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The Myelin Disorders Biorepository Project

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
No age limits stated
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this 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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Official 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.

Study sites (23)

CA

  • Children's Hospital of Los Angeles · Los Angeles, CA 90027Jonathan Santoro, MD · Study contactMore trials in Los Angeles
  • Children's Hospital of Orange County · Orange, CA 92868Changrui Xiao, MD · Study contactMore trials in Orange
  • Stanford University (Lucile Packard Children's Hospital) · Palo Alto, CA 94304Keith Van Haren, MD · Study contactMore trials in Palo Alto
  • University of California, Davis (UC Davis Health) · Sacramento, CA 95817William Benko, MD · Study contactMore trials in Sacramento
  • University of California, San Diego (Rady Children's Hospital) · San Diego, CA 92123Jennifer Yang, MD · Study contactMore trials in San Diego
  • UCSF Benioff Children's Hospital · San Francisco, CA 94158Alexander Fay, MD, PhD · Study contactMore trials in San Francisco

DC

GA

  • Emory University (Children's Healthcare of Atlanta) · Atlanta, GA 30342Stephanie Keller, MD · Study contactMore trials in Atlanta

IL

  • Ann & Robert H. Lurie Children's Hospital of Chicago · Chicago, IL 60611Jennifer P Rubin, MD · Study contactMore trials in Chicago

MA

  • Massachusetts General Hospital (MGH) · Boston, MA 02114Florian Eichler, MD · Study contactMore trials in Boston

MD

MN

NC

OH

PA

  • The Children's Hospital of Philadelphia · Philadelphia, PA 19104Omar Sherbini, MPH · Study contact · 215-590-3068 · sherbinio@chop.eduAdeline Vanderver, MD · Principal investigatorLaura Adang, MD, PhD, MSTR · Sub-investigatorAmy Waldman, MD, MSCE · Sub-investigatorMore trials in Philadelphia
  • University of Pennsylvania · Philadelphia, PA 19104Jennifer Orthmann-Murphy, MD, PhD · Study contactMore trials in Philadelphia
  • University of Pittsburgh Medical Center · Pittsburgh, PA 15219Deepa Rajan, MD · Study contactMore trials in Pittsburgh

TX

  • Baylor College of Medicine (Texas Children's Hospital) · Houston, TX 77030Lisa Emrick, MD · Study contactMore trials in Houston
  • UT Health Houston · Houston, TX Mary Kay Koenig, MD · Study contactMore trials in Houston

UT

  • University of Utah (Primary Children's Hospital) · Salt Lake City, UT 84112Joshua Bonkowsky, MD, PhD · Study contactMore trials in Salt Lake City

WA