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

Aspa Therapeutics

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Canavan Disease

Treatments: Biological: AAV9 BBP-812

Study type: Interventional

Funder types: Industry

Identifiers

NCT04998396

CVN-102

Details and patient eligibility

About

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.

Full 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.

Enrollment: 26 estimated patients

Sex: All

Ages: Under 30 months old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

• 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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26 participants in 3 patient groups

Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1)

Experimental group

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.

Treatment:

Biological: AAV9 BBP-812

Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2)

Experimental group

Description:

Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study.

Treatment:

Biological: AAV9 BBP-812

Enrollment Expansion Phase: BBP-812

Experimental group

Description:

Participants will receive a single IV infusion of BBP-812 at the selected dose from the dose-finding phase on Day 0 in expansion phase of the study.

Treatment:

Biological: AAV9 BBP-812