rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease
Public ClinicalTrials.gov record NCT04833907. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Study identification
- NCT ID
- NCT04833907
- Recruitment status
- Enrolling by invitation
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Myrtelle Inc.
- Industry
- Enrollment
- 24 participants
Conditions and interventions
Conditions
Interventions
- Levetiracetam Drug
- Prednisone Drug
- rAAV-Olig001-ASPA Drug
Drug
Eligibility (public fields only)
- Age range
- 3 Months to 60 Months
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 31, 2021
- Primary completion
- Aug 30, 2026
- Completion
- Aug 30, 2027
- Last update posted
- Nov 13, 2025
2021 – 2027
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Dayton Children's Hospital | Dayton | Ohio | 45404 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04833907, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 13, 2025 · Synced May 11, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04833907 live on ClinicalTrials.gov.