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Contact the study team using the details below to take part. If there are no contact details below please ask your doctor in the first instance.

Contact Information:

Prevail Therapeutics (917) 336-9310
Prevail.Patients@lilly.com


Study Location:

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Be Part of Research - Trial Details - A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

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

Recruiting

Open to: ALL

Age: 18.0 - N/A

Medical Conditions

Gaucher Disease
Gaucher Disease, Type 1


This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the study.


Study J3Z-MC-OJAE is a Phase 1/2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral manifestations of GD.

Up to 3 dose levels of LY3884961 will be assessed in 3 dose-finding cohorts of 3 patients. Following this, up to 6 patients may be enrolled in an expansion cohort.

For each enrolled patient, the study will be approximately 5 years in duration, including up to a 60-day screening period. During the first 18 months after dosing, subjects will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed for an additional 42 months to monitor safety, immunogenicity, and selected biomarker and efficacy parameters.

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

Dec 2022 Nov 2031

INTERVENTIONAL

Intervention Type : GENETIC
Intervention Description : • LY3884961 is a replication-incompetent recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.

Intervention Arm Group : LY3884961;



You can take part if:



You may not be able to take part if:


This is in the inclusion criteria above


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • Royal Free Hospital NHS Trust
    London


The study is sponsored by Prevail Therapeutics and is in collaboration with Eli Lilly and Company.




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Read full details for Trial ID: NCT05487599
Last updated 05 December 2025

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