Phase
Condition
N/ATreatment
AAV9 BBP-812
Clinical Study ID
Ages < 30 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Maximum age for inclusion is 30 months.
Participant has stable health in the opinion of the investigator and as confirmed bymedical 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 inthe participant's medical history.
Active clinical signs of Canavan disease
Participant is up to date on all immunizations per local guidelines
Exclusion
Key Exclusion Criteria:
Tests positive for total anti-AAV9 antibodies determined by enzyme-linkedimmunosorbent 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-epilepticmedications
Study Design
Study Description
Connect with a study center
UCSF Benioff Children's Hospital Oakland
Oakland, California 94609
United StatesActive - Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United StatesActive - Recruiting
Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
Boston, Massachusetts 02114
United StatesActive - Recruiting
Weill Cornell Medicine; Division of Pediatric Neurology
New York, New York 10065
United StatesCompleted
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