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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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, month 12

Awards & highlights

CANaspire Trial 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.Check my eligibility

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.See study design

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.

CANaspire Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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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.

CANaspire Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, month 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, month 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, month 12 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS)

Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels

Number of Participants with Adverse Events (AEs)

Secondary outcome measures

Change from Baseline to Week 52 in Adaptive Function, Vineland-3

Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4

Change from Baseline to Week 52 in Communication Assessment, Bayley-4

+2 more

CANaspire 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.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Aspa TherapeuticsLead Sponsor

1 Previous Clinical Trials

40 Total Patients Enrolled

1 Trials studying Canavan Disease

40 Patients Enrolled for Canavan Disease

Media Library

Canavan Disease Research Study Groups: Enrollment Expansion Phase: BBP-812, Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1), Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2)

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many test subjects are being given this medication?

"Yes, the latest information from clinicaltrials.gov supports that this trial is still ongoing and recruiting patients. The trial was first posted on September 8th, 2021 and 18 patients are needed at a single location."

Answered by AI

Are we still looking for people to enroll in this trial?

"Yes, as seen on clinicaltrials.gov, this research study is open and looking for 18 patients at a single site. The trial was first advertised on September 8th, 2021 and has had 3/12/2022 as its most recent update."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study of AAV9 Gene Therapy in Participants With Canavan Disease

2021. "A Study of AAV9 Gene Therapy in Participants With Canavan Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04998396.