A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
This study is testing a new medicine called etavopivat for people living with sickle cell disease. The main goal is to find out if etavopivat can reduce the number of painful events called vaso-occlusive crises (VOCs), which happen when blood vessels get blocked. We also want to see if this medicine can lessen damage to organs, help people exercise more easily, and reduce feelings of tiredness. Half of the participants will receive etavopivat, and the other half will receive a 'dummy pill' (placebo). Who gets which treatment is decided by chance. This study is expected to last for about two years and is part of the ongoing research into etavopivat.
At a glance
What is this study about?
This study is investigating a new medicine called etavopivat for people with sickle cell disease. Sickle cell disease is a lifelong condition that can cause severe pain and other serious health problems. These painful episodes are often called 'crises' or Vaso-occlusive crises (VOCs). They happen when the sickle-shaped red blood cells block small blood vessels, stopping blood from reaching parts of the body. This study wants to see if etavopivat can help reduce how often these painful crises occur.
Beyond reducing pain crises, the research team is also looking at other important aspects of living with sickle cell disease. They want to understand if etavopivat can help protect different organs in the body from the damage that can happen over time with this condition. They will also check if the medicine improves how well people can exercise and whether it reduces the common feeling of extreme tiredness, known as fatigue, that many people with sickle cell disease experience.
Overall, the aim is to confirm if etavopivat is a safe and effective treatment that could improve the lives of adults and teenagers with sickle cell disease. The study will compare etavopivat to a 'placebo', which is a dummy medicine with no active ingredients, to clearly see the effects of the new treatment.
Key takeaways
- This study is testing a new medicine called etavopivat for sickle cell disease.
- It aims to reduce painful crises, protect organs, and lessen tiredness.
- Participants will receive either etavopivat or a dummy pill (placebo) by chance.
- The study involves regular clinic visits and health checks over about two years.
- If you have sickle cell and are aged 12 or older, you might be able to join.
- You can stop participating at any time without affecting your medical care.
Who may be eligible?
To join this study, you need to be at least 12 years old, male or female, and have been diagnosed with sickle cell disease. This diagnosis needs to be confirmed by past medical tests like a blood test (haemoglobin electrophoresis or HPLC). You must also have experienced between 1 and 15 painful sickle cell crises (VOCs) in the 12 months before the study starts. These crises need to be recorded in your medical notes, not just remembered.
Your blood test results at the start of the study (called 'screening') also need to show that your haemoglobin levels are somewhere between 5.0 and 10.0 grams per decilitre. This helps ensure that the medicine is being tested in people who fit certain medical criteria for the study.
There are also some reasons why you couldn't take part. For example, if you've had more than 15 painful crises in the last year, or if you're currently having regular blood transfusions. Also, if you use certain other medicines for sickle cell disease, like voxelotor or crizanlizumab, you might not be able to join, or you might need to stop them a certain amount of time before starting the study.
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 12 years old or older?
- Do you have a confirmed diagnosis of sickle cell disease?
- Have you had between 1 and 15 painful sickle cell crises (VOCs) in the last year, documented by a doctor?
- Are you NOT currently receiving regular, planned blood transfusions?
- Are you NOT currently taking certain other specific sickle cell medicines?
- Is your haemoglobin level, as measured by a doctor, between 5.0 and 10.0 g/dL?
What does participation involve?
If you decide to take part in this study, you'll either receive the new medicine, etavopivat, or a dummy pill (placebo). Which one you get will be decided by chance, like flipping a coin. You won't know which one you're receiving, and neither will your study doctor. This is to make sure the results are fair.
Throughout the study, you'll have regular visits to the clinic where the study team will monitor your health, ask about your symptoms (like pain crises and tiredness), and perform tests. These tests might include blood samples and checks on how well your organs are working. They'll also check your exercise ability. The total study duration is expected to be about two years, involving ongoing treatment and follow-up during this time.
Potential risks and benefits
Locations (172)
- Uni of Alabama at BirminghamVerified postcodeBirmingham, United States· Recruiting
- Univer South Alabama Ped/OncVerified postcodeMobile, United States· Recruiting
- Phoenix Children's HsptlVerified postcodePhoenix, United States· Recruiting
- Arkansas Children's HospitalVerified postcodeLittle Rock, United States· Recruiting
- Children's Hospital Los Angeles - EndocrinologyVerified postcodeLos Angeles, United States· Recruiting
- UCLA HealthVerified postcodeLos Angeles, United States· Recruiting
- Valley Children's HospitalVerified postcodeMadera, United States· Recruiting
- University Of California IrvineVerified postcodeOrange, United States· Completed
- Stanford University_Palo AltoVerified postcodePalo Alto, United States· Recruiting
- Harbor-UCLA Medical CenterVerified postcodeTorrance, United States· Recruiting
- Clinical and Transl Res CenterVerified postcodeAurora, United States· Completed
- Nemours/AI duPont Hosp-ChldVerified postcodeWilmington, United States· Recruiting
Common questions
What is etavopivat?
Etavopivat is a new medicine being tested to see if it helps people with sickle cell disease by possibly reducing painful crises and improving other symptoms.
What is a 'placebo'?
A placebo is a 'dummy' pill that looks like the study medicine but doesn't contain any active ingredients. It's used to compare the real medicine's effects.
How long will the study last?
The study is expected to last for about two years for each participant.
Will I know if I'm getting the real medicine or the placebo?
No, neither you nor your study doctor will know whether you are receiving etavopivat or the placebo. This helps ensure fair and accurate study results.
Will taking part in this study change my regular medical care?
Participating in the study shouldn't stop you from receiving your usual care, but some treatments might need to be paused or adjusted during the study. This will be discussed in detail.
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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