A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

The main purpose of this study is to evaluate the safety and tolerability of LY3884961 (PR001) in adult participants with Type 1 Gaucher Disease.

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Trial Summary

Age Range
18 - 65 years
Conditions the trial is for
Gaucher Disease, Type 1
What the trial is testing?
LY3884961, an investigational gene therapy
Could I receive a Placebo?
Enrollment Goal
Trial Dates
Dec 20, 2022 - Oct 2030
How long will I be in the trial?
5 years (1.5 years for the main study period and 3.5 years for the follow-up period)
Trial Phase

Key Requirements

Clinical trials have inclusion and exclusion requirements to determine which individuals qualify for participation. Listed below are key requirements for this clinical trial, but there may be additional requirements to qualify.

Participants Must:

  • Be 18 to 65 years old

  • Have bi-allelic GBA1 gene mutations.  If you do not know your genetic status, centralized testing will be performed

  • On Enzyme Replacement Therapy or Substrate Reduction Therapy

Participants Must Not:

  • Have had complete spleen removal

  • Have a history of significant liver disease

  • Have serious illnesses that could interfere with this study (will be assessed by the investigator)

  • Have taken certain medicines or had certain treatments, such as any type of prior gene or cell therapy

  • Become pregnant or impregnate others during the study

  • Have problems undergoing an MRI

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Clinical Trial Resources

Clinical Trial Participation: What to Expect

A clinical trial is a scientific study. It helps us learn if a new medicine or treatment works and how it works. Clinical trials can also be called clinical research studies. Learn more about clinical research studies and what to expect when taking part in a clinical trial.