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

NCT06162338 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2025-02-14

No results posted yet for this study

Summary

This is a prospective single-center, open, single-arm, single-dose intravenous infusion study to evaluate the safety and initial efficacy, pharmacodynamic characteristics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection.This study mainly includes the main study stage and the long-term follow-up study stage.

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

Sponsors & Collaborators

  • Lingyi Biotech Co., Ltd.

    collaborator INDUSTRY

  • He Huang

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-11-15
Primary Completion
2026-12-17
Completion
2031-12-17

Countries

  • China

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06162338 on ClinicalTrials.gov