NCT06818838

A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease

A Multicenter, Open, Single-arm, Single-dose, Dose-escalation, and Expanded Phase I/II Study Evaluating the Safety, Tolerability, and Efficacy of LY-M001 Injection in Adult Patients With Type I Gaucher Disease

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Lingyi Biotech Co., Ltd. · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate, Glucosylceramide (GlcCer), in macrophages in the liver, spleen, kidney, bone, lung, and even the brain, inducing their transformation into Gaucher cells whose cell cytoplasm presenting a characteristic "crumpled tissue paper" appearance, leading to pathological changes in involved tissues and organs.LY-M001 Injection is an rAAV8 vector gene therapy product. It can specifically transduce the target organ liver after a single intravenous administration and express the GCase protein in liver cells for a long period of time.

Detailed description

The purpose of this study is to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacokinetics and pharmacokinetics of LY-M001 injection in patients with GD1 using a multicenter, open, single-arm, single-dose, dose-escalation and extended clinical design. This study includes the main study phase and the long-term follow-up study phase. The primary study period is 52 weeks after LY-M001 infusion, and the long-term follow-up period is 53 weeks to 5 years after LY-M001 infusion.Subjects who complete the 52 weeks follow up period or who prematurely withdraw in this study will enter the long-term follow-up study phase to obtain long-term assessment data. Phase I is a dose escalation study consists of three preset dose groups, including one rollback dose group and two incremental dose groups, which are: Backdose (5 × 10\^12 vg/kg) group, dose group 1 (1.5 × 10\^13 vg/kg) and dose group 2 (3.0 × 10\^13 vg/kg), where dose group 1 was the starting dose of the Phase I study. Three subjects are enrolled in each dose group one by one, and each subject is added to the next subject after at least 28 days of DLT observation to determine safety. Phase I studies enrolled approximately 6 to 12 (up to 12) evaluable subjects. Phase II is a dose expansion study. After all subjects in Phase I study have completed the Day 28 (D28) observation following LY-M001 infusion, the Recommended Phase 2 Dose (RP2D) will be determined by the Safety Review Committee (SRC), which will also decide whether to proceed to the dose-expansion Phase II study.

Conditions

• Gaucher Disease Type 1

Interventions

Type	Name	Description
GENETIC	LY-M001	Single Intravenous Infusion of LY-M001 Injection.

Timeline

• Start date: 2024-07-05
• Primary completion: 2027-07-30
• Completion: 2031-07-30
• First posted: 2025-02-11
• Last updated: 2026-01-26

Locations

3 sites across 1 country: China