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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: Clinical Trial Phase 1 study, sponsored by Terence Flotte.
NCT04669535 is a Phase 1 study that was terminated before completion, run by Terence Flotte. The registered enrollment target is 9 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
The measure of total numbers of total treatment emergent adverse events (TEAEs), serious adverse events (SAEs) after treatment with the vector whether related or unrelated to treatment as well as the number of adverse events (AEs) related to AXO-AAV-GM2 vector and neurosurgical procedures.
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 |
Why NCT04669535 stopped before completion
NCT04669535 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower).
The record links to 0 conditions, and to 4 interventions - of which AXO-AAV-GM2 Starting Dose is the first listed.
NCT04669535 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04669535's enrollment target sits among peer trials
9 1955th of 2000 higher than 42 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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