Trial Now Recruiting: A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) (NCT04998396)
WHY IT MATTERS
This 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.
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.
NCT ID: NCT04998396 Status: RECRUITING Conditions: Canavan Disease Phase: PHASE1, PHASE2 Enrollment: 26 Sponsor: Aspa Therapeutics Summary: 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.
YOU CAN ACT ON THIS
Contact Aspa Therapeutics or your child's neurologist immediately to ask about eligibility for the CANaspire trial (NCT04998396), as recruitment is currently open.