Status:
ENROLLING_BY_INVITATION
rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease
Lead Sponsor:
Myrtelle Inc.
Conditions:
Canavan Disease
Eligibility:
All Genders
3-60 years
Phase:
PHASE1
PHASE2
Brief Summary
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 pr...
Detailed Description
rAAV-Olig001-ASPA is the first gene therapy designed to target the oligodendrocytes, which are critical for myelination and brain development. This study is a Phase 1/2 First-In-Human protocol design...
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.
Key Trial Info
Start Date :
April 1 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 31 2027
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT04833907
Start Date
April 1 2021
End Date
August 31 2027
Last Update
November 14 2025
Active Locations (1)
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1
Dayton Children's Hospital
Dayton, Ohio, United States, 45404