A Phase 1/2 interventional study of AAV9 BBP-812 in Canavan Disease, sponsored by Aspa Therapeutics. Recruiting at 4 sites in United States. Open to participants aged Up to 30 Months. Per ClinicalTrials.gov, last updated 2026-04-17.
Sponsored by Aspa Therapeutics · Phase 1/2, Interventional, and Treatment
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.
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.
592 studies on the registry are indexed under Rare Diseases; 125 are open to participants now.
This study's planned enrollment of 26 is below the median of 45 across 339 interventional studies indexed under Rare Diseases.
Browse Rare Diseases studies →Aspa Therapeutics is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Participant has biochemical, genetic, and clinical diagnosis of Canavan disease:
Key Exclusion Criteria:
Participant has significantly progressed Canavan disease characterized as:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study.
Biological: AAV9 BBP-812
Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study.
Biological: AAV9 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.
Biological: AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump
Number of Participants with Adverse Events (AEs)
Time frame: Baseline up to Week 52
Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels
Time frame: Baseline, Month 12
Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS)
Time frame: Baseline, Month 12
Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88
Time frame: Baseline, Week 52
Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4
Time frame: Baseline, Week 52
Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4
Time frame: Baseline, Week 52
Change from Baseline to Week 52 in Communication Assessment, Bayley-4
Time frame: Baseline, Week 52
Change from Baseline to Week 52 in Adaptive Function, Vineland-3
Time frame: Baseline, Week 52
Not enrolling at this site.
Plan to share: No
Started Sep 2021 (actual). Primary completion is estimated for Oct 2026, 61 months after the start.
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Aspa Therapeutics