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Gene Therapy for Children with Canavan Disease

This study is testing a new gene therapy called rAAV-Olig001-ASPA for children with typical Canavan Disease. Canavan Disease is a genetic condition that affects the brain's white matter, leading to problems with movement, vision, and development. The gene therapy aims to deliver a working copy of the ASPA gene, which is faulty in Canavan Disease. Participants will receive a single dose of rAAV-Olig001-ASPA directly into the fluid surrounding the brain. They will also receive Levetiracetam (Keppra) to prevent seizures and Prednisone to manage potential immune responses. The main goal of this study is to evaluate the safety of the gene therapy over 12 months. The study plans to enroll up to 24 children between 3 months and 60 months old who have a definite diagnosis of typical Canavan Disease. The current recruitment status is unclear.

Study design
This is an open-label study, meaning both you and the study team will know what treatments are being given. It is a single-arm study, meaning all participants will receive the gene therapy. Up to 24 children will be enrolled.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety evaluation for 12 months after receiving the dose.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04833907

rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

Enrolling by Invitation
PHASE1Ages 3–60InterventionalTreatment
Myrtelle Inc.
~24 participants
Updated 2025-11-14 on ClinicalTrials.gov
What's tested:rAAV-Olig001-ASPALevetiracetamPrednisone

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety evaluation
Measured over 12 Months Post Dose

Conditions

Canavan Disease

Explore related trials

Canavan DiseaseASPA Gene

Where it's being run

1 sites across 1 states
Ohio1

Study leadership

  • Robert Lober, MD, PhD · PRINCIPAL_INVESTIGATOR · Dayton Children's Hospital

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Definitive diagnosis of typical CD by a board certified neurologist.
Written informed consent from parent(s)/guardian(s). Consent to enroll into the study will include a written agreement to comply with all the conditions of the study, including attendance at follow-up visits.
For cohort 1: age more than 36 months and up to 60 months.
For cohort 2: age between 15 months and 36 months.
For cohort 3: age less than 15 months.

Exclusion

At the discretion of the PI, any significant chronic medical condition, including, but not limited to neurological, cardiac, hepatic, renal, hematological, gastrointestinal, endocrine, pulmonary, or infectious disease, which would put the subject at increased risk during surgery or which would interfere with participation in the study, interpretation of safety monitoring, or the integrity of the study data.
History of severe allergic reaction or anaphylaxis.
Past participation in gene therapy trials or receipt of any other investigational product within 6 months prior to enrollment.
Prior intracranial surgery.
Any absolute contraindication to immunosuppression.
Any absolute contraindication to MRI.
Any vaccination less than 1 month prior to gene therapy.
Anticipated life expectancy of less than 12 months for any reason.
GMFM-88 total raw score \>35%.
Clinically significant out-of-range lab values, at the discretion of clinical PI.

What this trial measures

  • Safety evaluation12 Months Post Dose

    Number, severity, and causal relationship of any adverse event (to either the gene therapy and/or surgical trial procedures required for vector administration) using the Common Terminology Criteria for Adverse Events (CTCAE) v5.0.