RecruitingNCT03047369

The Myelin Disorders Biorepository Project

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

Sponsor

Children's Hospital of Philadelphia

Enrollment

12,000 participants

Start Date

Dec 8, 2016

Study Type

OBSERVATIONAL

Conditions

Adrenoleukodystrophy Cockayne Syndrome Refsum Disease Leukodystrophy Labrune Syndrome Allan-Herndon-Dudley Syndrome Cadasil Sjögren White Matter Disease Leukoencephalopathies 4H Syndrome 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 &#X7C; Blood or Tissue &#X7C; Mutations Pelizaeus Merzbacher Like Disease Peroxisomal Biogenesis Disorder Zellweger Syndrome Salla Disease Sialic Storage Disease Sjogren-Larsson Syndrome Van Der Knapp Disease Vanishing White Matter Disease Charcot-Marie-Tooth CMT Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency Multiple Sulfatase Deficiency Gangliosidoses GM2 Gangliosidosis BPAN LCC Mucopolysaccharidoses TBCK-Related Intellectual Disability Syndrome

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

Plain Language Summary

Simplified for easier understanding

This project collects medical information, DNA, and biological samples from patients with leukodystrophies and other rare white matter brain diseases — disorders that damage the protective coating (myelin) around nerve fibers — to help researchers find better diagnoses and treatments. **You may be eligible if...** - You (or your child) have a confirmed or suspected diagnosis of a leukodystrophy or genetic white matter brain disease, based on brain imaging or genetic testing - You are willing to provide clinical information, complete assessments, and/or donate biological samples - Family members without the disease can also participate as healthy controls **You may NOT be eligible if...** - Your condition has been clearly diagnosed as an acquired (non-genetic) white matter disease such as multiple sclerosis or an infection-caused condition (with some exceptions) - You are unable to provide consent Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(23)

Children's Hospital of Los Angeles

Los Angeles, California, United States

Children's Hospital of Orange County

Orange, California, United States

Stanford University (Lucile Packard Children's Hospital)

Palo Alto, California, United States

University of California, Davis (UC Davis Health)

Sacramento, California, United States

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

San Diego, California, United States

UCSF Benioff Children's Hospital

San Francisco, California, United States

Children's National Medical Center

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

Emory University (Children's Healthcare of Atlanta)

Atlanta, Georgia, United States

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

Chicago, Illinois, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

Massachusetts General Hospital (MGH)

Boston, Massachusetts, United States

University of Minnesota

Minneapolis, Minnesota, United States

Mayo Clinic

Rochester, Minnesota, United States

Atrium Health Wake Forest Baptist

Winston-Salem, North Carolina, United States

Akron Children's Hospital

Akron, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Baylor College of Medicine (Texas Children's Hospital)

Houston, Texas, United States

UT Health Houston

Houston, Texas, United States

University of Utah (Primary Children's Hospital)

Salt Lake City, Utah, United States

Seattle Children's Hospital

Seattle, Washington, United States

View Full Details on ClinicalTrials.gov

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NCT03047369

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