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

September 20, 2023 updated by: Aspa Therapeutics

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

18

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: clinicaltrials@aspatx.com

Study Contact Backup

Study Locations

  • United States
    • California
      • Oakland, California, United States, 94609
        • Recruiting
        • UCSF Benioff Children's Hospital Oakland
        • Principal Investigator:
          • Alexander Fay, MD
        • Contact:
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Not yet recruiting
        • Ann & Robert H. Lurie Children's Hospital of Chicago
        • Contact:
        • Principal Investigator:
          • Jennifer Rubin, MD
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
        • Principal Investigator:
          • Florian Eichler, MD
        • Contact:
        • Contact:
    • New York
      • New York, New York, United States, 10065
        • Recruiting
        • Weill Cornell Medicine; Division of Pediatric Neurology
        • Contact:
        • Principal Investigator:
          • Zuhal Ergonul, MD PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 2 years (Child)

Accepts Healthy Volunteers

No

Description

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 medications

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
Biological: AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump
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.
Biological: AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump
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.
Biological: AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants with Adverse Events (AEs)
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels
Time Frame: Baseline, Month 12
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
Baseline, Month 12

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88
Time Frame: Baseline, Week 52
Baseline, Week 52
Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4
Time Frame: Baseline, Week 52
Baseline, Week 52
Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4
Time Frame: Baseline, Week 52
Baseline, Week 52
Change from Baseline to Week 52 in Communication Assessment, Bayley-4
Time Frame: Baseline, Week 52
Baseline, Week 52
Change from Baseline to Week 52 in Adaptive Function, Vineland-3
Time Frame: Baseline, Week 52
Baseline, Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Aspa Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 8, 2021

Primary Completion (Estimated)

October 15, 2024

Study Completion (Estimated)

March 15, 2028

Study Registration Dates

First Submitted

August 5, 2021

First Submitted That Met QC Criteria

August 9, 2021

First Posted (Actual)

August 10, 2021

Study Record Updates

Last Update Posted (Actual)

September 21, 2023

Last Update Submitted That Met QC Criteria

September 20, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CVN-102

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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