RecruitingPHASE1, PHASE2INTERVENTIONAL

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

This clinical trial, called PROCEED, is looking into a new medicine, LY3884961 (also known as PR001), for adults who have Gaucher disease, type 1, affecting parts of the body other than the brain. It's a Phase 1/2 study, meaning it's in early stages of testing. Researchers want to find the safest and most effective dose of LY3884961. They will first test different low doses in a small group of patients, and then may expand to include more patients. Participants will be monitored closely for about 5 years to understand the medicine's effects, ensuring their safety throughout the study.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Prevail Therapeutics

Enrolment target

Start

20 Dec 2022

Estimated completion

30 Aug 2032

Gaucher Disease Gaucher Disease, Type 1

What is this study about?

Gaucher disease, type 1, is a rare inherited condition. It means your body doesn't produce enough of a specific enzyme, which leads to a build-up of certain fatty substances in your cells and organs, like the spleen, liver, and bones. This can cause various problems, including pain, fatigue, and issues with your blood.

Currently, there are treatments available for Gaucher disease that help manage these symptoms. However, researchers are always looking for new and improved ways to treat the condition. This particular study, called PROCEED, is a clinical trial that aims to test a new investigational medicine, LY3884961. The goal is to see if this new medicine can be a safe and effective treatment option for adults with Gaucher disease, type 1, specifically those whose symptoms affect their body but not their brain.

By participating in this study, you would be helping researchers learn more about Gaucher disease and potentially contribute to finding a new treatment that could benefit many people in the future. The study is carefully designed to ensure participant safety and to gather as much information as possible about the new medicine.

Key takeaways

Tests a new medicine (LY3884961) for type 1 Gaucher disease.
Aims to find a safe and effective dose.
Suitable for adults with body-affecting, not brain-affecting, symptoms.
Participation involves about 5 years of monitoring.
Requires stable current Gaucher disease treatment for at least 3 months.
You can withdraw from the study at any time.

Who may be eligible?

To join this study, you need to be at least 18 years old and have a confirmed diagnosis of Gaucher disease, type 1. You should also have been on a stable dose of your current Gaucher disease treatment (either enzyme replacement therapy or substrate reduction therapy) for at least three months, and on this treatment for at least two years.

There are some reasons why you might not be able to take part. For example, if you have significant brain or nerve problems related to Gaucher disease, or if you have severe bone issues that might need surgery soon, you would not be eligible. Also, if you have severe liver disease, very low blood platelet counts, or very high cholesterol levels, you might not be able to join.

Women and men who could potentially have children must agree to use effective contraception throughout the study. This is to ensure the safety of any potential future pregnancies, as the effects of the new medicine on unborn babies are not yet known. The study team will discuss all exclusion criteria with you in detail.

Quick self-check

Are you 18 years or older?
Do you have confirmed Gaucher disease, type 1?
Have your Gaucher disease symptoms mainly affected your body, not your brain?
Have you been on your current Gaucher disease treatment for at least 2 years, and at a stable dose for at least 3 months?
Are you able to use effective contraception if you or your partner could become pregnant?
Do you have a general good health apart from your Gaucher disease, without certain severe other medical conditions?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, your involvement would last for approximately 5 years. This includes an initial checking period of up to 60 days to make sure you meet all the study requirements. During the first 18 months after you start taking the study medicine, you'll have regular appointments where doctors will monitor your health, look for any side effects, and check how the medicine is affecting your Gaucher disease.

After these initial 18 months, you'll continue to be followed for another 42 months. During this long-term follow-up period, the main focus will be on your safety and continued health. You will have regular check-ups, and the medical team will continue to watch for any side effects and keep an eye on how well the medicine is working. You will need to attend these appointments, which will involve various tests and assessments, as outlined by the study team. You'll also be asked to avoid donating blood or organs for the entire duration of the study.

Potential risks and benefits

Taking part in any clinical trial has potential benefits and risks. The potential benefit of this study is that the new medicine, LY3884961, might improve your Gaucher disease symptoms. However, there's no guarantee it will work for everyone, and it's also possible it might not work at all. As with any new medicine, there's a risk of side effects, which could be mild or more serious. The study team will closely monitor you for any side effects and will explain all known risks before you decide to participate. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (9)

Cedars-Sinai
Los Angeles, United States· Recruiting
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, United States· Recruiting
Duke University Health System
Durham, United States· Recruiting
Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, United States· Recruiting
Westmead Hospital-Cnr Hawkesbury and Darcy Rds
Westmead, Australia· Completed
Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Brazil· Recruiting
SphinCS Clinical Science for LSD
Höchheim, Germany· Recruiting
Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn
Zaragoza, Spain· Recruiting
Royal Free Hospital NHS Trust
London, United Kingdom· Recruiting

Common questions

What is Gaucher disease, type 1?

It's an inherited condition where a specific enzyme is missing, causing fatty substances to build up, mainly affecting organs like the spleen, liver, and bones.

Is this medicine new?

Yes, LY3884961 is an investigational medicine, meaning it's still being studied and is not yet approved for general use.

How long will the study last if I join?

Your participation in the study, including initial checks and follow-up, will be around 5 years in total.

Can I stop participating in the study at any time?

Yes, you can choose to leave the study at any point without it affecting your usual medical care.

Will this medicine affect my brain or nerve problems?

This study specifically focuses on Gaucher disease symptoms affecting the body, not conditions that affect the brain or nervous system.

How to find out more

Prevail Therapeutics

Prevail.Patients@lilly.com (917) 336-9310 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.