A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Public ClinicalTrials.gov record NCT04998396. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official 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
Study identification
- NCT ID
- NCT04998396
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Aspa Therapeutics
- Industry
- Enrollment
- 26 participants
Conditions and interventions
Conditions
Interventions
- AAV9 BBP-812 Biological
Biological
Eligibility (public fields only)
- Age range
- Up to 30 Months
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 7, 2021
- Primary completion
- Oct 12, 2026
- Completion
- Oct 7, 2032
- Last update posted
- Apr 16, 2026
2021 – 2032
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCSF Benioff Children's Hospital Oakland | Oakland | California | 94609 | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | Recruiting |
| Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) | Boston | Massachusetts | 02114 | Recruiting |
| Weill Cornell Medicine; Division of Pediatric Neurology | New York | New York | 10065 | Completed |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04998396, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 16, 2026 · Synced Jul 23, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04998396 live on ClinicalTrials.gov.