Canavan Disease clinical trials at UCSF
3 research studies open to eligible people
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 Alexander Fay, MD.
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