The Myelin Disorders Biorepository Project

RecruitingOBSERVATIONAL
Enrollment

12,000

Participants

Timeline

Start Date

December 8, 2016

Primary Completion Date

December 8, 2030

Study Completion Date

December 8, 2030

Conditions
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies4H SyndromeAdrenoleukodystrophyAMNALDALD Gene MutationALD (Adrenoleukodystrophy)X-linked AdrenoleukodystrophyX-ALDAdrenomyeloneuropathyAicardi Goutieres SyndromeAGSAlexander DiseaseAlexanders LeukodystrophyAxDADLDCanavan DiseaseCTXCerebrotendinous XanthomatosesKrabbe DiseaseGALC DeficiencyGloboid LeukodystrophyTUBB4A-Related LeukodystrophyH-ABC - Hypomyelination, Atrophy of Basal Ganglia and CerebellumHBSLHBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg SpasticityLBSLLeukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate ElevationALSPCSF1R Gene MutationHCC - Hypomyelination and Congenital CataractMLC1Megalencephalic Leukoencephalopathy With Subcortical CystsMLDMetachromatic LeukodystrophyPMDPelizaeus-Merzbacher DiseasePLP1 Null SyndromePLP1 Gene Duplication | Blood or Tissue | MutationsPelizaeus Merzbacher Like DiseasePeroxisomal Biogenesis DisorderZellweger SyndromeRefsum DiseaseSalla DiseaseSialic Storage DiseaseSjögrenSjogren-Larsson SyndromeVan Der Knapp DiseaseVanishing White Matter DiseaseCharcot-Marie-ToothCMTMct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter DeficiencyAllan-Herndon-Dudley SyndromeCadasilCockayne SyndromeMultiple Sulfatase DeficiencyGangliosidosesGM2 GangliosidosisBPANLabrune SyndromeLCCMucopolysaccharidosesTBCK-Related Intellectual Disability Syndrome
Trial Locations (23)

15219

RECRUITING

University of Pittsburgh Medical Center, Pittsburgh

19104

RECRUITING

The Children's Hospital of Philadelphia, Philadelphia

RECRUITING

University of Pennsylvania, Philadelphia

20010

RECRUITING

Children's National Medical Center, Washington D.C.

21205

RECRUITING

Kennedy Krieger Institute, Baltimore

27157

RECRUITING

Atrium Health Wake Forest Baptist, Winston-Salem

30342

RECRUITING

Emory University (Children's Healthcare of Atlanta), Atlanta

43205

RECRUITING

Nationwide Children's Hospital, Columbus

44308

RECRUITING

Akron Children's Hospital, Akron

55454

RECRUITING

University of Minnesota, Minneapolis

55905

RECRUITING

Mayo Clinic, Rochester

60611

RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

77030

RECRUITING

Baylor College of Medicine (Texas Children's Hospital), Houston

84112

RECRUITING

University of Utah (Primary Children's Hospital), Salt Lake City

90027

RECRUITING

Children's Hospital of Los Angeles, Los Angeles

92123

RECRUITING

University of California, San Diego (Rady Children's Hospital), San Diego

92868

RECRUITING

Children's Hospital of Orange County, Orange

94158

RECRUITING

UCSF Benioff Children's Hospital, San Francisco

94304

RECRUITING

Stanford University (Lucile Packard Children's Hospital), Palo Alto

95817

RECRUITING

University of California, Davis (UC Davis Health), Sacramento

98105

RECRUITING

Seattle Children's Hospital, Seattle

02114

RECRUITING

Massachusetts General Hospital (MGH), Boston

Unknown

RECRUITING

UT Health Houston, Houston

All Listed Sponsors
collaborator

National Institutes of Health (NIH)

NIH

collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

collaborator

National Center for Advancing Translational Sciences (NCATS)

NIH

collaborator

Biogen

INDUSTRY

collaborator

Eli Lilly and Company

INDUSTRY

collaborator

Myrtelle Inc.

INDUSTRY

collaborator

Orchard Therapeutics Ltd.

UNKNOWN

collaborator

Passage Bio, Inc.

INDUSTRY

collaborator

Synaptix Biotherapeutics Ltd.

UNKNOWN

collaborator

Takeda

INDUSTRY

collaborator

Boehringer Ingelheim

INDUSTRY

collaborator

Ionis Pharmaceuticals, Inc.

INDUSTRY

collaborator

Sanofi Winthrop Industrie

UNKNOWN

collaborator

Sana Biotechnology

INDUSTRY

collaborator

Yaya Foundation for 4H Leukodystrophy

UNKNOWN

collaborator

University of Pennsylvania

OTHER

collaborator

United MSD Foundation

UNKNOWN

collaborator

Foundation to Fight H-ABC

UNKNOWN

collaborator

Calliope Joy Foundation

UNKNOWN

collaborator

Don't Forget Me Foundation

UNKNOWN

lead

Children's Hospital of Philadelphia

OTHER