NCT04998396 A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
| NCT ID | NCT04998396 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Aspa Therapeutics |
| Condition | Canavan Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 26 participants |
| Start Date | 2021-09-08 |
| Primary Completion | 2026-10-13 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 26 participants in total. It began in 2021-09-08 with a primary completion date of 2026-10-13.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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 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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT04998396 clinical trial?
This trial is open to participants of all sexes, up to 30 Months, studying Canavan Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04998396 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT04998396 currently recruiting?
Yes, NCT04998396 is actively recruiting participants. Contact the research team at CANaspire@aspatx.com for enrollment information.
Where is the NCT04998396 trial being conducted?
This trial is being conducted at Oakland, United States, Chicago, United States, Boston, United States, New York, United States.
Who is sponsoring the NCT04998396 clinical trial?
NCT04998396 is sponsored by Aspa Therapeutics. The trial plans to enroll 26 participants.