NCT03047369 The Myelin Disorders Biorepository Project
| NCT ID | NCT03047369 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Children's Hospital of Philadelphia |
| Condition | Leukodystrophy |
| Study Type | OBSERVATIONAL |
| Enrollment | 12,000 participants |
| Start Date | 2016-12-08 |
| Primary Completion | 2030-12-08 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 12,000 participants in total. It began in 2016-12-08 with a primary completion date of 2030-12-08.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
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.
Contact & Investigator
Adeline Vanderver, MD
PRINCIPAL INVESTIGATOR
Children's Hospital of Philadelphia
Frequently Asked Questions
Who can join the NCT03047369 clinical trial?
This trial is open to participants of all sexes, studying Leukodystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT03047369 currently recruiting?
Yes, NCT03047369 is actively recruiting participants. Contact the research team at sherbinio@chop.edu for enrollment information.
Where is the NCT03047369 trial being conducted?
This trial is being conducted at Los Angeles, United States, Orange, United States, Palo Alto, United States, Sacramento, United States and 11 additional locations.
Who is sponsoring the NCT03047369 clinical trial?
NCT03047369 is sponsored by Children's Hospital of Philadelphia. The principal investigator is Adeline Vanderver, MD at Children's Hospital of Philadelphia. The trial plans to enroll 12,000 participants.