Leukodystrophy: Clinical Trials

7 trials for Leukodystrophy, newest first

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5 recruiting, 2 completed. 1 Not applicable.

By phase and status

PhaseTrialsStatus
Not applicable1Recruiting 1

Follow Leukodystrophy: new trials by email Literature Review of Leukodystrophy trials Research Report on Leukodystrophy

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
Home Exercise for Individuals With Neurodegenerative Disease
Home exercise - Neurodegenerative Diseases, Leukodystrophy
Not applicableRecruitingHugo W. Moser Research Institute at Kennedy Krieger, Inc.302023-02-132026-09-16
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophy, White Matter Disease
CompletedChildren's Hospital of Philadelphia2362017-01-062025-11-10
The Myelin Disorders Biorepository Project
Leukodystrophy, White Matter Disease
RecruitingChildren's Hospital of Philadelphia120002016-12-082026-11-12
Longitudinal Study of Neurodegenerative Disorders
Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease
RecruitingUniversity of Pittsburgh15002012-01-112026-02-09
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingSanford Health200002010-07-012026-10-28
The Nosology and Etiology of Leukodystrophies of Unknown Causes
Leukodystrophy, Leukoencephalopathy
CompletedNational Human Genome Research Institute (NHGRI)762009-04-242018-08-03
Natural History, Diagnosis, and Outcomes for Leukodystrophies
Leukodystrophy
RecruitingUniversity of Utah6002007-01-192026-01-13

Narrower conditions

Leukoencephalopathies (4)  ·  Adrenoleukodystrophy (3)  ·  Cockayne Syndrome (3)  ·  Leukodystrophy, Globoid Cell (3)  ·  Leukodystrophy, Metachromatic (3)  ·  Leukoencephalopathy Brain Calcifications and Cysts (3)  ·  Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation (3)  ·  Multiple Sulfatase Deficiency Disease (3)  ·  Pelizaeus-Merzbacher Disease (3)  ·  Refsum Disease (3)  ·  Aicardi-Goutieres syndrome (2)  ·  Alexander Disease (2)  ·  Alexanders leukodystrophy (2)  ·  Allan-Herndon-Dudley syndrome (2)  ·  Ataxia (2)

Broader conditions

Brain Diseases (6)  ·  Central Nervous System Diseases (6)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6)  ·  Nervous System Diseases (6)  ·  Brain Diseases, Metabolic (5)  ·  Brain Diseases, Metabolic, Inborn (5)  ·  Demyelinating Diseases (5)  ·  Genetic Diseases, Inborn (5)  ·  Genetic Diseases, X-Linked (5)  ·  Hereditary Central Nervous System Demyelinating Diseases (5)  ·  Heredodegenerative Disorders, Nervous System (5)  ·  Metabolic Diseases (5)  ·  Metabolism, Inborn Errors (5)  ·  Nutritional and Metabolic Diseases (5)  ·  Pathological Conditions, Signs and Symptoms (5)

Interventions

Palliative Care (1)

Sponsors

Children's Hospital of Philadelphia (2)  ·  Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1)  ·  National Human Genome Research Institute (NHGRI) (1)  ·  Sanford Health (1)  ·  University of Pittsburgh (1)  ·  University of Utah (1)

Countries

United States (7)

Top investigators

Adeline Vanderver, MD (2)  ·  Jennifer Keller, PT (1)  ·  William A Gahl, M.D. (1)

Results publications

PMID 32342562, PMID 32749716

NIH funding

U01NS106845 (1), U54NS115052 (1)