The Myelin Disorders Biorepository Project
NCT03047369
This study is recruiting at 23 US sites in 13 states, according to its ClinicalTrials.gov record last updated .
Summary
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago.
Shortened from the sponsor's brief summary. View official record on ClinicalTrials.gov
Conditions studied
- Leukodystrophy
- White Matter Disease
- Leukoencephalopathies
- 4H Syndrome
- Adrenoleukodystrophy
- AMN
- ALD
- ALD Gene Mutation
- ALD (Adrenoleukodystrophy)
- X-linked Adrenoleukodystrophy
- X-ALD
- Adrenomyeloneuropathy
- Aicardi Goutieres Syndrome
- AGS
- Alexander Disease
- Alexanders Leukodystrophy
- AxD
- ADLD
- Canavan Disease
- CTX
- Cerebrotendinous Xanthomatoses
- Krabbe Disease
- GALC Deficiency
- Globoid Leukodystrophy
- TUBB4A-Related Leukodystrophy
- H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum
- HBSL
- HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity
- LBSL
- Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)
- Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation
- ALSP
- CSF1R Gene Mutation
- HCC - Hypomyelination and Congenital Cataract
- MLC1
- Megalencephalic Leukoencephalopathy With Subcortical Cysts
- MLD
- Metachromatic Leukodystrophy
- PMD
- Pelizaeus-Merzbacher Disease
- PLP1 Null Syndrome
- PLP1 Gene Duplication | Blood or Tissue | Mutations
- Pelizaeus Merzbacher Like Disease
- Peroxisomal Biogenesis Disorder
- Zellweger Syndrome
- Refsum Disease
- Salla Disease
- Sialic Storage Disease
- Sjögren
- Sjogren-Larsson Syndrome
- Van Der Knapp Disease
- Vanishing White Matter Disease
- Charcot-Marie-Tooth
- CMT
- Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency
- Allan-Herndon-Dudley Syndrome
- Cadasil
- Cockayne Syndrome
- Multiple Sulfatase Deficiency
- Gangliosidoses
- GM2 Gangliosidosis
- BPAN
- Labrune Syndrome
- LCC
- Mucopolysaccharidoses
- TBCK-Related Intellectual Disability Syndrome
Eligibility basics and study details
- Study type
- Observational
- Phase
- Not applicable
- Ages
- Any age
- Sex
- All
- Healthy volunteers
- Not accepted
- Sponsor
- Children's Hospital of Philadelphia
- Last updated on ClinicalTrials.gov
- October 23, 2025
These are the basic criteria only. The full inclusion and exclusion criteria are on the official record, and the study team decides who can take part.
US recruiting sites
California · 6
- Children's Hospital of Los Angeles · Los Angeles, CA
- Children's Hospital of Orange County · Orange, CA
- Stanford University (Lucile Packard Children's Hospital) · Palo Alto, CA
- University of California, Davis (UC Davis Health) · Sacramento, CA
- University of California, San Diego (Rady Children's Hospital) · San Diego, CA
- UCSF Benioff Children's Hospital · San Francisco, CA
District of Columbia · 1
- Children's National Medical Center · Washington D.C., DC
Georgia · 1
- Emory University (Children's Healthcare of Atlanta) · Atlanta, GA
Illinois · 1
- Ann & Robert H. Lurie Children's Hospital of Chicago · Chicago, IL
Maryland · 1
- Kennedy Krieger Institute · Baltimore, MD
Massachusetts · 1
- Massachusetts General Hospital (MGH) · Boston, MA
Minnesota · 2
- University of Minnesota · Minneapolis, MN
- Mayo Clinic · Rochester, MN
North Carolina · 1
- Atrium Health Wake Forest Baptist · Winston-Salem, NC
Ohio · 2
- Akron Children's Hospital · Akron, OH
- Nationwide Children's Hospital · Columbus, OH
Pennsylvania · 3
- The Children's Hospital of Philadelphia · Philadelphia, PA
- University of Pennsylvania · Philadelphia, PA
- University of Pittsburgh Medical Center · Pittsburgh, PA
Texas · 2
- Baylor College of Medicine (Texas Children's Hospital) · Houston, TX
- UT Health Houston · Houston, TX
Utah · 1
- University of Utah (Primary Children's Hospital) · Salt Lake City, UT
Washington · 1
- Seattle Children's Hospital · Seattle, WA
Source: ClinicalTrials.gov, data processed . Recruiting US sites filtered by distance and site status; summaries shortened.