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CANaspire Clinical Trial: AAV9 Gene Therapy for Canavan Disease

This study is testing an investigational gene therapy called BBP-812 for children with Canavan disease. Canavan disease is a very rare and serious condition with no approved treatments. BBP-812 is designed to deliver a specific gene (ASPA) to help the body make a missing protein. Researchers want to see how safe BBP-812 is and if it can change levels of a substance called N-acetylaspartate (NAA) in urine and the brain, which are important markers for Canavan disease. This trial is open to children up to 30 months old who are otherwise in stable health. The study plans to enroll 26 participants, but the current recruitment status is unclear.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It is planned to enroll 26 participants, but the phase is not specified.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety up to 52 weeks and for changes in NAA levels for 12 months after receiving the infusion.

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NCT04998396

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

Recruiting
PHASE1Up to 30InterventionalTreatment
Aspa Therapeutics
~26 participants
Updated 2026-04-17 on ClinicalTrials.gov
What's tested:AAV9 BBP-812

At a glance

Recruiting sites
3 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants with Adverse Events (AEs)
Measured over Baseline up to Week 52
+2 more outcomes measured

Conditions

Canavan Disease

Explore related trials

Canavan DiseaseASPA GeneNACA Gene

Where it's being run

4 sites across 4 states
California1
Illinois1
Massachusetts1
New York1

Who to contact

Alicia Gomez
[email protected]
833-764-2267 or 617-861-4617
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

Exclusion

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

What this trial measures

  • 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) LevelsBaseline, 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