The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
This research study is testing a new medicine called Rilzabrutinib for people with sickle cell disease who are between 10 and 65 years old. We want to find out if this medicine can help reduce the number of painful episodes, known as sickle cell crises, and if it's safe to use. Some people in the study will receive Rilzabrutinib, while others will get a placebo (a dummy pill with no active medicine). This helps us compare the effects. The study will last for a year, with some participants continuing on the active medicine afterwards. We are hoping to gather important information that could potentially lead to new and better treatments for sickle cell disease.
At a glance
What is this study about?
Sickle cell disease is a lifelong condition that causes your red blood cells to become stiff and crescent-shaped. These cells can block blood flow, leading to severe pain, called sickle cell crises, and other serious health problems. It's a condition that affects many people across the UK, and finding new ways to manage it and improve quality of life is really important.
This study is looking into a new medication called Rilzabrutinib. It's a 'Phase 3' study, which means it's one of the final steps before a new drug might be made available to everyone. We want to see how well Rilzabrutinib works at preventing or reducing the painful crises that people with sickle cell disease experience, and if it's safe to use.
To do this, we'll compare Rilzabrutinib to a 'placebo'. A placebo looks exactly like the study medicine but contains no active drug. This is a standard way to test new medicines and helps us know for sure if any changes people feel or experience are due to the new drug or something else. Neither you nor your study doctor will know whether you are receiving Rilzabrutinib or the placebo during the first part of the study. This helps keep the results fair and unbiased.
Key takeaways
- Tests a new medicine, Rilzabrutinib, for sickle cell disease.
- Aims to reduce painful sickle cell crises.
- Compares Rilzabrutinib to a placebo (dummy pill).
- Study lasts for at least 52 weeks, with potential for longer participation.
- Open to individuals with sickle cell disease aged 10 to 65.
- Regular health check-ups and monitoring by a study team.
Who may be eligible?
To be considered for this study, you must have been diagnosed with sickle cell disease. You should also have experienced between 2 and 10 painful sickle cell crises in the last year. Participants must be aged between 10 and 65 years old.
If you are currently taking other sickle cell medications like hydroxyurea or L-glutamine, you need to have been on a stable dose for at least three months, or not be taking them at all and not planning to during the study. Children and teenagers aged 10 to 17 will need a parent or guardian to give permission for them to join.
There are also some reasons why you might not be able to join. These include having certain types of cancer, serious heart problems, a history of stroke, or some active infections like Hepatitis B or C, or HIV. If you've recently had COVID-19 or are taking a specific medication called crizanlizumab (Adakveo), you also wouldn't be able to participate.
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 you have a confirmed diagnosis of sickle cell disease?
- Have you had between 2 and 10 painful sickle cell crises in the last year?
- Are you between 10 and 65 years old?
- Are you on a stable dose of other sickle cell meds (like hydroxyurea) for at least 3 months, or not taking them?
- Do you have good general health, aside from your sickle cell disease?
What does participation involve?
If you decide to take part in this study, you'll be assigned to receive either the study drug, Rilzabrutinib, or a placebo (a dummy pill). This first part of the study will last for 52 weeks, which is about a year. During this time, you will have regular visits to the clinic so the study team can check on your health, see how you're responding to the treatment, and monitor for any side effects. The exact schedule of these visits will be shared with you.
After completing the first 52-week period, if you wish to continue and meet certain conditions, you might be able to join an 'open-label' extension part of the study. In this part, everyone receives Rilzabrutinib, and you and your doctor will know which medicine you are taking. This extension period could last for another year or longer. The total time you spend in the study will depend on when you join and how long the extension part runs, but it will involve ongoing check-ups and assessments by the study team.
Potential risks and benefits
Locations (54)
- University of Alabama at Birmingham- Site Number : 8400003Verified postcodeBirmingham, United States· Recruiting
- Phoenix Children's Hospital- Site Number : 8400028Verified postcodePhoenix, United States· Recruiting
- University of California San Francisco- Site Number : 8400040Verified postcodeFresno, United States· Recruiting
- Oncology & Hematology Associates of West Broward- Site Number : 8400029Verified postcodeCoral Springs, United States· Recruiting
- Sylvester Comprehensive Cancer Center- Site Number : 8400020Verified postcodeMiami, United States· Recruiting
- University of Illinois-Chicago - College of Medicine- Site Number : 8400054Verified postcodeChicago, United States· Recruiting
- Indiana University Health Riley Hospital for Children- Site Number : 8400056Verified postcodeIndianapolis, United States· Recruiting
- Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037Verified postcodeShreveport, United States· Recruiting
- University of Maryland School of Medicine - Baltimore- Site Number : 8400041Verified postcodeBaltimore, United States· Recruiting
- University of Michigan Health System - Ann Arbor- Site Number : 8400035Verified postcodeAnn Arbor, United States· Recruiting
- Southern Specialty Research- Site Number : 8400059Verified postcodeFlowood, United States· Recruiting
- Richmond University Medical Center- Site Number : 8400038Verified postcodeStaten Island, United States· Recruiting
Common questions
What is sickle cell disease?
Sickle cell disease is a inherited condition where red blood cells become stiff and shaped like crescents. These can block blood flow, causing pain and other health problems.
What is a placebo?
A placebo is a 'dummy pill' that looks like the study medicine but doesn't contain any active drug. It helps us compare the effects of the new medicine fairly.
Will I know if I'm getting the drug or the placebo?
During the first year, neither you nor your study doctor will know if you're getting Rilzabrutinib or the placebo. This is called 'double-blind' and helps keep the study fair.
How long will the study last?
The main part of the study is 52 weeks (about a year). After that, some participants may be able to continue in an open-label part where everyone gets the active drug.
Can children join this study?
Yes, children and teenagers aged 10 to 17 years old can join, but their parents or guardians must give permission.
How to find out more
Trial Transparency email recommended (Toll free for US & Canada)
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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