RECRUITING

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

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Conditions

Canavan DiseaseAAVAAV9Gene therapyAspartoacylaseASPAASPA generAAV9ACY2Aminoacylase 2Spongy degenerationN-acetyl-L-aspartic acid (NAA)N-acetylaspartateRare diseaseInherited Metabolic DisordersLeukodystrophyLeukoencephalopathiesAutosomal Recessive DisorderNeurodevelopmental diseasesCANaspire Clinical Trial

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2021-09-08
Study Completion:2030-10-08
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04998396

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.

Ages Eligible for Study:Not specified to 30 Months
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Maximum age for inclusion is 30 months.
  2. * 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).
  3. * Participant has biochemical, genetic, and clinical diagnosis of Canavan disease:
  4. * Elevated urinary NAA and
  5. * Biallelic mutation of the ASPA gene determined at Screening or documented in the participant's medical history.
  6. * Active clinical signs of Canavan disease
  7. * Participant is up to date on all immunizations per local guidelines
  1. * Tests positive for total anti-AAV9 antibodies determined by enzyme-linked immunosorbent assay (ELISA).
  2. * Received prior gene therapy or other therapy (including vaccines) involving AAV.
  3. * Participant is receiving high-dose therapy with immunosuppressants.
  4. * Participant has significantly progressed Canavan disease characterized as:
  5. * Presence of continuous/constant decerebrate or decorticate posturing,
  6. * Recurrent status epilepticus, or
  7. * Recalcitrant seizures that do not respond while on 3 or more anti-epileptic medications

Contacts and Locations

Study Contact

Mary Rohrer, RN, BSN
CONTACT
833-764-2267 or 617-861-4617
CANaspire@aspatx.com
clinicaltrials@aspatx.com
CONTACT

Study Locations (Sites)

UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
Boston, Massachusetts, 02114
United States
Weill Cornell Medicine; Division of Pediatric Neurology
New York, New York, 10065
United States

Collaborators and Investigators

Sponsor: Aspa Therapeutics

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 Registration Dates

Study Start Date2021-09-08
Study Completion Date2030-10-08

Study Record Updates

Study Start Date2021-09-08
Study Completion Date2030-10-08

Terms related to this study

Keywords Provided by Researchers

  • 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

Additional Relevant MeSH Terms

  • Canavan Disease