A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This study, called RISE UP, is testing a new medicine called mitapivat for people with sickle cell disease. It has two main goals: first, to find the best dose of mitapivat, and second, to see how well it works and if it's safe. Specifically, researchers want to know if mitapivat can help increase the amount of a healthy red blood cell chemical (hemoglobin) in the blood and reduce painful episodes called sickle cell crises. Over time, the study will also look at the longer-term effects of this medicine. If you have sickle cell disease, this study could be an opportunity to explore a potential new treatment.
At a glance
What is this study about?
Sickle cell disease is a lifelong condition that affects your red blood cells. Normally, these cells are round and flexible, carrying oxygen around your body. But with sickle cell disease, they can become stiff and C-shaped, like a farm tool called a sickle. These 'sickled' cells can block blood flow, leading to severe pain, called sickle cell crises, and other health problems.
This study is investigating a new medicine called mitapivat. It's designed to help your blood cells work better. The researchers want to find out the best amount of mitapivat to give and how well it helps people with sickle cell disease. They will be looking to see if it can increase healthy red blood cells and reduce the number of painful crises you might experience. It's important to remember that this is a research study, and we're still learning about this medicine.
The study is split into two main parts, called Phase 2 and Phase 3. The first part helps find the right dose. Then, a larger group of people will take part in Phase 3 to get a clearer picture of how effective and safe mitapivat is compared to a dummy pill (placebo). After these main parts, there's an option to continue taking mitapivat for a longer period to see its long-term effects.
Key takeaways
- This study is testing a new medicine called mitapivat for sickle cell disease.
- It aims to find the right dose and see if it helps increase red blood cells and reduce painful crises.
- The study involves taking either mitapivat or a dummy pill (placebo).
- Participation includes regular clinic visits and health checks.
- Potential benefits might include access to a new treatment, but there are also potential side effects.
- You can leave the study at any time without affecting your normal care.
Who may be eligible?
To join this study, you need to be at least 16 years old. (In France and Germany, you must be 18 or older). If you're 16 or 17, you must have finished puberty. You need to have a clear diagnosis of sickle cell disease, including common types like HbSS or HbSC.
Over the past year, you should have experienced at least two, but no more than ten, painful sickle cell crises. Your haemoglobin level (a measure of your red blood cells) needs to be within a specific range. If you're currently taking a medicine called hydroxyurea, your dose must have been stable for at least 3 months. Women who could become pregnant must agree to use two types of birth control during the study.
There are also some reasons you wouldn't be able to join. For example, if you are pregnant or breastfeeding, or if you regularly receive blood transfusions. You would also be excluded if you have certain severe liver or kidney problems, or if you've had a stem cell transplant or gene therapy. Also, if you're taking certain other ongoing sickle cell medicines or investigational treatments, you might not be eligible.
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 16 years or older? (18+ in France/Germany)
- Do you have a diagnosis of sickle cell disease?
- Have you had 2 to 10 painful sickle cell crises in the last year?
- Are you able to use two forms of contraception if you could become pregnant?
- Are you NOT pregnant or breastfeeding?
- Are you NOT regularly having blood transfusions?
What does participation involve?
If you decide to take part, the study will involve several visits to a clinic. You'll be given either mitapivat or a dummy pill (placebo) to take. Initially, the study will involve different doses of mitapivat being tested in smaller groups. Then, in the main part, you'll either take the recommended dose of mitapivat or a placebo for about 52 weeks (roughly a year). This will be decided by chance, like flipping a coin. You won't know if you're getting the active medicine or the placebo, and neither will your local study doctor.
Throughout the study, you'll have regular checks, which might include blood tests, physical exams, and discussions about how you're feeling and any symptoms you have. The study team will explain all the procedures in detail. After the main part of the study, you might have the option to continue taking mitapivat for up to 216 weeks (over 4 years) in an open-label part, meaning everyone in this part would receive mitapivat.
Potential risks and benefits
Locations (91)
- University of California San DiegoVerified postcodeLa Jolla, United States
- UCLA HealthVerified postcodeLos Angeles, United States
- Children's Hospital OaklandVerified postcodeOakland, United States
- University of Connecticut Health CenterVerified postcodeFarmington, United States
- Children's National HospitalVerified postcodeWashington D.C., United States
- MedStar Washington Hospital CenterVerified postcodeWashington D.C., United States
- Sylvester Comprehensive Cancer Center-MiamiVerified postcodeMiami, United States
- University of Chicago Medical CenterVerified postcodeChicago, United States
- Riley Hospital For ChildrenVerified postcodeIndianapolis, United States
- LSU Health Sciences Center - ShreveportVerified postcodeShreveport, United States
- National Heart Lung and Blood InstituteVerified postcodeBethesda, United States
- Kaiser Permanente - Largo Medical CenterVerified postcodeLargo, United States
Common questions
What is sickle cell disease?
It's a blood condition where red blood cells become stiff and C-shaped, causing pain and other health issues.
What is mitapivat?
It's a new medicine being tested to help make red blood cells healthier and reduce pain crises in people with sickle cell disease.
What is a placebo?
A placebo is a dummy pill that looks like the real medicine but contains no active ingredients. It helps researchers see if the real medicine is truly effective.
How long would I need to be in the study?
The main part of the study lasts about a year, with an option to continue for several more years afterwards.
Can I still take my regular medicines?
You might be able to, but some medicines need to be stopped or stable for a certain time before joining. The study team will tell you which ones.
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.