A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Researchers are conducting a study to see if a new tablet, venglustat, can help people with Fabry disease. This condition can sometimes cause the heart muscle to become thicker than it should be. The study will compare venglustat with existing treatments for Fabry disease, such as agalsidase alfa, agalsidase beta, or migalastat, to see how it affects this heart thickening. The main goal is to find out if venglustat can reduce heart thickness in adults aged 18 to 65 who have Fabry disease and a thickened heart.
At a glance
What is this study about?
This study is looking into a new tablet medicine called venglustat for people who have Fabry disease. Fabry disease is a rare condition that can affect many parts of the body, and for some people, it can cause the walls of the heart to become thicker than normal. This thickening is called 'left ventricular hypertrophy' and it can make it harder for the heart to pump blood effectively.
The main aim of this study is to find out if taking venglustat tablets can help to reduce this heart thickening. Researchers will be comparing venglustat with the treatments that are currently available for Fabry disease in the UK, such as agalsidase alfa, agalsidase beta, or migalastat. This will help them understand if venglustat could be a new and effective way to manage heart problems linked to Fabry disease.
Taking part would mean you'd be helping doctors learn more about Fabry disease and how to treat it. The information gathered from this study could lead to new treatment options for people living with this condition in the future.
Key takeaways
- This study evaluates venglustat, a new tablet for Fabry disease.
- It aims to see if venglustat can reduce heart wall thickening.
- It lasts about 18 months, with an option to extend for several years.
- Compares venglustat to existing Fabry disease treatments.
- Regular hospital visits for health checks and heart assessments.
Who may be eligible?
To join this study, you need to be an adult between 18 and 65 years old. You must have a confirmed diagnosis of Fabry disease and have experienced symptoms of the condition. Importantly, you also need to have a thickened heart wall, a condition known as left ventricular hypertrophy. You might already be on treatment for Fabry disease, or you might not be – either way, you could still be considered for the study.
There are certain health conditions that would prevent you from taking part. For example, if you've had a stroke, heart attack, or major heart surgery, or if you have severe kidney problems, you wouldn't be able to join. Certain severe heart rhythm problems, some infections like hepatitis or HIV, and a recent history of drug or alcohol abuse also mean you wouldn't be eligible. The study also can't include people with specific advanced heart damage or other serious health issues that might affect the study results or your safety.
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 between 18 and 65 years old?
- Do you have a confirmed diagnosis of Fabry disease?
- Do you have a condition where your heart muscle is thicker than normal?
- Have you had recent major heart events (e.g., stroke, heart attack, surgery)? (If yes, you may not be eligible)
- Do you have severe kidney or liver problems, or certain infections (e.g., HIV, hepatitis)? (If yes, you may not be eligible)
What does participation involve?
If you decide to take part, you'll first go through a screening period to check if the study is right for you. If you qualify, you'll be randomly assigned to either receive the new venglustat tablet or one of the existing Fabry disease treatments. This initial part of the study will last for about 18 months.
Throughout this time, you'll need to attend study visits at a hospital or clinic approximately every 3 to 6 months. These visits will involve various checks to see how you're feeling and how the treatment is working, including tests to measure your heart thickness. After the initial 18 months, if you wish and if you meet certain criteria, you might be able to continue receiving venglustat in a 'long-term extension' part of the study for up to an additional 45 months. This means the total time you could be involved in the study could be up to 5.3 years.
Potential risks and benefits
Locations (54)
- University of Alabama -The Kirklin Clinic- Site Number : 8400010Verified postcodeBirmingham, United States
- University of California Los Angeles Medical Center- Site Number : 8400008Verified postcodeLos Angeles, United States
- Emory University School of Medicine - Atlanta- Site Number : 8400009Verified postcodeAtlanta, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005Verified postcodeChicago, United States
- Maryam Banikazemi, MD- Site Number : 8400001Verified postcodeHawthorne, United States
- Renal Disease Research Institute- Site Number : 8400012Verified postcodeDallas, United States
- University of Utah Health Hospital- Site Number : 8400006Verified postcodeSalt Lake City, United States
- Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004Verified postcodeFairfax, United States
- Investigational Site Number : 0400001Verified postcodeGraz, Austria
- Investigational Site Number : 1240003Verified postcodeCalgary, Canada
- Investigational Site Number : 1240006Verified postcodeEdmonton, Canada
- Investigational Site Number : 1240002Verified postcodeVancouver, Canada
Common questions
What is the main goal of this study?
The main goal is to see if a new tablet, venglustat, can reduce heart thickening in adults with Fabry disease, compared to existing treatments.
How long would I need to be in the study?
The initial part of the study lasts about 18 months, but you could potentially take part for up to 5.3 years if you continue into the long-term extension.
Will I know if I'm getting the new treatment or a standard one?
No, you'll be randomly assigned to either the new treatment (venglustat) or a standard one (agalsidase alfa, agalsidase beta, or migalastat), and neither you nor your doctor will know which you are receiving during the main 18-month part of the study.
What kind of visits will I have?
You'll have hospital or clinic visits approximately every 3 to 6 months for health checks and tests, including heart scans.
Can I leave the study at any time?
Yes, you are free to leave the study at any point, and your decision will not affect your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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