Clinical trialCLINICALTRIALSApr 17
Trial Now Recruiting: A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) (NCT04998396)
Researchers are testing a new gene therapy called BBP-812 to treat Canavan disease, a rare brain disorder that affects children. The therapy uses a modified virus to deliver a healthy copy of a gene that's missing or broken in people with this disease. This early-stage trial will check if the treatment is safe and whether it helps patients.
WHY IT MATTERSThis trial is now actively recruiting children with Canavan disease — if your child has been diagnosed, you may be eligible to participate in one of the first human tests of this gene therapy approach.