Canavan Disease clinical trials at UCSF

3 research studies open to eligible people

Canavan disease is a rare genetic disorder that affects the brain. UCSF is studying a new gene therapy to see if it is safe for people with this disease. Another study looks at past medical records to understand how the disease progresses. UCSF is also collecting samples to learn more about myelin disorders.

Showing trials for

  • AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

    open to eligible people ages up to 30 months

    The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.

    Oakland, California and other locations

  • Patients With Canavan Disease (CANinform Study)

    open to all eligible people

    This study uses medical records that allow retrospective data extraction of critical milestone and motor function data. In addition, prospective assessments collect data relevant to the natural history of Canavan disease in children.

    Oakland, California and other locations

  • Myelin Disorders Biorepository Project

    open to all eligible people

    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.

    San Francisco, California and other locations

Our lead scientists for Canavan Disease research studies include .

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