A Study of AAV9 Gene Therapy in Participants With Canavan Disease

Brief Title

A Study of AAV9 Gene Therapy in Participants With Canavan Disease

Official Title

A Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA Gene

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.

Detailed Description

      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.

Study Phase

Phase 1/Phase 2

Study Type


Primary Outcome

Number of Participants with Adverse Events (AEs)

Secondary Outcome

 Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88


Canavan Disease


AAV9 BBP-812

Study Arms / Comparison Groups

 Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1)
Description:  Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

September 8, 2021

Completion Date

March 15, 2028

Primary Completion Date

October 15, 2024

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

        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




N/A - 30 Months

Accepts Healthy Volunteers



, 833-764-2267 or 617-861-4617, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Aspa Therapeutics

Study Sponsor

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Verification Date

February 2023