Clinical trialNCT03047369RecruitingObservational

The Myelin Disorders Biorepository Project

SponsorChildren's Hospital of PhiladelphiaEnrollment12,0004H SyndromeADLDAGSALDALD (Adrenoleukodystrophy)ALD Gene Mutation+60 more conditions

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

Adeline Vanderver, MD
Children's Hospital of Philadelphia
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

23 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

Children'S Hospital of Los Angeles
Los Angeles, California, United States
Recruiting
Children'S Hospital of Orange County
Orange, California, United States
Recruiting
Stanford University (lucile Packard Children'S Hospital)
Palo Alto, California, United States
Recruiting
University of California, Davis (uc Davis Health)
Sacramento, California, United States
Recruiting
University of California, San Diego (rady Children'S Hospital)
San Diego, California, United States
Recruiting
Ucsf Benioff Children'S Hospital
San Francisco, California, United States
Recruiting
Children'S National Medical Center
Washington D.C., District of Columbia, United States
Recruiting
Emory University (children'S Healthcare of Atlanta)
Atlanta, Georgia, United States
Recruiting
Ann & Robert H. Lurie Children'S Hospital of Chicago
Chicago, Illinois, United States
Recruiting
Kennedy Krieger Institute
Baltimore, Maryland, United States
Recruiting
Massachusetts General Hospital (mgh)
Boston, Massachusetts, United States
Recruiting
University of Minnesota
Minneapolis, Minnesota, United States
Recruiting
Mayo Clinic
Rochester, Minnesota, United States
Recruiting
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, United States
Recruiting
Akron Children'S Hospital
Akron, Ohio, United States
Recruiting
Nationwide Children'S Hospital
Columbus, Ohio, United States
Recruiting
the Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Adeline Vanderver, MDAmy Waldman, MD, MSCELaura Adang, MD, PhD, MSTR
Recruiting
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Recruiting
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Recruiting
Baylor College of Medicine (texas Children'S Hospital)
Houston, Texas, United States
Recruiting
Ut Health Houston
Houston, Texas, United States
Recruiting
University of Utah (primary Children'S Hospital)
Salt Lake City, Utah, United States
Recruiting
Seattle Children'S Hospital
Seattle, Washington, United States
Recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 8 Dec 2016
Primary completion: 8 Dec 2030
Completion: 8 Dec 2030
Last update posted: 23 Oct 2025
Registered enrollment: 12,000
Registry id: NCT03047369

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.