🔎 Trials Near Me

Home › Leukoencephalopathies › NCT03047369

The Myelin Disorders Biorepository Project

RecruitingObservational study

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

Who can join

All ages · All sexes

Full eligibility criteria
Inclusion Criteria (Affected Subjects):

* 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.

Exclusion Criteria (Affected Subjects)

* 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.

Inclusion Criteria (Healthy Controls)

* 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 Criteria (Healthy Controls)

\- Inability to provide consent.

About the study

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.

Sponsor: Children's Hospital of Philadelphia · Participants: 12,000 · Started: Dec 8, 2016

Contact the study team

Official record on ClinicalTrials.gov — NCT03047369

Locations in the U.S.

CaliforniaChildren's Hospital of Los Angeles, Los Angeles
Children's Hospital of Orange County, Orange
Stanford University (Lucile Packard Children's Hospital), Palo Alto
University of California, Davis (UC Davis Health), Sacramento
University of California, San Diego (Rady Children's Hospital), San Diego
UCSF Benioff Children's Hospital, San Francisco
District of ColumbiaChildren's National Medical Center, Washington D.C.
GeorgiaEmory University (Children's Healthcare of Atlanta), Atlanta
IllinoisAnn & Robert H. Lurie Children's Hospital of Chicago, Chicago
MarylandKennedy Krieger Institute, Baltimore
MassachusettsMassachusetts General Hospital (MGH), Boston
MinnesotaUniversity of Minnesota, Minneapolis
Mayo Clinic, Rochester
North CarolinaAtrium Health Wake Forest Baptist, Winston-Salem
OhioAkron Children's Hospital, Akron
Nationwide Children's Hospital, Columbus
PennsylvaniaThe Children's Hospital of Philadelphia, Philadelphia
University of Pennsylvania, Philadelphia
University of Pittsburgh Medical Center, Pittsburgh
TexasBaylor College of Medicine (Texas Children's Hospital), Houston
UT Health Houston, Houston
UtahUniversity of Utah (Primary Children's Hospital), Salt Lake City
WashingtonSeattle Children's Hospital, Seattle

Conditions

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.