PROCEED : A Study of LY3884961 in Gaucher Disease 1
Research type
Research Study
Full title
An Open-label, Dose-Finding, Phase 1/2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Patients with Peripheral Manifestations of Gaucher Disease
IRAS ID
1005513
Contact name
Patricia Sondergaard
Contact email
Sponsor organisation
Prevail Therapeutics, A Wholly Owned Subsidiary of Eli Lilly & Company
Clinicaltrials.gov Identifier
Research summary
Gaucher Disease (GD) is a rare inherited disorder. People with GD are either missing or do not have enough of the enzyme β- glucocerebrosidase (also called GCase), which breaks down certain fats in the body. Enzymes are proteins that speed up a chemical reaction, such as the body breaking down food.
If the body does not have enough GCase, then fatty chemicals build up in organs, mainly in the spleen, liver, and bone marrow, affecting their function, destroying blood cells and weakening bones. GCase is usually low in people who have GD with a GBA1 mutation (a genetic mutation is a change in a gene).
The purpose of the study is to test LY3884961, an investigational gene therapy. Gene therapy is a form of treatment that adds a normal gene or changes an abnormal gene in order to make a normal protein intended to correct a medical condition. LY3884961 is not yet approved for treating GD. It is designed to give patients a normal copy of the GBA1 gene and therefore increase GCase. The new GBA1 gene will remain in the participants body cells for many years and possibly for the rest of their life.
The population for this study will include 15 participants ages 18-50 years who have GD with a GBA1 mutation.
Each participant will take part in the study for approximately 5 years. Study procedures will include but not limited to 12-lead ECG, lung function testing, imaging procedures (DEXA scan, bone and abdominal MRI scans), blood & urine testing and questionnaires.The study is sponsored by Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly & Co.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
22/SC/0394
Date of REC Opinion
11 Jul 2023
REC opinion
Further Information Favourable Opinion