A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients with Gaucher Disease Type I

NARecruitingINTERVENTIONAL
Enrollment

3

Participants

Timeline

Start Date

November 15, 2023

Primary Completion Date

December 17, 2026

Study Completion Date

December 17, 2031

Conditions
Gaucher Disease Type I
Interventions
BIOLOGICAL

LY-M001 Injection

With 5.0 × 10\^12 vg/kg as the initial effective dose (first dose group), 1 to 2 subjects are expected to be included. The first dose group was enrolled by sentinel method, and the first subject in this group was observed for at least 28 days after receiving LY-M001 (dose-limited toxicity \[DLT\] observation period) to enroll the next subject. After the first participant completes the safety evaluation of dosing for at least 28 days, the investigator (PI) and the partner discuss the safety and efficacy data and decide to maintain the first dose or increase/reduce it to another dose group

Trial Locations (1)

310003

RECRUITING

The First Affiliated Hospital of Zhejiang University School of Medicine, Hanzhou

All Listed Sponsors
collaborator

Lingyi Biotech Co., Ltd.

INDUSTRY

lead

He Huang

OTHER