A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
This research study is investigating a new medication called Etavopivat for people living with sickle cell disease or thalassaemia. Both conditions are inherited blood disorders that affect how red blood cells carry oxygen around the body. Etavopivat aims to improve this process. This particular study focuses on gathering long-term information about the safety and effectiveness of Etavopivat. Participants will be those who are already taking Etavopivat in another study and who have shown benefit from the treatment. The study could last for several years, up to 264 weeks, but may finish earlier if the medicine gets approval for use in a participant's country. The aim is to help us understand if Etavopivat can offer a safe and lasting treatment option.
At a glance
What is this study about?
This study is about a new medication named Etavopivat, which is being developed to help people with sickle cell disease and thalassaemia. These are genetic conditions that affect a substance in your red blood cells called haemoglobin. Haemoglobin is really important because it’s responsible for carrying oxygen from your lungs to all parts of your body. In sickle cell and thalassaemia, the haemoglobin doesn’t work as it should, leading to various health problems.
Researchers want to see how safe Etavopivat is and how well it continues to work over a long period of time. This is especially for people who have already been taking Etavopivat in a previous study and have experienced some benefits from it. By continuing to monitor these participants, the study aims to gather important information that will help researchers understand the medication's long-term effects and whether it could become a widely available treatment.
The information collected will help doctors and scientists understand if Etavopivat can provide a lasting solution for managing these conditions, potentially improving the quality of life for many people. The study is designed to be thorough and careful, ensuring that participants' safety and well-being are a top priority throughout the process.
Key takeaways
- This study is for people already taking Etavopivat for sickle cell disease or thalassaemia.
- It aims to understand the long-term safety and effectiveness of the existing treatment.
- Participation could last up to five years, but may end sooner if the drug is approved.
- Regular health checks will be part of taking part.
- Your safety and well-being are the top priority throughout the study.
Who may be eligible?
To join this study, you must already be taking part in an earlier study for Etavopivat for either sickle cell disease or thalassaemia, and have completed at least one treatment period in that original study. Importantly, the doctors overseeing the study must also believe that you have been benefiting from the Etavopivat treatment.
If you're currently taking other medications for your condition, such as hydroxyurea, crizanlizumab, or L-glutamine (Endari®), you might still be able to join. However, it's crucial that you have been on a steady dose of these medications for a while in your previous study. Small adjustments to your dose that are due to changes in your weight, age, or temporary medical reasons, won't necessarily stop you from being included, as long as your doctor believes your dose is stable overall.
However, some situations would mean you cannot take part. These include if you had to stop Etavopivat permanently in a previous study, or if you needed a permanent reduction in your dose for more than 28 days. Also, if you have any other health condition that the study doctors believe could make your participation unsafe or difficult to manage within the study's rules, you would not be able to join. Your doctors will discuss all these points with you carefully.
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 currently in an earlier Etavopivat study for sickle cell disease or thalassaemia?
- Have your doctors noticed you've been benefiting from Etavopivat treatment?
- Are you able to attend regular check-ups and appointments?
- Are you generally healthy, apart from your sickle cell disease or thalassaemia?
What does participation involve?
As this is a long-term study, it will involve regular check-ups and assessments to monitor your health and how you are responding to Etavopivat. The specific details of visits and tests will be explained by the study team, but generally, they will include blood tests, physical examinations, and discussions about your well-being. You will continue to receive Etavopivat as part of the study. The study is planned to run for up to 264 weeks (which is about five years), but it might finish sooner if Etavopivat receives approval for use in your country. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.
Potential risks and benefits
Locations (103)
- Univ of Alabama BirminghamVerified postcodeBirmingham, United States· Active not recruiting
- Phoenix Children's HsptlVerified postcodePhoenix, United States· Recruiting
- Children's Hospital Los Angeles - EndocrinologyVerified postcodeLos Angeles, United States· Active not recruiting
- Children's Hospital Los Angeles - EndocrinologyVerified postcodeLos Angeles, United States· Recruiting
- UCSF Oakland Benioff ChildHospVerified postcodeOakland, United States· Active not recruiting
- UCSF Oakland Benioff ChildHospVerified postcodeOakland, United States· Recruiting
- Children's Hosp Of OrangeVerified postcodeOrange, United States· Completed
- University Of California IrvineVerified postcodeOrange, United States· Recruiting
- University of ConnecticutVerified postcodeFarmington, United States· Active not recruiting
- Children's National Medical CenterVerified postcodeWashington D.C., United States· Completed
- Foundation for Sickle Cell Disease ResearchVerified postcodeHollywood, United States· Recruiting
- Univ of Miami/SCCCVerified postcodeMiami, United States· Completed
Common questions
What is Etavopivat?
Etavopivat is a new medicine being developed to help people with sickle cell disease and thalassaemia by improving how red blood cells carry oxygen.
How long will this study last?
The study could last for up to 264 weeks (around five years), but it might end earlier if Etavopivat becomes approved for use in the UK.
Will I receive any new treatment?
You will continue to receive the Etavopivat treatment that you have already started in a previous study.
Can I take part if I'm on other medications?
Possibly, especially if you're on a stable dose of medications like hydroxyurea. The study doctor will review all your medications.
What if I want to stop participating?
You are free to leave the study at any time, for any reason, and your medical care will not be affected.
How to find out more
Novo Nordisk
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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