Actively Recruiting

All Genders
NCT03047369

The Myelin Disorders Biorepository Project

Led by Children's Hospital of Philadelphia · Updated on 2025-10-23

12000

Participants Needed

23

Research Sites

730 weeks

Total Duration

On this page

Sponsors

C

Children's Hospital of Philadelphia

Lead Sponsor

N

National Institutes of Health (NIH)

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

The Myelin Disorders Biorepository Project

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Male or female of any age
  • Suspected or confirmed diagnosis of leukodystrophy or other white matter brain disorder based on neuroimaging or existing diagnosis
  • Documentation of informed consent by the subject, parent, or legal guardian, with assent if appropriate
  • Willingness to provide clinical data, participate in assessments, and/or provide biological samples
  • For healthy controls: no confirmed or suspected leukodystrophy or white matter disorder, including caregivers
  • Documentation of informed consent by the subject, parent, or legal guardian, with assent if appropriate
Not Eligible

You will not qualify if you...

  • Established diagnosis at referral that is not a genetic white matter disorder, such as acquired demyelinating diseases (e.g., multiple sclerosis) or infectious causes except congenital infections like CMV
  • Inability to provide consent

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 23 locations

1

Children's Hospital of Los Angeles

Los Angeles, California, United States, 90027

Actively Recruiting

2

Children's Hospital of Orange County

Orange, California, United States, 92868

Actively Recruiting

3

Stanford University (Lucile Packard Children's Hospital)

Palo Alto, California, United States, 94304

Actively Recruiting

4

University of California, Davis (UC Davis Health)

Sacramento, California, United States, 95817

Actively Recruiting

5

University of California, San Diego (Rady Children's Hospital)

San Diego, California, United States, 92123

Actively Recruiting

6

UCSF Benioff Children's Hospital

San Francisco, California, United States, 94158

Actively Recruiting

7

Children's National Medical Center

Washington D.C., District of Columbia, United States, 20010

Actively Recruiting

8

Emory University (Children's Healthcare of Atlanta)

Atlanta, Georgia, United States, 30342

Actively Recruiting

9

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States, 60611

Actively Recruiting

10

Kennedy Krieger Institute

Baltimore, Maryland, United States, 21205

Actively Recruiting

11

Massachusetts General Hospital (MGH)

Boston, Massachusetts, United States, 02114

Actively Recruiting

12

University of Minnesota

Minneapolis, Minnesota, United States, 55454

Actively Recruiting

13

Mayo Clinic

Rochester, Minnesota, United States, 55905

Actively Recruiting

14

Atrium Health Wake Forest Baptist

Winston-Salem, North Carolina, United States, 27157

Actively Recruiting

15

Akron Children's Hospital

Akron, Ohio, United States, 44308

Actively Recruiting

16

Nationwide Children's Hospital

Columbus, Ohio, United States, 43205

Actively Recruiting

17

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

18

University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

19

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States, 15219

Actively Recruiting

20

Baylor College of Medicine (Texas Children's Hospital)

Houston, Texas, United States, 77030

Actively Recruiting

21

UT Health Houston

Houston, Texas, United States

Actively Recruiting

22

University of Utah (Primary Children's Hospital)

Salt Lake City, Utah, United States, 84112

Actively Recruiting

23

Seattle Children's Hospital

Seattle, Washington, United States, 98105

Actively Recruiting

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Research Team

O

Omar S. Sherbini, MPH

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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