Gene therapy for children with Canavan Disease

Phase 1/2, Open Label, Sequential Cohort Study of a Single Intracranial Dose of AVASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

This study is testing a new gene therapy for children with Canavan Disease to see if it can help improve their brain function and safety after receiving a single dose directly into the brain.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 3 Months to 60 Months
• Sex: All
• Sponsor: Myrtelle Inc. (Industry-sponsored)
• Locations: 1 site (Dayton, Ohio)
• Trial ID: NCT04833907 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and efficacy of rAAV-Olig001-ASPA gene therapy in children diagnosed with typical Canavan Disease. The therapy aims to restore the function of the aspartoacylase enzyme in oligodendrocytes, which is crucial for myelination in the brain. The study is designed as a Phase 1/2 open-label trial, where participants will receive a single dose of the gene therapy delivered directly into the brain. Up to 24 children will be enrolled, and their progress will be monitored for safety and therapeutic effects.

Who should consider this trial

Good fit: Ideal candidates for this study are children aged 15 months to 60 months with a definitive diagnosis of typical Canavan Disease.

Not a fit: Patients with significant chronic medical conditions or those who have previously participated in gene therapy trials may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve neurological function and quality of life for children suffering from Canavan Disease.

How similar studies have performed: While gene therapy approaches for neurological disorders are emerging, this specific application for Canavan Disease is novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

* 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 Criteria:

* 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.

Where this trial is running

Dayton, Ohio

• Dayton Children's Hospital — Dayton, Ohio, United States (Recruiting)

Study contacts

• Principal investigator: Robert Lober, MD, PhD — Dayton Children's Hospital
• Study coordinator: Jordana Holovach

Conditions Canavan Disease; Gene Therapy; Aspartoacylase; N-Acetyl-Aspartate; Oligodendrocyte; Leukodystrophy; Adeno-Associated Virus Vector; Autosomal Recessive Disorder