Gene Therapy for Canavan Disease
(CAN-GT Trial)
Trial Summary
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
How is the rAAV-Olig001-ASPA treatment different from other treatments for Canavan disease?
The rAAV-Olig001-ASPA treatment is unique because it uses a viral vector to deliver a healthy version of the ASPA gene directly to the brain, aiming to restore enzyme function and reduce harmful substances in the brain. This gene therapy is novel as it targets the central nervous system and is administered through a specific route to reach affected brain areas, offering hope for a condition with no current standard treatment.12345
What is the purpose of this trial?
Canavan Disease is a congenital white matter disorder caused by mutations to the gene encoding for aspartoacylase (ASPA). Expression of ASPA is restricted to oligodendrocytes, the sole white matter producing lineage in the brain. ASPA supports myelination in the capacity of its sole known function, namely, the catabolism of N-acetylaspartate (NAA). Inherited mutations that result in loss of ASPA catabolic activity result in a typically severe phenotype of Canavan Disease, characterized by chronically elevated brain NAA, gross motor abnormalities, hypomyelination, progressive spongiform degeneration of the brain, epilepsy, blindness, and a short life expectancy. Disease severity is correlated with residual levels of enzyme activity. Reconstitution of ASPA function in oligodendrocytes of the brains of Canavan patients is expected to rescue NAA metabolism in its natural cellular compartment and support myelination/remyelination by resident white matter producing cells. This protocol directly targets oligodendrocytes in the brain, which are intimately involved with disease initiation and progression. Targeting oligodendrocytes offers the safest and most direct therapy for affected individuals.The latest generation AAV viral vector (rAAV-Olig001-ASPA) will be administered to patients using neurosurgical procedure which involves direct administration of gene therapy to affected regions of the brain. Outcome measures for the open label clinical trial include longitudinal clinical assessments and brain imaging.Currently, there is no effective treatment for Canavan Disease. The purpose of this study is to validate a new technology targeted to the cells most affected by Canavan Disease in the safest way possible.The study investigators are committed to supporting the Rare Disease \& Canavan Disease Communities. For more information, please contact Jordana Holovach, Head of Communications and Community at PatientAdvocacy@myrtellegtx.com.
Research Team
Robert Lober, MD, PhD
Principal Investigator
Dayton Children's Hospital
Eligibility Criteria
This trial is for children with typical Canavan Disease, a brain disorder. Eligible participants are aged 3-60 months and diagnosed by a neurologist. They can't have had gene therapy or intracranial surgery before, severe allergies, MRI contraindications, immune system issues, or other serious health problems.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Neurosurgical administration of a single dose of rAAV-Olig001-ASPA delivered intracerebroventricularly
Follow-up
Participants are monitored for safety and effectiveness after treatment, including neurological evaluations and imaging
Long-term follow-up
Extended monitoring of participants for long-term safety and efficacy outcomes
Treatment Details
Interventions
- rAAV-Olig001-ASPA
rAAV-Olig001-ASPA is already approved in United States, European Union for the following indications:
- Orphan Drug, Fast-Track and Rare Pediatric Disease Designations for the treatment of Canavan disease
- Orphan Drug Designation for the treatment of Canavan disease
Find a Clinic Near You
Who Is Running the Clinical Trial?
Myrtelle LLC
Lead Sponsor
Myrtelle Inc.
Lead Sponsor