Canavan Disease clinical trials at UCSF

2 in progress, 1 open to eligible people

Canavan disease is a rare brain disorder that affects children. UCSF is running trials to study a new gene therapy called BBP-812 to check if it is safe and how it works. These trials focus on finding better treatments for kids with Canavan disease.

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)

    Sorry, not currently recruiting here

    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

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