HEMolyse and Organ damage imPROvement in sickle cell disease by VoxElotor. An open-label one stage phase II design.HEMOPROVE
The HEMOPROVE study is investigating a new medication called Oxbryta (500 mg tablets) for adults living with sickle cell disease. This is a Phase II study, meaning researchers are trying to learn more about how well the medicine works and its safety. The main goal is to see if Oxbryta can significantly reduce 'intravascular haemolysis.' This is a process where red blood cells break down inside blood vessels, releasing harmful substances like plasma haemoglobin. If the medicine can drop this harmful haemoglobin by 20% or more over 48 weeks, it would be considered a success. The study will also look at many other aspects of health, including blood flow, brain function, kidney health, and physical activity levels, to understand how Oxbryta might help people with sickle cell disease feel better overall.
At a glance
What is this study about?
This study, called HEMOPROVE, is for adults who have sickle cell disease. Sickle cell disease is a lifelong condition that affects your red blood cells. Normally, red blood cells are round and flexible, carrying oxygen all over your body. But with sickle cell disease, some red blood cells become C-shaped (like a sickle), rigid, and sticky. These sickle cells can block blood flow, leading to pain, organ damage, and a process called 'haemolysis,' where red blood cells break down too quickly in your blood vessels.
The researchers are studying a new medicine called Oxbryta. The main aim of this study is to see if Oxbryta can reduce the breakdown of red blood cells in your blood vessels. They will measure a harmful substance called plasma haemoglobin. If the medicine can lower this by at least 20% over 48 weeks, it would mean it's helping to protect your red blood cells from breaking down.
_Why is this important?_ When red blood cells break down too quickly, it releases substances that can damage your organs, like your kidneys and brain. By hopefully reducing this breakdown, Oxbryta could help improve overall organ health and reduce some of the long-term problems associated with sickle cell disease. The study will also check many other things, like how well your blood flows, how your brain and kidneys are working, and how far you can walk, to get a full picture of the medicine's effects.
Key takeaways
- The study tests a new medicine, Oxbryta, for adults with sickle cell disease.
- It aims to reduce harmful red blood cell breakdown in the body.
- The study measures various health aspects, including brain, kidney, and physical function.
- Participation involves regular clinic visits, blood tests, and scans over 48 weeks.
- Both men and women aged 18 and over can participate if they meet certain health criteria.
Who may be eligible?
This study is open to both men and women who are 18 years old or older. You will need to have a confirmed diagnosis of sickle cell disease to take part.
As this is a medical study, there will be specific health checks and medical history reviews to make sure the study is right for you and safe for you to participate. These checks ensure that the results of the study are accurate and that all participants are treated with the utmost care.
Before you can join, the study team will go through all the requirements with you, answer any questions you might have, and make sure you understand everything involved.
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 18 years old or older?
- Do you have a diagnosis of sickle cell disease?
- Are you able to attend regular clinic appointments for almost a year?
- Are you willing to take study medication (Oxbryta) every day?
- Are you prepared for various health checks and tests, including blood tests and scans?
What does participation involve?
If you decide to take part in this study, you will be given the medicine Oxbryta as 500 mg tablets. This study is 'open-label,' which means both you and the study doctors will know you are receiving the medicine. The study will last for 48 weeks, which is about 11 months.
Throughout these 48 weeks, you will have regular visits to the clinic. At these visits, doctors will measure various things to see how the medicine is affecting you. This includes regular blood tests to check your haemoglobin levels and other markers related to red blood cell breakdown. They will also assess your overall health, including how well your organs are working, your physical activity, and brain function. This might involve special scans like MRI for your brain and kidneys, specific breathing tests, and walking tests.
The study also involves checking for any side effects and observing if other treatments you might be taking, like EPO, can be adjusted. After the 48-week treatment period, there will be follow-up checks to continue monitoring your health and the effects of the medication.
Potential risks and benefits
Locations (1)
- —UnverifiedFrance
Common questions
What is 'intravascular haemolysis'?
It's when red blood cells break down inside your blood vessels, releasing substances that can cause harm to your body.
What is Oxbryta?
Oxbryta is the name of the medicine being tested in this study for people with sickle cell disease.
How long will the study last?
The main treatment part of the study will last for 48 weeks, which is just under a year.
Will I know if I'm getting the study medicine?
Yes, this is an 'open-label' study, meaning you and the study team will know you are receiving Oxbryta.
What does a 'Phase II' study mean?
It means the researchers are trying to learn more about how well the medicine works and its safety in a group of people.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.