NCT03047369Recruiting
The Myelin Disorders Biorepository Project
The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
Source: ClinicalTrials.gov · Last updated 23 Oct 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 12000 (estimated)
- First submitted
- 1 Feb 2017
- Start date
- 8 Dec 2016
- Primary completion date
- 8 Dec 2030
- Lead sponsor
- Children's Hospital of Philadelphia
- Diseases
- Sjögren's disease
- NCT number
- NCT03047369
What you commit to
- Study type: Observational
- Planned participants: 12000
- From 8 Dec 2016 to the primary completion date, 8 Dec 2030
- 23 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Established diagnosis at the time of referral that is not consistent with a genetic disorder of the white matter, such as an acquired demyelinating condition (e.g. multiple sclerosis), or an infectious etiology, with the exception of sequelae of congenital infections such as CMV;
Other
- Inability to provide consent.
- \- Inability to provide consent.
Who the study is looking for
- Male or female of any age;
- Suspected or confirmed diagnosis of leukodystrophy or other disorder affecting the white matter of the brain based primarily on the finding of central nervous system neuroimaging consistent with this diagnosis or on an existing diagnosis of a leukodystrophy or genetic leukoencephalopathy as defined in existing classification systems, or in the presence of variant(s) of uncertain significance or genotype consistent with leukodytrophy;
- Documentation of informed consent by the subject, parent, or legal guardian, and, if appropriate, documentation of assent;
- Willingness to provide clinical data, participate in standardized assessments, and/or provide biologic samples.
- Male or female of any age;
- Individuals with no confirmed or suspected diagnosis of leukodystrophy or other disorder affecting the white matter of the brain (including affected patients' caregivers);
- Documentation of informed consent by the subject, parent, or legal guardian, and, if appropriate, documentation of assent.
Sex: All · Does not accept healthy volunteers
Sites (23)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Akron Children's Hospital Coordinator of this site? Confirm its status | Akron | United States | Recruiting |
| Emory University, Children's Healthcare of Atlanta Coordinator of this site? Confirm its status | Atlanta | United States | Recruiting |
| Kennedy Krieger Institute Coordinator of this site? Confirm its status | Baltimore | United States | Recruiting |
| Massachusetts General Hospital (MGH) Coordinator of this site? Confirm its status | Boston | United States | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago Coordinator of this site? Confirm its status | Chicago | United States | Recruiting |
| Nationwide Children's Hospital Coordinator of this site? Confirm its status | Columbus | United States | Recruiting |
| Baylor College of Medicine/ Texas Children's Hospital Coordinator of this site? Confirm its status | Houston | United States | Recruiting |
| UT Health Houston Coordinator of this site? Confirm its status | Houston | United States | Recruiting |
| Children's Hospital of Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Recruiting |
| University of Minnesota Coordinator of this site? Confirm its status | Minneapolis | United States | Recruiting |
| Children's Hospital of Orange County Coordinator of this site? Confirm its status | Orange | United States | Recruiting |
| Stanford University (Lucile Packard Children's Hospital) Coordinator of this site? Confirm its status | Palo Alto | United States | Recruiting |
| University of Pennsylvania Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
| The Children's Hospital Of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
| University of Pittsburgh Medical Center Coordinator of this site? Confirm its status | Pittsburgh | United States | Recruiting |
| Mayo Clinic Coordinator of this site? Confirm its status | Rochester | United States | Recruiting |
| University of California, Davis (UC Davis Health) Coordinator of this site? Confirm its status | Sacramento | United States | Recruiting |
| University of Utah, Primary Children's Hospital Coordinator of this site? Confirm its status | Salt Lake City | United States | Recruiting |
| University of California, San Diego Rady Children's Hospital Coordinator of this site? Confirm its status | San Diego | United States | Recruiting |
| UCSF Benioff Children's Hospital Coordinator of this site? Confirm its status | San Francisco | United States | Recruiting |
| Seattle Children's Hospital Coordinator of this site? Confirm its status | Seattle | United States | Recruiting |
| Children's National Medical Center Coordinator of this site? Confirm its status | Washington D.C. | United States | Recruiting |
| Atrium Health Wake Forest Baptist Coordinator of this site? Confirm its status | Winston-Salem | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.