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NCT03047369 · ClinicalTrials.gov registry record
The Myelin Disorders Biorepository Project
A clinical trial of Adrenoleukodystrophy and White Matter Disease, sponsored by Children's Hospital of Philadelphia.
- Recruiting
- Registry status
- 12,000
- Enrollment target
- 20
- Study locations
NCT03047369: Recruiting study of Adrenoleukodystrophy and White Matter Disease, sponsored by Children's Hospital of Philadelphia.
NCT03047369 is a study of Adrenoleukodystrophy and White Matter Disease that is actively recruiting participants, run by Children's Hospital of Philadelphia. The registered enrollment target is 12,000 participants, above the 221-participant average among 6 other Adrenoleukodystrophy trials with a reported enrollment target (5330% higher). The trial reports 20 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03047369, a study of Adrenoleukodystrophy and White Matter Disease, is actively recruiting participants, sponsored by Children's Hospital of Philadelphia.
- RECRUITING
- Registry status
- 12,000 participants
- Enrollment target
- 20
- Study locations
Study 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.
Primary Outcome
In patients with an unclassified leukodystrophy, the study team will collect as much information as available from existing medical records including existing clinical evaluations, neuropsychological/rehabilitation evaluations, and results from blood, urine, spinal fluid, radiological, and peripheral tissue pathological tests. This data will be evaluated to create nosologic groups amongst patients with unclassified leukodystrophy. Additionally, this aim includes the collection and long-term bank
Conditions Studied
Study Locations (20)
California
- Children's Hospital of Los Angeles - Los Angeles
- Children's Hospital of Orange County - Orange
- Stanford University (Lucile Packard Children's Hospital) - Palo Alto
- University of California, Davis (UC Davis Health) - Sacramento
- University of California, San Diego (Rady Children's Hospital) - San Diego
- UCSF Benioff Children's Hospital - San Francisco
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
- University of Pennsylvania - Philadelphia
- University of Pittsburgh Medical Center - Pittsburgh
Minnesota
- University of Minnesota - Minneapolis
- Mayo Clinic - Rochester
Ohio
- Akron Children's Hospital - Akron
- Nationwide Children's Hospital - Columbus
District of Columbia
- Children's National Medical Center - Washington D.C.
Georgia
- Emory University (Children's Healthcare of Atlanta) - Atlanta
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Maryland
- Kennedy Krieger Institute - Baltimore
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12,000 participants |
| Start Date | 2016-12-08 |
| Est. Completion | 2030-12-08 |
What NCT03047369 shows while recruiting
NCT03047369 is an observational study that tracks outcomes without assigning an intervention. Its 12,000 participants enrollment target places it among the larger protocols in the corpus, above the 221-participant average among 6 other Adrenoleukodystrophy trials with a reported enrollment target (5330% higher).
The record links to 10 conditions, with Adrenoleukodystrophy appearing as the primary indexed condition, and to 0 interventions.
NCT03047369 names 20 study sites across 11 states, led by California, Pennsylvania, Minnesota.
Frequently Asked Questions
What is clinical trial NCT03047369 about?
NCT03047369 is a clinical study titled "The Myelin Disorders Biorepository Project". 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,...
What is the current status of trial NCT03047369?
This trial is currently recruiting. The enrollment target is 12,000 participants. The study started on 2016-12-08. Estimated completion is 2030-12-08.
What conditions does trial NCT03047369 study?
This clinical trial studies the following conditions: Adrenoleukodystrophy, White Matter Disease, Leukodystrophy, ALD (Adrenoleukodystrophy), Leukoencephalopathies.
Who is sponsoring clinical trial NCT03047369?
This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03047369 being conducted?
This trial has 20 study locations across California, District of Columbia, Georgia, Illinois, Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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