Actively Recruiting
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Led by Aspa Therapeutics · Updated on 2026-04-17
26
Participants Needed
4
Research Sites
578 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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...
- Participant is younger than 30 months old
- Participant has a confirmed biochemical, genetic, and clinical diagnosis of Canavan disease
- Elevated urinary NAA levels
- Biallelic mutation of the ASPA gene confirmed at screening or documented
- Participant shows active clinical signs of Canavan disease
- Participant is up to date with all immunizations according to local guidelines
- Participant has stable health with no acute or chronic blood, kidney, liver, immune, or neurological diseases other than Canavan disease
You will not qualify if you...
- Participant tests positive for total anti-AAV9 antibodies by ELISA
- Participant has received prior gene therapy or other AAV-based therapy, including vaccines
- Participant is receiving high-dose immunosuppressant therapy
- Participant has significantly progressed Canavan disease with continuous decerebrate or decorticate posturing
- Participant experiences recurrent status epilepticus
- Participant has seizures that do not respond to treatment with three or more anti-epileptic medications
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
C
clinicaltrials@aspatx.com
CONTACT
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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