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Results for “Vanishing White Matter Disease”

8 trials

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Showing 8 of 8 results

Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Not applicableNo Longer AvailableNCT06594016
What this trial is testing

Expanded Access to Fosigotifator

Who this might be right for
Vanishing White MatterCree Leukoencephalopathy
AbbVie
Not applicableLooking for participantsNCT04880356
What this trial is testing

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Who this might be right for
Inherited DiseaseRare DiseasesMetabolic Disease+3 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Not applicableActive Not RecruitingNCT07300397
What this trial is testing

Single Patient Investigational Treatment for Cree Leukoencephalopathy

Who this might be right for
Cree Leukoencephalopathy
McGill University Health Centre/Research Institute of the McGill University Health Centre 1
Very early researchActive Not RecruitingNCT07272525
What this trial is testing

Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease

Who this might be right for
Cree LeukoencephalopathyVanishing White Matter
McGill University Health Centre/Research Institute of the McGill University Health Centre 1
Not applicableLooking for participantsNCT03047369
What this trial is testing

The Myelin Disorders Biorepository Project

Who this might be right for
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of Philadelphia 12,000
Testing effectiveness (Phase 2)Looking for participantsNCT05757141
What this trial is testing

An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

Who this might be right for
Vanishing White Matter Disease
Calico Life Sciences LLC 50