A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
ConditionCanavan Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
AgeUp to 30
SponsorAspa Therapeutics
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.
Eligibility criteria
Qualifiers
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).
Elevated urinary NAA and
Biallelic mutation of the ASPA gene determined at Screening or documented in the participant's medical history.
Disqualifiers
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.
Presence of continuous/constant decerebrate or decorticate posturing,
Trial design
Treatments tested in this trial
- AAV9 BBP-812
Treatment groups
26 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov