NCT04998396

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

A Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA Gene

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 26 (estimated)
• Sponsor: Aspa Therapeutics · Industry
• Sex: All
• Age: 30 Months
• Healthy volunteers: Not accepted

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.

Detailed description

Canavan disease is an ultra-rare, profoundly disabling and fatal disease with no approved therapy. The Sponsor is developing BBP-812, an investigational gene therapy product for systemic delivery in participants with Canavan disease. BBP-812 is a recombinant adeno-associated virus serotype 9 (rAAV9) vector engineered to deliver the aspartoacylase (ASPA) transgene under control of a ubiquitous promoter to restore ASPA expression in both neuronal and non-neuronal cell types.

Conditions

• Canavan Disease

Interventions

Type	Name	Description
BIOLOGICAL	AAV9 BBP-812	Sterile solution for injection for 1-time use via volumetric infusion pump

Timeline

• Start date: 2021-09-08
• Primary completion: 2026-10-13
• Completion: 2032-10-08
• First posted: 2021-08-10
• Last updated: 2026-04-17

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study