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

Description

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

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Canavan Disease
Intervention / Treatment

-

Contacts and Locations

Oakland

UCSF Benioff Children's Hospital Oakland, Oakland, California, United States, 94609

Chicago

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Boston

Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND), Boston, Massachusetts, United States, 02114

New York

Weill Cornell Medicine; Division of Pediatric Neurology, New York, New York, United States, 10065

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

to 30 Months

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Aspa Therapeutics,

Study Record Dates

2030-10-08