# A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

> **NCT04273269** · PHASE1,PHASE2 · TERMINATED · sponsor: **LYSOGENE** · enrollment: 5 (actual)

## Conditions studied

- GM1 Gangliosidosis

## Interventions

- **GENETIC:** LYS-GM101

## Key facts

- **NCT ID:** NCT04273269
- **Lead sponsor:** LYSOGENE
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2021-05-11
- **Primary completion:** 2023-05-22
- **Final completion:** 2023-05-22
- **Target enrollment:** 5 (ACTUAL)
- **Why stopped:** The clinical trial is closed due to Lysogene's cessation of activities. This study closure is not due to safety reasons.
- **Last updated:** 2023-06-09


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04273269

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04273269, "A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT04273269. Licensed CC0.

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