Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Clinical Trial Phase 1 study, sponsored by LYSOGENE.
NCT04273269 is a Phase 1 study that was terminated before completion, run by LYSOGENE. The registered enrollment target is 5 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Physical examination by body system: normal/abnormal and change from previous assessment
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 |
Why NCT04273269 stopped before completion
NCT04273269 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 1 intervention - of which LYS-GM101 is the first listed.
NCT04273269 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04273269's enrollment target sits among peer trials
5 1989th of 2000 higher than 6 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI