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NCT04669535 · ClinicalTrials.gov registry record · Phase 1
A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
A Phase 1 study, sponsored by Terence Flotte.
- Terminated
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
NCT04669535 is a Phase 1 study that was terminated before completion, run by Terence Flotte. The registered enrollment target is 9 participants.
The verdict
NCT04669535, a Phase 1 study, was terminated before completion, sponsored by Terence Flotte.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
Study Summary
The AXO-GM2-001 study is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of a bilateral thalamic and intracisternal/intrathecal infusion of AXO-AAV-GM2 in pediatric participants with GM2 Gangliosidosis (also known as Tay-Sachs or Sandhoff Diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the β-hexosaminidase A (HexA) enzyme. AXO-AAV-GM2 is an investigational gene therapy that aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via co-administration of two vectors utilizing the neurotropic adeno-associated virus recombinant human 8 serotype (AAVrh.8) capsid carrying the human HEXA or HEXB cDNA. The trial is expected to enroll pediatric participants with Tay-Sachs or Sandhoff Diseases, where infantile-onset participants will range from 6 months to 20 months old, and juvenile-onset participants will range from 2 years to 12 years old.
Interventions
- BIOLOGICAL AXO-AAV-GM2 Starting Dose
- BIOLOGICAL AXO-AAV-GM2 Low Dose
- BIOLOGICAL AXO-AAV-GM2 Middle Dose
- BIOLOGICAL AXO-AAV-GM2 High Dose
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2021-01-15 |
| Est. Completion | 2024-12-16 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04669535
The ClinicalTrials.gov registry entry for NCT04669535 describes a study currently listed as terminated, categorized as Phase 1. The registered enrollment target is 9 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Terence Flotte, which has 2 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 4 interventions - of which AXO-AAV-GM2 Starting Dose is the first listed.
NCT04669535 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT04669535 about?
NCT04669535 is a clinical study titled "A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease". The AXO-GM2-001 study is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of a bilateral thalamic and intracisternal/intrathecal infusion of AXO-AAV-GM2 in pediatric participants with GM2 Gangliosidosis (also known as...
What is the current status of trial NCT04669535?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2021-01-15. Estimated completion is 2024-12-16.
What interventions are being tested in trial NCT04669535?
The interventions under investigation include: AXO-AAV-GM2 Starting Dose (BIOLOGICAL), AXO-AAV-GM2 Low Dose (BIOLOGICAL), AXO-AAV-GM2 Middle Dose (BIOLOGICAL), AXO-AAV-GM2 High Dose (BIOLOGICAL).
Who is sponsoring clinical trial NCT04669535?
This trial is sponsored by Terence Flotte, which has 2 total clinical trials registered on ClinicalTrials.gov.
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