Canavan DiseasePhase 1/2NCT04998396
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
A Phase 1/2 study testing AAV9 BBP-812 in people with Canavan Disease, run by Aspa Therapeutics at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 30 months
- Conditions the study is for
- Canavan Disease
- What is being tested?
- AAV9 BBP-812
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 4 sitesUnited States
- Study dates
- 8 Sep 2021to 8 Oct 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 30 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
clinicaltrials@aspatx.com contact
United States4 sites, 3 recruiting
- Recruiting
Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
Boston, Massachusetts, 02114
Sam Kartsonis, 610-766-2583, skartsonis@mgh.harvard.edu
Aliza (Hattie) Wilson, awilson65@bwh.harvard.edu
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Prachi Patel, 312-227-0067, prachipatel@luriechildrens.org
Jennifer Rubin, MD
- Completed
Weill Cornell Medicine; Division of Pediatric Neurology
New York, New York, 10065
- Recruiting
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Marissa Evans, 510-428-3885 ext. 5421, Marissa.Evans@ucsf.edu
Grace Sobrero, 510-428-3885 ext. 5421, grace.sobrero@ucsf.edu
About this study
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
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.
Read the 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.
- Conditions
- Canavan Disease
- Being tested
- AAV9 BBP-812
- Keywords
- 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
- Medical subject headings
- Canavan Disease, Rare Diseases, Leukoencephalopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV9 BBP-812Biological
Sterile solution for injection for 1-time use via volumetric infusion pump
| Study group | Role | What they receive |
|---|---|---|
| Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1) | Experimental | Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study. Receives: Biological: AAV9 BBP-812 |
| Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2) | Experimental | Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study. Receives: Biological: AAV9 BBP-812 |
| Enrollment Expansion Phase: BBP-812 | Experimental | 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. Receives: Biological: AAV9 BBP-812 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events (AEs)
Measured: Baseline up to Week 52
Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels
Measured: Baseline, Month 12
Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS)
Measured: Baseline, Month 12
Secondary outcomes (5)
Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88
Measured: Baseline, Week 52
Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4
Measured: Baseline, Week 52
Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4
Measured: Baseline, Week 52
Change from Baseline to Week 52 in Communication Assessment, Bayley-4
Measured: Baseline, Week 52
Change from Baseline to Week 52 in Adaptive Function, Vineland-3
Measured: Baseline, Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2021
- Study start8 Sep 2021
- Primary completion13 Oct 2026 (estimated)
- Study completion8 Oct 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Canavan DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aspa Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04998396
- Sponsor's protocol ID
- CVN-102
- Status last verified
- Apr 2026
- First submitted
- 5 Aug 2021
- Last updated
- 17 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04998396
- Acronym / sponsor ID
- CVN-102
- Conditions
- Canavan Disease
- Keywords
- Canavan Disease, AAV, AAV9, Gene therapy, Aspartoacylase, ASPA, ASPA gene, rAAV9
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 26 participants (estimated)
- Lead sponsor
- Aspa TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 8 Sep 2021 (actual)
- Primary completion
- 13 Oct 2026 (estimated)
- Study completion
- 8 Oct 2032 (estimated)
- First posted
- 10 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1) | Experimental | Biological: AAV9 BBP-812Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study. |
| Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2) | Experimental | Biological: AAV9 BBP-812Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study. |
| Enrollment Expansion Phase: BBP-812 | Experimental | Biological: AAV9 BBP-812Participants 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. |
Who may qualify
- Ages
- Up to 30 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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:
- Participant is up to date on all immunizations per local guidelines
Key exclusion criteria (7 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV9 BBP-812Sterile solution for injection for 1-time use via volumetric infusion pump | Biological | Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1), Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2), Enrollment Expansion Phase: BBP-812 |
Primary outcome
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
5 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2026.
- Aspa Therapeutics Company WebsiteLink provided by the sponsor.
- CANaspire Clinical Trial WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Active, not recruitingObservational study
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rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease
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The Myelin Disorders Biorepository Project
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
clinicaltrials@aspatx.com contact
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AAV9 BBP-812 in people with Canavan Disease, run by Aspa Therapeutics at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alicia Gomez (833-764-2267 or 617-861-4617, CANaspire@aspatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Adverse Events (AEs) (time frame: Baseline up to Week 52). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Apr 2026.