponsor
Minoryx Therapeutics, S.L.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
3
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 17, 2025 · Synced Jul 23, 2026, 2:05 PM EDT
Completed Phase 2 Interventional Results available

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

NCT03771898
View directory record Official record
Conditions
Metachromatic Leukodystrophy (MLD)
Interventions
SHP611
Drug
Lead sponsor
Shire
Industry
Eligibility
6 Months to 72 Months
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
11
States / cities
Torrance, California • Aurora, Colorado • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Jul 23, 2026, 2:05 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Transfer Clinical Trial for Krabbe Disease

NCT04693598
View directory record Official record
Conditions
Krabbe Disease
Interventions
FBX-101
Biological
Lead sponsor
Forge Biologics, Inc
Industry
Eligibility
1 Day to 12 Months
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Jul 23, 2026, 2:05 PM EDT
Completed Not applicable Interventional

A Randomized Exercise Trial for Wheelchair Users

NCT00866112
View directory record Official record
Conditions
Spinal Cord Injury, Multiple Sclerosis, Muscular Dystrophy, Cerebral Palsy, Spina Bifida, Amputation, Arthritis, Stroke
Interventions
Educational session to promote physical activity, Minimal contact
Other
Lead sponsor
University of Kansas
Other
Eligibility
18 Years to 65 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Lawrence, Kansas
Source: ClinicalTrials.gov public record
Updated Jun 14, 2012 · Synced Jul 23, 2026, 2:05 PM EDT
Completed Phase 3 Interventional Results available

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT03852498
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 23, 2024 · Synced Jul 23, 2026, 2:05 PM EDT
Recruiting No phase listed Observational

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

NCT06224413
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
No Intervention
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2047
U.S. locations
3
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 1, 2025 · Synced Jul 23, 2026, 2:05 PM EDT
Active, not recruiting Phase 4 Interventional

Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy

NCT05003648
View directory record Official record
Conditions
Adrenoleukodystrophy, Restless Legs Syndrome
Interventions
Pramipexole, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 75 Years · Female only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Jul 23, 2026, 2:05 PM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Jul 23, 2026, 2:05 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Unhide® Project: A Digital Health Platform to Collect Lifestyle Data for Brain Inflammation Research

NCT04806620
View directory record Official record
Conditions
Post-Acute COVID-19 Syndrome, ME/CFS, Rheumatic Arthritis, Juvenile Rheumatoid Arthritis (JRA), Psoriatic Arthritis (PsA), Ankylosing Spondylitis (AS), Autoimmune Encephalitis, Celiac Disease, Celiac Disease in Children, Chronic Lyme Disease, Post-treatment Lyme Disease Syndrome, Crohn's Disease, Dysautonomia, Anorexia Nervosa, Bulimia Nervosa, ARFID, Avoidant / Restrictive Food Intake Disorder, Ehlers Danlos Syndrome, Endometriosis, Fibromyalgia (FM), Long COVID, Lupus, Migraines, Mast Cell Activation Syndrome, Multiple Sclerosis, Myalgic Encephalomyelitis (ME), Myasthenia Gravis, Generalized, Myasthenia Gravis in Children, Narcolepsy, Obsessive Compulsive Disorder (OCD), PANDAS, Pediatric Acute-onset Neuropsychiatric Syndrome (PANS), POTS - Postural Orthostatic Tachycardia Syndrome, General Anxiety Disorder, Social Anxiety Disorder, PTSD - Post Traumatic Stress Disorder, Psoriasis, Traumatic Brain Injury, Tourette's Syndrome, Inflammatory Bowel Disease (IBD), Autoimmune Diseases, Neurological Diseases or Conditions, Psychiatric Disorder, Sjogren's Syndrome, Ulcerative Colitis and Crohn's Disease
Interventions
Not listed
Lead sponsor
Brain Inflammation Collaborative
Other
Eligibility
2 Years and older
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2030
U.S. locations
1
States / cities
Delafield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Jul 23, 2026, 2:05 PM EDT