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.
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¿De qué trata este estudio?
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.
Puntos clave
- 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.
- Puede retirarse del estudio en cualquier momento.
¿Quién puede ser elegible?
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.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- ¿Tienes 18 años o más?
- 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?
¿En qué consiste la participación?
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.
Riesgos y beneficios potenciales
Locations (9)
- Cedars-SinaiCódigo postal verificadoLos Angeles, United States· Reclutamiento
- Ann and Robert H Lurie Children's Hospital of ChicagoCódigo postal verificadoChicago, Estados Unidos· Reclutamiento
- Duke University Health SystemCódigo postal verificadoDurham, Estados Unidos· Reclutamiento
- Lysosomal & Rare Disorders Research and Treatment CenterCódigo postal verificadoFairfax, United States· Reclutamiento
- Westmead Hospital-Cnr Hawkesbury and Darcy RdsCódigo postal verificadoWestmead, Australia· Completado
- Hospital de Clinicas de Porto Alegre (HCPA)Código postal verificadoPorto Alegre, Brasil· Reclutamiento
- SphinCS Clinical Science for LSDCódigo postal verificadoHöchheim, Germany· Reclutamiento
- Hospital Quironsalud Zaragoza, Paseo Mariano Renovales SnCódigo postal verificadoZaragoza, Spain· Reclutamiento
- Royal Free Hospital NHS TrustCódigo postal verificadoLondres, Reino Unido· Reclutamiento
Preguntas comunes
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.
¿Puedo dejar de participar en el estudio en cualquier momento?
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.
Cómo obtener más información
Prevail Therapeutics
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
¿Interesado en participar?
Discusión
Discusión comunitaria
Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.