Recruiting
Phase 1
Phase 2

AAV9 Gene Therapy

Sponsor:

Aspa Therapeutics

Code:

NCT04998396

Conditions

Canavan Disease

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

Interventions

AAV9 BBP-812

Study Details

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.

Conditions

Canavan Disease

Study ID

NCT04998396

Start date

Sep 8, 2021

Status verified date

Apr, 2026

Completion date

Oct 8, 2032

Anticipated

Primary completion date

Oct 13, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

26 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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

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

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

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

experimental: Enrollment Expansion Phase: BBP-812

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.

Interventions

AAV9 BBP-812

Sterile solution for injection for 1-time use via volumetric infusion pump

Primary outcome measure

  • Number of Participants with Adverse Events (AEs) [ Time Frame: Baseline up to Week 52 ]
  • Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels [ Time Frame: Baseline, Month 12 ]
  • Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS) [ Time Frame: Baseline, Month 12 ]

Central Contacts and Locations

Central contacts

Alicia Gomez

833-764-2267 or 617-861-4617CANaspire@aspatx.com

Locations

UCSF Benioff Children's Hospital Oakland

Recruiting

Oakland, California, United States, 94609

Contacts

Marissa Evans

510-428-3885Marissa.Evans@ucsf.edu

Grace Sobrero

510-428-3885grace.sobrero@ucsf.edu

Principal Investigator:

Alexander Fay, MD

Ann & Robert H. Lurie Children's Hospital of Chicago

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Prachi Patel

312-227-0067prachipatel@luriechildrens.org

Principal Investigator:

Jennifer Rubin, MD

Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Sam Kartsonis

610-766-2583skartsonis@mgh.harvard.edu

Aliza (Hattie) Wilson

awilson65@bwh.harvard.edu

Principal Investigator:

Florian Eichler, MD

More Information

Sponsor

Aspa Therapeutics

Last update posted

Apr 17, 2026

Last verified

Apr, 2026

Keywords

  • Canavan Disease
  • AAV
  • AAV9
  • Gene therapy
  • Aspartoacylase
  • ASPA
  • ASPA gene
  • rAAV9
  • ACY2
  • Aminoacylase 2
  • Spongy degeneration
  • N-acetyl-L-aspartic acid (NAA)
  • N-acetylaspartate
  • Rare disease
  • Inherited Metabolic Disorders
  • Leukodystrophy
  • Leukoencephalopathies
  • Autosomal Recessive Disorder
  • Neurodevelopmental diseases
  • CANaspire Clinical Trial

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Aspa Therapeutics on 2026-04-17.