The Myelin Disorders Biorepository Project

Recruiting

Trial ID: NCT03047369

Purpose

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.

Official Title

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

Stanford Investigator(s)

Eligibility


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;

   - 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;

   - 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.

Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Keith Van Haren, MD