A Single Arm, Open Label, Phase 1/2 Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients with Sickle Cell Disease
This study is looking into a new medication, etavopivat, for children and teenagers affected by sickle cell disease. The main goals are to understand how safe the medicine is for young patients and how their bodies handle it. Researchers will also be checking to see if etavopivat can help improve their condition, for example, by increasing the number of healthy red blood cells the body produces and reducing painful sickle cell crises. This is a crucial step to see if this new treatment could be a good option for managing sickle cell disease in younger individuals. The study will carefully monitor participants for any side effects and track their progress over time.
At a glance
What is this study about?
Sickle cell disease is a lifelong condition that affects red blood cells, which carry oxygen around the body. In sickle cell disease, these cells can become stiff and sickle-shaped, leading to blockages in blood vessels, pain, and other serious health problems. Finding new and better treatments is really important, especially for children and young people.
This study is testing a new medicine called etavopivat. It's designed to help red blood cells work better and be less likely to form the damaging sickle shape. Researchers want to understand two main things: firstly, how safe this medicine is for children and teenagers, and secondly, how their bodies process the medicine. They will check how much medicine stays in the blood and for how long.
The study will also look at whether etavopivat helps improve important aspects of health for those with sickle cell disease. This includes seeing if it increases healthy red blood cells (measured by something called haemoglobin), reduces how often painful sickle cell crises happen, and improves how tired patients feel. This research is a vital step in trying to find effective new ways to manage sickle cell disease in younger patients.
Key takeaways
- This study is for children and young people with sickle cell disease.
- It tests a new medicine called etavopivat to see if it's safe and helpful.
- The goal is to improve red blood cells and reduce painful crises.
- Participation involves regular clinic visits for blood tests and check-ups.
- You can stop participating at any time.
- The study lasts for at least 24 weeks.
Who may be eligible?
This study is for children and young people with sickle cell disease. To be considered, you will have specific types of sickle cell disease, such as sickle cell anaemia, or certain variations like sickle beta-thalassaemia. You will need to be well enough to take part and not have other serious health problems that might make the study unsafe for you.
There might be other health conditions or medications that would prevent someone from joining, as these could interfere with the study medicine or your safety. The study team will review your full medical history carefully to make sure it's safe for you to participate.
Importantly, there are no age limits mentioned for children, meaning it could include a wide range of young people. Both boys and girls can 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.
- Do I have a confirmed diagnosis of sickle cell disease (e.g., sickle cell anaemia or certain beta-thalassaemia types)?
- Am I a child or young person under the care of a doctor for my sickle cell disease?
- Am I generally well enough to participate in a medical study?
- Do I have any other serious health conditions that might make taking a new medicine risky?
- Am I currently taking any medications that might interact with a new treatment?
What does participation involve?
If you decide to take part, you'll be given the study medicine, etavopivat, to take. The study team will need to see you regularly for appointments. These appointments will involve check-ups, taking blood samples to see how the medicine is working and how your body is handling it, and asking about any side effects you might be experiencing. They will also measure your haemoglobin levels and ask about any painful sickle cell crises you might have.
Some appointments will involve special scans, like a transcranial Doppler ultrasound, which is a simple and painless way to look at blood flow in the brain. They will also ask you to fill out questionnaires about how you're feeling, especially regarding tiredness. The study will last for a primary treatment period of 24 weeks, but there might be an extension period afterwards where you continue to receive the medicine and be monitored. Throughout your participation, the study team will be there to support you and answer any questions.
Potential risks and benefits
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Common questions
What is sickle cell disease?
It's a condition where red blood cells, which carry oxygen, become stiff and C-shaped, leading to pain and other health issues.
What is etavopivat?
It's a new medicine being tested to help red blood cells work better in people with sickle cell disease.
Will I have to take daily medicine if I join?
Yes, you will take the study medicine, etavopivat, as prescribed by the study team.
What kind of tests will I have?
You'll have blood tests, physical exams, and possibly scans like a transcranial Doppler ultrasound, plus questionnaires about how you feel.
How long will the study last?
The main part of the study is 24 weeks, but there might be an option to continue for a longer period.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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