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

NCT04998396 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CVN-102
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 4

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

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

Outcome Measures

Primary Outcomes (3)

• Number of Participants with Adverse Events (AEs)

  Baseline up to Week 52

• Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels

  Baseline, Month 12

• Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS)

  Baseline, Month 12

Secondary Outcomes (5)

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

  Baseline, Week 52

• Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4

  Baseline, Week 52

• Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4

  Baseline, Week 52

• Change from Baseline to Week 52 in Communication Assessment, Bayley-4

  Baseline, Week 52

• Change from Baseline to Week 52 in Adaptive Function, Vineland-3

  Baseline, Week 52

Study Arms (3)

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

EXPERIMENTAL

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

Biological: AAV9 BBP-812

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

EXPERIMENTAL

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

Biological: AAV9 BBP-812

Enrollment Expansion Phase: BBP-812

EXPERIMENTAL

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.

Biological: AAV9 BBP-812

Interventions

AAV9 BBP-812 BIOLOGICAL

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

Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1); Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2); Enrollment Expansion Phase: BBP-812

Eligibility Criteria

• Age: Up to 30 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Aspa Therapeutics: lead

Study Sites (4)

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

RECRUITING

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

Chicago, Illinois, 60611, United States

RECRUITING

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

Boston, Massachusetts, 02114, United States

RECRUITING

Weill Cornell Medicine; Division of Pediatric Neurology

New York, New York, 10065, United States

COMPLETED

Related Links

• Aspa Therapeutics Company Website
• CANaspire Clinical Trial Website

MeSH Terms

Conditions

Canavan Disease; Rare Diseases; Leukoencephalopathies

Condition Hierarchy (Ancestors)

Hereditary Central Nervous System Demyelinating Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Demyelinating Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Alicia Gomez

CONTACT

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

clinicaltrials@aspatx.com

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2021
• First Posted: August 10, 2021
• Study Start: September 8, 2021
• Primary Completion (Estimated): October 13, 2026
• Study Completion (Estimated): October 8, 2032
• Last Updated: April 17, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)