Phase 12 Study of Single-Dose AAV9 BBP-812 Gene Therapy for Children with Canavan Disease up to 30 Months Old

What this Trial Is About

Researchers are studying BBP-812, an investigational gene therapy using an AAV9-based vector, to evaluate its safety, tolerability, and pharmacodynamic effects in children with Canavan disease. Canavan disease is a very rare, severe, and fatal disorder with no approved treatments. BBP-812 is designed to deliver a gene called ASPA to restore its expression in brain and other cells. Participants will receive a single intravenous infusion of BBP-812. The study includes a dose-finding phase where participants receive either a low or high dose of BBP-812 on Day 0, followed by an expansion phase where participants receive the selected dose from the earlier phase. This gene therapy is given only once during the study. During the trial, participants will be closely monitored through clinical evaluations, urine and brain imaging tests measuring N-acetylaspartate levels, and assessments of motor, cognitive, communication, and adaptive functions up to one year after infusion. Safety is tracked by recording any adverse events. The total study duration includes baseline assessments and follow-ups up to 12 months post-treatment.

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)