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

  • End date
    Oct 31, 2028
  • participants needed
  • sponsor
    Aspa Therapeutics
Updated on 25 April 2022


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.


Canavan disease is an ultra-rare, profoundly disabling and fatal disease with no approved therapy. The Sponsor is developing BBP-812, an investigational gene therapy product for systemic delivery in participants with Canavan disease. BBP-812 is a recombinant adeno-associated virus serotype 9 (rAAV9) vector engineered to deliver the aspartoacylase (ASPA) transgene under control of a ubiquitous promoter to restore ASPA expression in both neuronal and non-neuronal cell types.

Condition Canavan Disease
Treatment AAV9 BBP-812
Clinical Study IdentifierNCT04998396
SponsorAspa Therapeutics
Last Modified on25 April 2022


Yes No Not Sure

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

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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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