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
Study identification
- NCT ID
- NCT03047369
- Recruitment status
- Recruiting
- Study type
- Observational
- Phase
- Not listed
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Enrollment
- 12,000 participants
Conditions and interventions
Conditions
- 4H Syndrome
- ADLD
- AGS
- ALD
- ALD (Adrenoleukodystrophy)
- ALD Gene Mutation
- ALSP
- AMN
- Adrenoleukodystrophy
- Adrenomyeloneuropathy
- Aicardi Goutieres Syndrome
- Alexander Disease
- Alexanders Leukodystrophy
- Allan-Herndon-Dudley Syndrome
- AxD
- BPAN
- CMT
- CSF1R Gene Mutation
- CTX
- Cadasil
- Canavan Disease
- Cerebrotendinous Xanthomatoses
- Charcot-Marie-Tooth
- Cockayne Syndrome
- GALC Deficiency
- GM2 Gangliosidosis
- Gangliosidoses
- Globoid Leukodystrophy
- H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum
- HBSL
- HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity
- HCC - Hypomyelination and Congenital Cataract
- Krabbe Disease
- LBSL
- LCC
- Labrune Syndrome
- Leukodystrophy
- Leukoencephalopathies
- Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)
- Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation
- MLC1
- MLD
- Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency
- Megalencephalic Leukoencephalopathy With Subcortical Cysts
- Metachromatic Leukodystrophy
- Mucopolysaccharidoses
- Multiple Sulfatase Deficiency
- PLP1 Gene Duplication | Blood or Tissue | Mutations
- PLP1 Null Syndrome
- PMD
- Pelizaeus Merzbacher Like Disease
- Pelizaeus-Merzbacher Disease
- Peroxisomal Biogenesis Disorder
- Refsum Disease
- Salla Disease
- Sialic Storage Disease
- Sjogren-Larsson Syndrome
- Sjögren
- TBCK-Related Intellectual Disability Syndrome
- TUBB4A-Related Leukodystrophy
- Van Der Knapp Disease
- Vanishing White Matter Disease
- White Matter Disease
- X-ALD
- X-linked Adrenoleukodystrophy
- Zellweger 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 Apr 29, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03047369 live on ClinicalTrials.gov.