Actively Recruiting
A Phase 1/2 Open-Label Study of Gene Therapy Using AAV9 Vector Encoding the Human ASPA Gene for Canavan Disease
Led by Aspa Therapeutics · Updated on 2026-04-17
26
Participants Needed
4
Research Sites
312 weeks
Total Duration
On this page
AI-Summary
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.
CONDITIONS
Brief Title
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Maximum age of 30 months at enrollment
- Stable health with no acute or chronic hematologic, kidney, liver, immune, or neurologic diseases other than Canavan disease
- Biochemical, genetic, and clinical diagnosis of Canavan disease including elevated urinary NAA and biallelic ASPA gene mutation
- Presence of active clinical signs of Canavan disease
- Up to date on all immunizations per local guidelines
You will not qualify if you...
- Positive test for anti-AAV9 antibodies
- Previous treatment with gene therapy or therapies involving AAV
- Currently receiving high-dose immunosuppressant therapy
- Advanced Canavan disease with continuous decerebrate or decorticate posturing
- Recurrent status epilepticus or seizures not controlled by three or more anti-epileptic medications
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 12 months
Participants receive a single intravenous infusion of the gene therapy BBP-812 on Day 0.
1 treatment visit and multiple follow-up visits up to 12 months
Trial Site Locations
Total: 4 locations
1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Actively Recruiting
2
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
3
Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
Boston, Massachusetts, United States, 02114
Actively Recruiting
4
Weill Cornell Medicine; Division of Pediatric Neurology
New York, New York, United States, 10065
Completed
Research Team
A
Alicia Gomez
C
clinicaltrials@aspatx.com
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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