Canavan Disease Clinical Trial
— CANaspireOfficial title:
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
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.
Status | Recruiting |
Enrollment | 26 |
Est. completion date | October 8, 2030 |
Est. primary completion date | October 13, 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 30 Months |
Eligibility | Key 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 Key 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 |
Country | Name | City | State |
---|---|---|---|
United States | Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) | Boston | Massachusetts |
United States | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois |
United States | Weill Cornell Medicine; Division of Pediatric Neurology | New York | New York |
United States | UCSF Benioff Children's Hospital Oakland | Oakland | California |
Lead Sponsor | Collaborator |
---|---|
Aspa Therapeutics |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Participants with Adverse Events (AEs) | Baseline up to Week 52 | ||
Primary | Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels | Baseline, Month 12 | ||
Primary | Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS) | Baseline, Month 12 | ||
Secondary | Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88 | Baseline, Week 52 | ||
Secondary | Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4 | Baseline, Week 52 | ||
Secondary | Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4 | Baseline, Week 52 | ||
Secondary | Change from Baseline to Week 52 in Communication Assessment, Bayley-4 | Baseline, Week 52 | ||
Secondary | Change from Baseline to Week 52 in Adaptive Function, Vineland-3 | Baseline, Week 52 |
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