Study of Single Intravenous LY-M001 Injection for Safety and Early Effectiveness in Adults with Gaucher Disease Type I

What this Trial Is About

This research aims to evaluate the safety, initial efficacy, pharmacodynamics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection in adult patients with Gaucher Disease Type I. The study is prospective, single-center, open-label, single-arm, and involves a single intravenous dose. The study includes a main phase and a long-term follow-up phase, focusing on adults aged 18 to 60 with confirmed Gaucher Disease Type I. Participants receive a single intravenous infusion of LY-M001 at a dose calculated based on their body weight, administered at a rate of 1 mL per minute. The initial dose group uses 5.0 1012 vgkg, starting with one subject observed for 28 days for safety before enrolling additional subjects. Dose adjustments may be made depending on safety and efficacy data. The study includes a screening period, baseline period, treatment and safety observation period, and a short-term follow-up lasting up to 38 weeks. Those completing the main study or withdrawing early may enter a long-term follow-up lasting up to five years. Throughout the study, participants undergo various assessments including monitoring for treatment-related adverse events and dose-limiting toxicities within 38 weeks post-infusion. Laboratory tests measure glucocerebrosidase protein levels and enzyme activity, as well as glucosylsphingosine in plasma. Participants complete all required evaluations at the end of the study or early withdrawal, and are monitored for safety and efficacy during both short-term and long-term follow-up periods.

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