Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

This study is investigating a new tablet called Venglustat for people aged 16 and older who have Fabry disease. The main goal is to see if Venglustat can ease nerve pain in the arms and legs, and tummy pain, compared to a placebo (a dummy tablet with no medicine). We are looking for people who haven't been treated for Fabry disease for at least six months, or who have never been treated before. Participants will take either Venglustat or the placebo for 12 months. After this, everyone will have the chance to take Venglustat in an open-label extension part of the study. Regular check-ups will happen about every three months to see how you are doing.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Sanofi

Enrolment target

122

Start

11 Mar 2022

Estimated completion

04 Jan 2027

Fabry Disease

What is this study about?

This study is focusing on Fabry disease, a rare genetic condition that can cause various health problems, including significant pain. Specifically, we are looking at nerve pain in the arms and legs (neuropathic pain) and tummy pain (abdominal pain).

We are testing a new medicine called Venglustat, which comes in tablet form. The main purpose of this research is to find out if Venglustat can help reduce these painful symptoms in people with Fabry disease. We compare it to a 'placebo', which looks exactly like the study medicine but doesn't contain any active drug. This helps us understand if the new medicine is truly effective.

This is a 'Phase 3' study, which means it's one of the final steps before a new medicine might become widely available. Doctors and scientists are hopeful that Venglustat could offer a new way to manage these challenging pain symptoms for people living with Fabry disease.

Key takeaways

Tests a new tablet (Venglustat) for Fabry disease pain.
Compares Venglustat to a dummy tablet (placebo) first.
Open to people aged 16+ who haven't recently been treated for Fabry.
Involves regular check-ups (every 3 months) for up to 4 years.
You can stop participating at any time.
Your health will be closely monitored throughout.

Who may be eligible?

You might be able to join this study if you are 16 years old or older and have a confirmed diagnosis of Fabry disease with symptoms. We are looking for people who haven't received treatment for Fabry disease in the last six months, or who have never been treated for it before. A key part of joining is having an average pain score of 3 or more (on a scale of 0 to 10, where 10 is the worst pain imaginable) for either nerve pain in your arms or legs, or tummy pain.

However, there are reasons why you might not be able to take part. For example, if you have certain other serious health conditions like a recent heart attack, stroke, or uncontrolled high blood pressure. You also wouldn't be able to join if you have certain infections like Hepatitis C, HIV, or Hepatitis B, or if your pain is known to be caused by something other than Fabry disease.

If you're thinking about joining, it's important that you or your partner (if you are a man) are using effective birth control methods, and women should not be pregnant or breastfeeding. We also need to check your kidney function and other health markers to make sure it's safe for you to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 16 years old or older?
Do you have a diagnosis of Fabry disease with symptoms?
Have you not received Fabry disease treatment for at least the past 6 months (or ever)?
Do you experience nerve pain in your arms/legs or tummy pain, scoring 3 or more out of 10?
Are you able to use effective birth control if required?
Do you generally have good health apart from your Fabry disease?

Answer every question to see your result.

What does participation involve?

If you decide to join, the study will have two main parts. For the first 12 months, you will take either the study medicine (Venglustat) or a dummy tablet (placebo). Neither you nor your study doctor will know which one you are taking – this is called 'double-blind'. During this time, you'll have study visits about every three months to check on your health and how you're feeling.

After these 12 months, if you wish to continue and have completed the first part, you will enter an 'open-label' period. This means everyone will receive Venglustat, and both you and your doctor will know you're taking the active medicine. This part could last for another 12 months, or potentially longer, until all participants finish the study at the same time. The total time you could be in the study, including initial checks and follow-up, could be up to about 46 months (nearly 4 years). Throughout, there will be regular checks to make sure you are safe and that the medicine is well tolerated.

Potential risks and benefits

Taking part in this study could offer you potential benefits, such as receiving a new investigational medicine that might improve your Fabry disease symptoms. You will also get close medical monitoring and care during the study. However, like all medicines, Venglustat may have side effects, and some people may experience no improvement or even worsening of symptoms. You might also receive the placebo during the first 12 months, meaning you wouldn’t be getting active treatment. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (58)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011
Verified postcode
Birmingham, United States
UCLA Medical Center_Investigational Site Number: 8400006
Verified postcode
Los Angeles, United States
University of California Irvine Medical Center- Site Number : 8400019
Verified postcode
Orange, United States
Advent Health Orlando_Investigational Site Number: 8400008
Verified postcode
Orlando, United States
Emory Genetics- Site Number : 8400010
Verified postcode
Atlanta, United States
Westchester Medical Center Healthcare Corporation- Site Number : 8400001
Verified postcode
Hawthorne, United States
Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400013
Verified postcode
Cincinnati, United States
Cleveland Clinic Site Number : 8400016
Verified postcode
Cleveland, United States
Children's Hospital Of Pittsburgh- Site Number : 8400009
Verified postcode
Pittsburgh, United States
Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012
Verified postcode
Dallas, United States
University Of Utah Health Sciences Center- Site Number : 8400005
Verified postcode
Salt Lake City, United States
Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004
Verified postcode
Fairfax, United States

Common questions

What is Fabry disease?

Fabry disease is a rare genetic condition that can affect many parts of the body, including the nerves, kidneys, and heart, often causing pain and other symptoms.

What does 'placebo' mean?

A placebo is a dummy tablet that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers compare the new medicine's effects.

How long will I be in the study?

The first main part of the study is 12 months. If you continue, the study could last up to about 46 months (nearly 4 years) in total.

Will I know if I'm getting the active medicine?

For the first 12 months, neither you nor your doctor will know if you're taking Venglustat or the placebo. After that, if you continue, you will know you are taking Venglustat.

What kind of pain is the study looking at?

This study is focused on nerve pain in the arms and legs, and tummy pain, that is related to your Fabry disease.