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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 : April 12, 2023
Sponsor:
Information provided by (Responsible Party):
Aspa Therapeutics

Tracking Information
First Submitted Date  ICMJE August 5, 2021
First Posted Date  ICMJE August 10, 2021
Last Update Posted Date April 12, 2023
Actual Study Start Date  ICMJE September 8, 2021
Estimated Primary Completion Date October 15, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 14, 2023)
  • Number of Participants with Adverse Events (AEs) [ Time Frame: Baseline up to Week 52 ]
  • Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels [ Time Frame: 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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: August 9, 2021)
  • Number of Participants With Adverse Events (AEs) [ Time Frame: Baseline up to Week 52 ]
  • Change From Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels [ Time Frame: 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 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 9, 2021)
  • Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88 [ Time Frame: Baseline, Week 52 ]
  • Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4 [ Time Frame: Baseline, Week 52 ]
  • Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4 [ Time Frame: Baseline, Week 52 ]
  • Change from Baseline to Week 52 in Communication Assessment, Bayley-4 [ Time Frame: Baseline, Week 52 ]
  • Change from Baseline to Week 52 in Adaptive Function, Vineland-3 [ Time Frame: Baseline, Week 52 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of AAV9 Gene Therapy in Participants With Canavan Disease
Official Title  ICMJE 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
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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Canavan Disease
Intervention  ICMJE Biological: AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump
Study Arms  ICMJE
  • 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.
    Intervention: Biological: AAV9 BBP-812
  • 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.
    Intervention: Biological: AAV9 BBP-812
  • 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.
    Intervention: Biological: AAV9 BBP-812
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 9, 2021)		 18
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 15, 2028
Estimated Primary Completion Date October 15, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 30 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
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
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04998396
Other Study ID Numbers  ICMJE CVN-102
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Aspa Therapeutics
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Aspa Therapeutics
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Aspa Therapeutics
Verification Date April 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP