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

Last updated: October 21, 2024
Sponsor: Aspa Therapeutics
Overall Status: Active - Recruiting

Phase

1/2

Condition

N/A

Treatment

AAV9 BBP-812

Clinical Study ID

NCT04998396
CVN-102
  • Ages < 30
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Maximum age for inclusion is 30 months.

  • Participant has stable health in the opinion of the investigator and as confirmed bymedical 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 inthe participant's medical history.

  • Active clinical signs of Canavan disease

  • Participant is up to date on all immunizations per local guidelines

Exclusion

Key Exclusion Criteria:

  • Tests positive for total anti-AAV9 antibodies determined by enzyme-linkedimmunosorbent 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-epilepticmedications

Study Design

Total Participants: 26
Treatment Group(s): 1
Primary Treatment: AAV9 BBP-812
Phase: 1/2
Study Start date:
September 08, 2021
Estimated Completion Date:
October 08, 2030

Study 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.

Connect with a study center

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California 94609
    United States

    Active - Recruiting

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

    Chicago, Illinois 60611
    United States

    Active - Recruiting

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

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • Weill Cornell Medicine; Division of Pediatric Neurology

    New York, New York 10065
    United States

    Completed

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