Study of LY-M001 Gene Therapy for Adults Aged 18 to 60 with Type 1 Gaucher Disease Evaluating Safety, Tolerability, and Effectiveness with Single Intravenous Dose

What this Trial Is About

Gaucher disease type 1 GD1 is caused by mutations in the GBA1 gene, leading to a deficiency in the enzyme glucocerebrosidase GCase. This deficiency results in the buildup of glucosylceramide GlcCer in various organs, including the liver, spleen, kidney, bone, lung, and brain, causing cells to change and leading to tissue and organ problems. Researchers are evaluating LY-M001, a gene therapy using an rAAV8 vector, which aims to deliver the GCase protein to liver cells after a single intravenous infusion to address this enzyme deficiency. This clinical trial includes two phases Phase I is a dose escalation study with three dose groups, starting at a lower dose and increasing to higher doses to assess safety. Participants receive a single intravenous infusion of LY-M001 at one of the specified doses. Phase II is a dose expansion study where the recommended dose from Phase I is given to more participants to further evaluate safety and efficacy. The study has a main period of 52 weeks following infusion and a long-term follow-up phase lasting from 53 weeks up to 5 years. Participants will undergo regular assessments including monitoring for adverse events, liver function tests, blood enzyme activity, blood counts, imaging of liver and spleen volumes, bone mineral density, and bone marrow evaluation. Safety evaluations also include electrocardiograms, vital signs, and laboratory tests. The study aims to track the effects of LY-M001 over time, with extended follow-up to gather long-term data on safety and treatment impact throughout the study duration.

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