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NCT04273269 · ClinicalTrials.gov registry record · Phase 1
A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis
A Phase 1 study, sponsored by LYSOGENE.
- Terminated
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
NCT04273269 is a Phase 1 study that was terminated before completion, run by LYSOGENE. The registered enrollment target is 5 participants.
The verdict
NCT04273269, a Phase 1 study, was terminated before completion, sponsored by LYSOGENE.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
Study Summary
LYS-GM101 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the central nervous system. This study will assess, in a 2-stage adaptive-design, the safety and efficacy of treatment in subjects with infantile GM1 gangliosidosis.
Interventions
- GENETIC LYS-GM101
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2021-05-11 |
| Est. Completion | 2023-05-22 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04273269
The ClinicalTrials.gov registry entry for NCT04273269 describes a study currently listed as terminated, categorized as Phase 1. The registered enrollment target is 5 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is LYSOGENE, which has 2 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which LYS-GM101 is the first listed.
NCT04273269 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT04273269 about?
NCT04273269 is a clinical study titled "A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis". LYS-GM101 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the central nervous system. This study will assess, in a 2-stage adaptive-design, the safety and efficacy of treatment in subjects with infantile GM1 gangliosidosis.
What is the current status of trial NCT04273269?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2021-05-11. Estimated completion is 2023-05-22.
What interventions are being tested in trial NCT04273269?
The interventions under investigation include: LYS-GM101 (GENETIC).
Who is sponsoring clinical trial NCT04273269?
This trial is sponsored by LYSOGENE, which has 2 total clinical trials registered on ClinicalTrials.gov.
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