A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
Public ClinicalTrials.gov record NCT04669535. 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
A Two-Stage, Dose-Escalation and Safety & Efficacy Study of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
Study identification
- NCT ID
- NCT04669535
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Terence Flotte
- Other
- Enrollment
- 9 participants
Conditions and interventions
Conditions
Interventions
- AXO-AAV-GM2 Starting Dose Biological
- AXO-AAV-GM2 Low Dose Biological
- AXO-AAV-GM2 Middle Dose Biological
- AXO-AAV-GM2 High Dose Biological
Biological
Eligibility (public fields only)
- Age range
- 6 Months to 12 Years
- 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
- Jan 14, 2021
- Primary completion
- May 22, 2024
- Completion
- Dec 15, 2024
- Last update posted
- Sep 1, 2025
2021 – 2024
United States locations
- U.S. sites
- 2
- U.S. states
- 1
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Massachusetts General Hospital, Center for Rare Neurological Diseases | Boston | Massachusetts | 02114 | — |
| University of Massachusetts Medical Health Center | Worcester | Massachusetts | 01655 | — |
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 NCT04669535, 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 Sep 1, 2025 · Synced Jul 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04669535 live on ClinicalTrials.gov.