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

NCT06528080 · Status: ACTIVE_NOT_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2026-02-04

No results posted yet for this study

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.

Conditions

  • Gaucher Disease

Interventions

GENETIC

LY-M001

LY-M001 Injection by Single Intravenous Infusion

Sponsors & Collaborators

  • Shanghai Jiao Tong University School of Medicine

    lead OTHER

Principal Investigators

  • xiumin wang, PhD · Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2024-08-01
Primary Completion
2027-05-30
Completion
2030-05-30

Countries

  • China

Study Locations

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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 NCT06528080 on ClinicalTrials.gov