A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
NCT ID: NCT04998396
Last Updated: 2024-10-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1/PHASE2
26 participants
INTERVENTIONAL
2021-09-08
2030-10-08
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Canavan-Single Patient IND
NCT05317780
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
NCT04783181
A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria
NCT05972629
Natural History Study of Patients With Canavan Disease (CANinform Study)
NCT04126005
The Safety and Efficacy of Canakinumab in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada
NCT01105507
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
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.
AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump
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.
AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump
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.
AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
Exclusion Criteria
* 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
30 Months
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Aspa Therapeutics
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
Boston, Massachusetts, United States
Weill Cornell Medicine; Division of Pediatric Neurology
New York, New York, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
[email protected]
Role: CONTACT
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Related Links
Access external resources that provide additional context or updates about the study.
Aspa Therapeutics Company Website
CANaspire Clinical Trial Website
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
CVN-102
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.