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NCT06614569 · ClinicalTrials.gov registry record
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
A clinical trial of Tay-Sachs Disease and Sandhoff Disease, sponsored by Terence Flotte.
- Active
- Registry status
- 7
- Enrollment target
- 1
- Study location
NCT06614569: Active study of Tay-Sachs Disease and Sandhoff Disease, sponsored by Terence Flotte.
NCT06614569 is a study of Tay-Sachs Disease and Sandhoff Disease that is active but no longer recruiting, run by Terence Flotte. The registered enrollment target is 7 participants, below the 326-participant average among 6 other Tay-Sachs Disease trials with a reported enrollment target (98% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06614569, a study of Tay-Sachs Disease and Sandhoff Disease, is active but no longer recruiting, sponsored by Terence Flotte.
- ACTIVE NOT RECRUITING
- Registry status
- 7 participants
- Enrollment target
- 1
- Study location
Study Summary
This study is to continue Long-Term Follow-Up of Patients who were previously treated with AXO-AAV-GM2 Gene Therapy as treatment for Tay-Sachs or Sandhoff Disease to follow the subjects through 5 years after their initial gene therapy treatment.
Primary Outcome
Participants will be monitored for adverse events, including delayed adverse events, as a result of AXO-AAV-GM2 including de novo cancer, neurologic, rheumatologic, and hematologic/immunologic disorders.
Conditions Studied
Interventions
- DRUG AXO-AAV-GM2
Study Locations (1)
Massachusetts
- UMass Chan Medical School - Worcester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 7 participants |
| Start Date | 2024-09-17 |
| Est. Completion | 2028-12 |
What the registry record for NCT06614569 still lists
NCT06614569 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 7 participants, a relatively small participant target, below the 326-participant average among 6 other Tay-Sachs Disease trials with a reported enrollment target (98% lower).
The record links to 3 conditions, with Tay-Sachs Disease appearing as the primary indexed condition, and to 1 intervention - of which AXO-AAV-GM2 is the first listed.
NCT06614569 reports a single indexed study location in Massachusetts.
Frequently Asked Questions
What is clinical trial NCT06614569 about?
NCT06614569 is a clinical study titled "Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease". This study is to continue Long-Term Follow-Up of Patients who were previously treated with AXO-AAV-GM2 Gene Therapy as treatment for Tay-Sachs or Sandhoff Disease to follow the subjects through 5 years after their initial gene therapy treatment.
What is the current status of trial NCT06614569?
This trial is currently active not recruiting. The enrollment target is 7 participants. The study started on 2024-09-17. Estimated completion is 2028-12.
What conditions does trial NCT06614569 study?
This clinical trial studies the following conditions: Tay-Sachs Disease, Sandhoff Disease, GM2 Gangliosidosis.
What interventions are being tested in trial NCT06614569?
The interventions under investigation include: AXO-AAV-GM2 (DRUG).
Who is sponsoring clinical trial NCT06614569?
This trial is sponsored by Terence Flotte, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06614569 being conducted?
This trial has 1 study location across Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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