This study is testing a new intervention called LY3884961 for adults with Gaucher Disease Type 1 who have peripheral manifestations (symptoms affecting organs like the spleen or liver). LY3884961 is a type of gene therapy that uses a modified virus (adeno-associated virus or AAV) to deliver genetic material. The main goal is to see how safe LY3884961 is and if people can tolerate it, by tracking any side effects over 5 years. To join, you must be at least 18 years old, have specific genetic changes (bi-allelic pathogenic GBA1 variants), and have been on stable enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study plans to enroll 15 participants.

Study design

This is a Phase 1/2, open-label study, meaning both you and the study team will know which intervention you are receiving. It will enroll up to 15 participants in different dose groups to find the safest and most effective dose.

What's involved

The study will last approximately 5 years, including a screening period of up to 60 days. You will be evaluated for safety, tolerability, immune responses, and how well the intervention works, especially during the first 18 months after receiving the intervention.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for an additional 42 months after the initial 18-month evaluation period to continue monitoring safety, immune responses, and other effects.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.