Massive biorepository aims to unlock secrets of rare brain diseases

NCT ID NCT03047369

First seen Nov 01, 2025 · Last updated May 01, 2026 · Updated 26 times

Summary

This study is creating a large collection of medical information and biological samples from people with leukodystrophy, a group of rare brain disorders. Researchers will use this data to find new genetic causes, develop better tests, and understand how these diseases progress. The goal is to improve diagnosis and pave the way for future treatments. Anyone with a suspected or confirmed leukodystrophy can join.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Akron Children's Hospital

    RECRUITING

    Akron, Ohio, 44308, United States

    Contact

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

    RECRUITING

    Chicago, Illinois, 60611, United States

    Contact

  • Atrium Health Wake Forest Baptist

    RECRUITING

    Winston-Salem, North Carolina, 27157, United States

    Contact

  • Baylor College of Medicine (Texas Children's Hospital)

    RECRUITING

    Houston, Texas, 77030, United States

    Contact

  • Children's Hospital of Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

    Contact

  • Children's Hospital of Orange County

    RECRUITING

    Orange, California, 92868, United States

    Contact

  • Children's National Medical Center

    RECRUITING

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

    Contact

  • Emory University (Children's Healthcare of Atlanta)

    RECRUITING

    Atlanta, Georgia, 30342, United States

    Contact

  • Kennedy Krieger Institute

    RECRUITING

    Baltimore, Maryland, 21205, United States

    Contact

  • Massachusetts General Hospital (MGH)

    RECRUITING

    Boston, Massachusetts, 02114, United States

    Contact

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

    Contact

  • Nationwide Children's Hospital

    RECRUITING

    Columbus, Ohio, 43205, United States

    Contact

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

    Contact

  • Stanford University (Lucile Packard Children's Hospital)

    RECRUITING

    Palo Alto, California, 94304, United States

    Contact

  • The Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact

    Contact

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • UCSF Benioff Children's Hospital

    RECRUITING

    San Francisco, California, 94158, United States

    Contact

  • UT Health Houston

    RECRUITING

    Houston, Texas, United States

    Contact

  • University of California, Davis (UC Davis Health)

    RECRUITING

    Sacramento, California, 95817, United States

    Contact

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

    RECRUITING

    San Diego, California, 92123, United States

    Contact

  • University of Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55454, United States

    Contact

  • University of Pennsylvania

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact

  • University of Pittsburgh Medical Center

    RECRUITING

    Pittsburgh, Pennsylvania, 15219, United States

    Contact

  • University of Utah (Primary Children's Hospital)

    RECRUITING

    Salt Lake City, Utah, 84112, United States

    Contact

Conditions

Explore the condition pages connected to this study.

4H SYNDROME ADLD ADRENOLEUKODYSTROPHY ADRENOMYELONEUROPATHY AGS AICARDI GOUTIERES SYNDROME AICARDI GOUTIERES SYNDROME ALD ALD (ADRENOLEUKODYSTROPHY) ALD GENE MUTATION ALEXANDER DISEASE ALEXANDERS LEUKODYSTROPHY ALLAN-HERNDON-DUDLEY SYNDROME ALLAN-HERNDON-DUDLEY SYNDROME ALSP ALSP AMN AXD BPAN CADASIL CADASIL CANAVAN DISEASE CEREBROTENDINOUS XANTHOMATOSES CHARCOT-MARIE-TOOTH CMT COCKAYNE SYNDROME CSF1R GENE MUTATION CSF1R GENE MUTATION CSF1R GENE MUTATION CTX GALC DEFICIENCY GANGLIOSIDOSES GLOBOID LEUKODYSTROPHY GM2 GANGLIOSIDOSIS H-ABC - HYPOMYELINATION, ATROPHY OF BASAL GANGLIA AND CEREBELLUM 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 LABRUNE SYNDROME LBSL LCC 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 MCT8 (SLC16A2)-SPECIFIC THYROID HORMONE CELL TRANSPORTER DEFICIENCY MEGALENCEPHALIC LEUKOENCEPHALOPATHY WITH SUBCORTICAL CYSTS METACHROMATIC LEUKODYSTROPHY MLC1 MLD MUCOPOLYSACCHARIDOSES MULTIPLE SULFATASE DEFICIENCY PELIZAEUS MERZBACHER LIKE DISEASE PELIZAEUS-MERZBACHER DISEASE PEROXISOMAL BIOGENESIS DISORDER PLP1 GENE DUPLICATION | BLOOD OR TISSUE | MUTATIONS PLP1 NULL SYNDROME PMD REFSUM DISEASE SALLA DISEASE SIALIC STORAGE DISEASE SJOGREN-LARSSON SYNDROME SJÖGREN TBCK-RELATED INTELLECTUAL DISABILITY SYNDROME TBCK-RELATED INTELLECTUAL DISABILITY SYNDROME TUBB4A-RELATED LEUKODYSTROPHY VAN DER KNAPP DISEASE VAN DER KNAPP DISEASE VANISHING WHITE MATTER DISEASE WHITE MATTER DISEASE X-ALD X-LINKED ADRENOLEUKODYSTROPHY ZELLWEGER SYNDROME