Clinical Trials in the USA / NCT04998396
Recruiting
Phase 1/2
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
NCT04998396 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Canavan Disease
Investigational drug(s) / intervention(s)
AAV9 BBP-812
AAV9 BBP-812: Sterile solution for injection for 1-time use via volumetric infusion pump
Study summary
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.
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
* Participant is up to date on all immunizations per local guidelines
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
Primary outcome measure(s)
- Number of Participants with Adverse Events (AEs) — Baseline up to Week 52
- Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels — Baseline, Month 12
- Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS) — Baseline, Month 12
Trial sites (4)
| Facility | City | Region | Status |
|---|---|---|---|
| UCSF Benioff Children's Hospital Oakland | Oakland | California | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | Recruiting |
| Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) | Boston | Massachusetts | Recruiting |
| Weill Cornell Medicine; Division of Pediatric Neurology | New York | New York | Completed |
Other trials for the same condition
rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease
Enrolling by invitation
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04998396 on ClinicalTrials.gov ↗ ← All trials in the USA