Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06614569
The ClinicalTrials.gov registry entry for NCT06614569 describes a study currently listed as active not recruiting, categorized as an unspecified phase. The registered enrollment target is 7 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 326-participant average among 6 other Tay-Sachs Disease trials with a reported enrollment target (98% lower). The listed sponsor is Terence Flotte, which has 2 total studies on file at ClinicalTrials.gov.
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 1 study location spanning 1 distinct geographic area - top geographies include 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.
Learn More About Clinical Trials
Similar trials for Tay-Sachs Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Longitudinal Study of Neurodegenerative Disorders
RECRUITING
-
A Natural History Study of the Gangliosidoses
RECRUITING
-
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
RECRUITING · Phase 1
-
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
COMPLETED
-
Stem Cell Transplant for Inborn Errors of Metabolism
COMPLETED · Phase 2
-
Gene Therapy for Tay-Sachs Disease
COMPLETED
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.