A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Recruiting
Phase 1
Phase 2
Interventional Study
Canavan Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- Up to 30 Months
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 26 (estimated)
- Sponsor
- Aspa Therapeutics · Industry
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About This Trial
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.
Trial Locations
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Eligibility Criteria
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 …
Contacts
clinicaltrials@aspatx.com
CONTACT