The Myelin Disorders Biorepository Project
Public ClinicalTrials.gov record NCT03047369. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03047369
- Recruitment status
- Recruiting
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 12,000 participants
Conditions and interventions
Conditions
- Leukodystrophy
- White Matter Disease
- Leukoencephalopathies
- 4H Syndrome
- Adrenoleukodystrophy
- AMN
- ALD
- ALD Gene Mutation
- ALD (Adrenoleukodystrophy)
- X-linked Adrenoleukodystrophy
- X-ALD
- Adrenomyeloneuropathy
- Aicardi Goutieres Syndrome
- AGS
- Alexander Disease
- Alexanders Leukodystrophy
- AxD
- ADLD
- Canavan Disease
- CTX
- Cerebrotendinous Xanthomatoses
- Krabbe Disease
- GALC Deficiency
- Globoid Leukodystrophy
- TUBB4A-Related Leukodystrophy
- H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum
- HBSL
- HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity
- LBSL
- Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)
- Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation
- ALSP
- CSF1R Gene Mutation
- HCC - Hypomyelination and Congenital Cataract
- MLC1
- Megalencephalic Leukoencephalopathy With Subcortical Cysts
- MLD
- Metachromatic Leukodystrophy
- PMD
- Pelizaeus-Merzbacher Disease
- PLP1 Null Syndrome
- PLP1 Gene Duplication | Blood or Tissue | Mutations
- Pelizaeus Merzbacher Like Disease
- Peroxisomal Biogenesis Disorder
- Zellweger Syndrome
- Refsum Disease
- Salla Disease
- Sialic Storage Disease
- Sjögren
- Sjogren-Larsson Syndrome
- Van Der Knapp Disease
- Vanishing White Matter Disease
- Charcot-Marie-Tooth
- CMT
- Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency
- Allan-Herndon-Dudley Syndrome
- Cadasil
- Cockayne Syndrome
- Multiple Sulfatase Deficiency
- Gangliosidoses
- GM2 Gangliosidosis
- BPAN
- Labrune Syndrome
- LCC
- Mucopolysaccharidoses
- TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 7, 2016
- Primary completion
- Dec 7, 2030
- Completion
- Dec 7, 2030
- Last update posted
- Oct 22, 2025
2016 – 2030
United States locations
- U.S. sites
- 23
- U.S. states
- 13
- U.S. cities
- 21
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | Recruiting |
| Children's Hospital of Orange County | Orange | California | 92868 | Recruiting |
| Stanford University (Lucile Packard Children's Hospital) | Palo Alto | California | 94304 | Recruiting |
| University of California, Davis (UC Davis Health) | Sacramento | California | 95817 | Recruiting |
| University of California, San Diego (Rady Children's Hospital) | San Diego | California | 92123 | Recruiting |
| UCSF Benioff Children's Hospital | San Francisco | California | 94158 | Recruiting |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | Recruiting |
| Emory University (Children's Healthcare of Atlanta) | Atlanta | Georgia | 30342 | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | Recruiting |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | Recruiting |
| Massachusetts General Hospital (MGH) | Boston | Massachusetts | 02114 | Recruiting |
| University of Minnesota | Minneapolis | Minnesota | 55454 | Recruiting |
| Mayo Clinic | Rochester | Minnesota | 55905 | Recruiting |
| Atrium Health Wake Forest Baptist | Winston-Salem | North Carolina | 27157 | Recruiting |
| Akron Children's Hospital | Akron | Ohio | 44308 | Recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | Recruiting |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Recruiting |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | Recruiting |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15219 | Recruiting |
| Baylor College of Medicine (Texas Children's Hospital) | Houston | Texas | 77030 | Recruiting |
| UT Health Houston | Houston | Texas | — | Recruiting |
| University of Utah (Primary Children's Hospital) | Salt Lake City | Utah | 84112 | Recruiting |
| Seattle Children's Hospital | Seattle | Washington | 98105 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03047369, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 22, 2025 · Synced Sep 2, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03047369 live on ClinicalTrials.gov.