Collects samples and data from people with leukodystrophy worldwide
Gathers clinical data and biological samples from people with leukodystrophy for future research.
Plain-language title generated from the registry record
Registry title: The Myelin Disorders Biorepository Project
Key facts
- Where
- Akron, Atlanta, Baltimore, Boston, Chicago, Columbus, Houston, Los Angeles, Minneapolis, Orange, Palo Alto, Philadelphia, Pittsburgh, Rochester, Sacramento, Salt Lake City, San Diego, San Francisco, Seattle, Washington D.C., and Winston-Salem, United StatesSee the 23 sites
Where in United States
- Akron Children's HospitalAkron, United StatesTaking people
- Emory University, Children's Healthcare of AtlantaAtlanta, United StatesTaking people
- Kennedy Krieger InstituteBaltimore, United StatesTaking people
- Massachusetts General Hospital (MGH)Boston, United StatesTaking people
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, United StatesTaking people
- Nationwide Children's HospitalColumbus, United StatesTaking people
- Baylor College of Medicine/ Texas Children's HospitalHouston, United StatesTaking people
- UT Health HoustonHouston, United StatesTaking people
- Children's Hospital of Los AngelesLos Angeles, United StatesTaking people
- University of MinnesotaMinneapolis, United StatesTaking people
- Children's Hospital of Orange CountyOrange, United StatesTaking people
- Stanford University (Lucile Packard Children's Hospital)Palo Alto, United StatesTaking people
- The Children's Hospital Of PhiladelphiaPhiladelphia, United StatesTaking people
- University of PennsylvaniaPhiladelphia, United StatesTaking people
- University of Pittsburgh Medical CenterPittsburgh, United StatesTaking people
- Mayo ClinicRochester, United StatesTaking people
- University of California, Davis (UC Davis Health)Sacramento, United StatesTaking people
- University of Utah, Primary Children's HospitalSalt Lake City, United StatesTaking people
- University of California, San Diego Rady Children's HospitalSan Diego, United StatesTaking people
- UCSF Benioff Children's HospitalSan Francisco, United StatesTaking people
- Seattle Children's HospitalSeattle, United StatesTaking people
- Children's National Medical CenterWashington D.C., United StatesTaking people
- Atrium Health Wake Forest BaptistWinston-Salem, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Children's Hospital of Philadelphia
- Trial code
- NCT03047369
- Record updated
- 23 October 2025
- Original description
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.Read full original descriptionShow less
The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
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.
Inclusion: 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.
Exclusion: 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; Inability to provide consent; Inability to provide consent.