NCT06528080

A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease

A Prospective, Single-center, Open-label, Single-arm Clinical Study to Evaluate the Safety and Efficacy of a Single Intravenous Infusion of LY-M001 Injection in Pediatric and Adolescent Patients With Type 1 Gaucher Disease

• Status: Active Not Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Shanghai Jiao Tong University School of Medicine · Academic / Other
• Sex: All
• Age: 6 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to evaluate the safety, tolerability, efficacy, immunogenicity, PD and PK characteristics of LY-M001 injection in children with GD1 aged 6 years ≤ age \< 18 years. This study mainly includes the main study stage and the long-term follow-up study stage.

Detailed description

The study planned to enroll 6-9 patients with GD1 for 5 years, with a total of 34 follow-up visits. The main study period was 52 weeks, and the long-term follow-up period was 53 weeks to 5 years after administration. In this study, three dose groups were preset, and the first subject was enrolled with 1.0× 10\^13 vg/kg as the initial dose (the first dose group). After the safety was determined by DLT observation, subsequent subjects were enrolled. Based on safety and efficacy data, it will be decided by SRC discussion to increase to the next dose group. This is an open clinical study without blindness.

Conditions

• Gaucher Disease

Interventions

Type	Name	Description
GENETIC	LY-M001	LY-M001 Injection by Single Intravenous Infusion

Timeline

• Start date: 2024-08-01
• Primary completion: 2027-05-30
• Completion: 2030-05-30
• First posted: 2024-07-30
• Last updated: 2026-02-04

Locations

1 site across 1 country: China