A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Purpose
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.
Condition
- Canavan Disease
Eligibility
- Eligible Ages
- Under 30 Months
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Maximum age for inclusion is 30 months. - Participant has stable health in the opinion of the investigator and as confirmed by medical history and laboratory studies with no acute or chronic hematologic, renal, liver, immunologic, or neurologic disease (other than Canavan disease). - Participant has biochemical, genetic, and clinical diagnosis of Canavan disease: - Elevated urinary NAA and - Biallelic mutation of the ASPA gene determined at Screening or documented in the participant's medical history. - Active clinical signs of Canavan disease - Participant is up to date on all immunizations per local guidelines
Exclusion Criteria
- Tests positive for total anti-AAV9 antibodies determined by enzyme-linked immunosorbent assay (ELISA). - Received prior gene therapy or other therapy (including vaccines) involving AAV. - Participant is receiving high-dose therapy with immunosuppressants. - Participant has significantly progressed Canavan disease characterized as: - Presence of continuous/constant decerebrate or decorticate posturing, - Recurrent status epilepticus, or - Recalcitrant seizures that do not respond while on 3 or more anti-epileptic medications
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1) |
Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study. |
|
Experimental Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2) |
Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study. |
|
Experimental Enrollment Expansion Phase: BBP-812 |
Participants will receive a single IV infusion of BBP-812 at the selected dose from the dose-finding phase on Day 0 in expansion phase of the study. |
|
Recruiting Locations
Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
Boston, Massachusetts 02114
Boston, Massachusetts 02114
More Details
- Status
- Recruiting
- Sponsor
- Aspa Therapeutics
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.