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

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

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.

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