Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A 4-part, open-label, multicenter, multinational study of the safety, tolerability, pharmacokinetics, pharmacodynamic, and exploratory efficacy of venglustat in combination with Cerezyme in adult patients with Gaucher disease Type 3 with venglustat monotherapy extension

This research study is for adults who have Gaucher disease, specifically Type 3, and also includes those with Type 1. It's testing a new medicine called venglustat. The study aims to understand if venglustat is safe to use and how well people tolerate it. Researchers will also look at how the body processes the medicine and if it might improve symptoms of Gaucher disease. For some parts of the study, venglustat will be given alongside an existing treatment called Cerezyme, and then also on its own. This is an early-stage study, meaning it's one of the first times this new medicine is being tested in a larger group of people.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Genzyme Corp.

Enrolment target

Start

08 Dec 2023

Gaucher disease type 3 Gaucher disease type 1

What is this study about?

This study is important for adults living with Gaucher disease, especially those with Type 3, but also including Type 1. Gaucher disease is a rare condition where certain fatty substances build up in the body’s cells, which can cause various health problems. Currently, treatments often involve replacing a missing enzyme in the body.

This research is looking at a new medicine called venglustat. The main goal is to find out if this new medicine is safe and if people can take it without too many unwanted effects. It will also help doctors understand how the medicine moves through the body and how it might affect the disease process. Researchers will be comparing venglustat when given with a current treatment (Cerezyme) and also seeing how it works on its own.

This is a Phase II study, which means it’s an early step in finding new treatments. It's about exploring whether the medicine could be helpful before moving on to larger studies. By taking part, people could help researchers learn more about venglustat and its potential to help others with Gaucher disease in the future.

Key takeaways

This study is for adults with Gaucher disease, primarily Type 3.
It's testing a new medicine called venglustat, either alone or with an existing treatment.
The main goals are to check safety and see how the body handles the medicine.
Participation involves regular clinic visits for monitoring and tests.
Your health and safety will be closely watched by the study team.

Who may be eligible?

This study is for adults who are 18 years old or older. Both men and women can take part.

You would need to have a diagnosis of Gaucher disease Type 3. The study also includes some people with Gaucher disease Type 1.

The research team will have a full list of things they check to make sure the study is safe and right for you. They will explain all the specific requirements if you are interested in joining.

Quick self-check

Are you 18 years old or older?
Do you have a diagnosis of Gaucher disease Type 3?
Are you comfortable with regular hospital visits and medical tests?
Are you interested in potentially trying a new treatment?
Are you able to commit to the study's requirements for its duration?

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

What does participation involve?

If you decide to join this study, you would be taking either the new medicine, venglustat, on its own or in combination with an existing treatment called Cerezyme. The study is split into four parts, which means the treatment plan might change over time, but you will always be carefully monitored.

You would need to attend regular appointments at a clinic or hospital. During these visits, doctors and nurses would check your health, ask about any side effects, and take blood and possibly other samples (like from your spinal fluid, if appropriate for your treatment plan). They will also measure things like your spleen and liver size, and your blood counts to see how the medicine is affecting your body. The total length of your participation would be explained by the study team.

Potential risks and benefits

Taking part in any study has potential benefits and risks. You might not directly benefit from the new medicine, but the information gained could help others with Gaucher disease in the future. As a new medicine, venglustat may have side effects that are not yet fully known; the study specifically tracks these carefully. The researchers are committed to your safety and will monitor you closely. You are always free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

—
Germany

Common questions

What is Gaucher disease?

Gaucher disease is a rare genetic condition where certain fatty substances build up in your body, which can affect organs like your spleen, liver, and bones.

What does 'Phase II' mean?

Phase II studies are early trials focused on checking if a new medicine is safe, finding a good dose, and seeing if it shows signs of helping with a condition.

What is venglustat?

Venglustat is a new medicine being tested. It's meant to reduce the amount of the fatty substances that cause Gaucher disease.

Will I get the new medicine or an existing one?

The study involves different parts; you might receive venglustat on its own, or venglustat combined with an existing treatment called Cerezyme. The study team will explain your specific treatment plan.

What are 'side effects'?

Side effects are any unwanted or unexpected effects that a medicine might cause. The study will carefully monitor and record these.