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Virus Therapy
Gene Therapy for Canavan Disease (CANaspire Trial)
Phase 1 & 2
Recruiting
Research Sponsored by Aspa Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participant has biochemical, genetic, and clinical diagnosis of Canavan disease:
Biallelic mutation of the ASPA gene determined at Screening or documented in the participant's medical history.
Must not have
Presence of continuous/constant decerebrate or decorticate posturing,
Participant has significantly progressed Canavan disease characterized as:
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group
Summary
This trial is testing a gene therapy for safety, tolerability, and how well it works in children with Canavan disease.
Who is the study for?
This trial is for children up to 30 months old with Canavan disease, a stable health condition, and no other serious diseases. They must have a genetic mutation in the ASPA gene and show signs of Canavan disease. Kids who've had previous gene therapy or certain immunosuppressants, or those with severe seizures uncontrolled by medication can't participate.
What is being tested?
The study tests BBP-812, an experimental gene therapy using AAV9 to treat Canavan disease in young children. It aims to assess how safe it is, how well patients tolerate it, and its effect on the body's functions related to the disease.
What are the potential side effects?
While specific side effects are not listed here, potential risks may include immune reactions due to the viral vector used (AAV9), issues at injection sites, and general symptoms like fever or fatigue as common responses to gene therapies.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with Canavan disease through tests and symptoms.
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I have a genetic mutation in the ASPA gene.
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I show symptoms of Canavan disease.
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I am 30 months old or younger.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I exhibit involuntary, rigid body postures.
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My Canavan disease has significantly worsened.
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I have previously received gene therapy or vaccines involving AAV.
Select...
My seizures do not improve despite taking 3 or more epilepsy drugs.
Select...
I have had more than one episode of prolonged seizures.
Select...
I am currently on high-dose immunosuppressant therapy.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
3Treatment groups
Experimental Treatment
Group I: Enrollment Expansion Phase: BBP-812Experimental Treatment1 Intervention
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.
Group II: Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2)Experimental Treatment1 Intervention
Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study.
Group III: Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1)Experimental Treatment1 Intervention
Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study.
Find a Location
Who is running the clinical trial?
Aspa TherapeuticsLead Sponsor
1 Previous Clinical Trials
70 Total Patients Enrolled
1 Trials studying Canavan Disease
70 Patients Enrolled for Canavan Disease
Media Library
Research Study Groups:
This trial has the following groups:- Group 1: Enrollment Expansion Phase: BBP-812
- Group 2: Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1)
- Group 3: Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2)