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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04998396
Recruitment Status : Recruiting
First Posted : August 10, 2021
Last Update Posted : February 16, 2023
Sponsor:
Information provided by (Responsible Party):
Aspa Therapeutics

Study Description
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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.

Condition or disease Intervention/treatment Phase
Canavan Disease Biological: AAV9 BBP-812 Phase 1 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : September 8, 2021
Estimated Primary Completion Date : October 15, 2024
Estimated Study Completion Date : March 15, 2028

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants with Adverse Events (AEs) [ Time Frame: Baseline up to Week 52 ]
  2. Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels [ Time Frame: Baseline, Month 12 ]
  3. 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 ]

Secondary Outcome Measures :
  1. Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88 [ Time Frame: Baseline, Week 52 ]
  2. Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4 [ Time Frame: Baseline, Week 52 ]
  3. Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4 [ Time Frame: Baseline, Week 52 ]
  4. Change from Baseline to Week 52 in Communication Assessment, Bayley-4 [ Time Frame: Baseline, Week 52 ]
  5. Change from Baseline to Week 52 in Adaptive Function, Vineland-3 [ Time Frame: Baseline, Week 52 ]

Eligibility Criteria
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Ages Eligible for Study:   up to 30 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04998396


Contacts
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Contact: Canavan Disease Study Team 833-764-2267 or 617-861-4617 CANaspire@aspatx.com
Contact: David Rintell, Ed.D., VP, Head of Patient Advocacy 617-734-6778 clinicaltrials@aspatx.com

Locations
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United States, California
UCSF Benioff Children's Hospital Oakland Not yet recruiting
Oakland, California, United States, 94609
Contact: Annie R Sako    510-428-3885 ext 7217    annie.sako@ucsf.edu   
Principal Investigator: Alexander Fay, MD         
United States, Massachusetts
Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) Recruiting
Boston, Massachusetts, United States, 02114
Contact: Stacy Maciel       smaciel@mgh.harvard.edu   
Contact    617-726-2986      
Principal Investigator: Florian Eichler, MD         
United States, New York
Weill Cornell Medicine; Division of Pediatric Neurology Recruiting
New York, New York, United States, 10021
Contact: Kery Gao    212-746-3280    keg4005@med.cornell.edu   
Principal Investigator: Eric Mallack, MD         
Sponsors and Collaborators
Aspa Therapeutics
More Information
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Additional Information:

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Responsible Party: Aspa Therapeutics
ClinicalTrials.gov Identifier: NCT04998396    
Other Study ID Numbers: CVN-102
First Posted: August 10, 2021    Key Record Dates
Last Update Posted: February 16, 2023
Last Verified: February 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Aspa Therapeutics:
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
Additional relevant MeSH terms:
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Canavan Disease
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Leukoencephalopathies
Demyelinating Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases