Clinical Trial

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

Active, Not Recruiting Early Phase 1
View on ClinicalTrials.gov →
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.
Protocol Amendment History 1 change
notable Enrollment reduced: 9 -> 3 participants 2026-08-11
Trial Details
NCT Number NCT06528080
Lead Sponsor Shanghai Jiao Tong University School of Medicine
Conditions Gaucher Disease
Enrollment 3 participants
Start Date 2024-08-01
Primary Completion 2027-05-30 (estimated)
Study Completion 2030-05-30 (estimated)
Updated on ClinicalTrials.gov 2026-08-10