An Adaptive, Open-Label, Dose-Finding, Phase 1/2 Study Investigating the Safety, Pharmacokinetics, and Clinical Activity of Rilzabrutinib (PRN1008), an Oral BTK Inhibitor, in Patients with Relapsed Immune Thrombocytopenia
This study is for adults over 18 who have Immune Thrombocytopenia (ITP), a condition where the body doesn't have enough blood platelets, leading to easy bruising and bleeding. Researchers are looking into a new oral medicine called Rilzabrutinib. It's an early-stage study (Phase 1 and 2), which means they are checking its safety, figuring out the best dose, and seeing if it helps patients. They will be monitoring for any side effects and how well the medicine increases and maintains healthy platelet levels. The goal is to find a treatment that can safely and effectively reduce bleeding issues for people with ITP.
At a glance
What is this study about?
This research study is looking at a new medicine called Rilzabrutinib for adults who have a medical condition called Immune Thrombocytopenia, or ITP. If you have ITP, your body's immune system mistakenly attacks and destroys your platelets. Platelets are tiny cells in your blood that help it to clot and stop bleeding. When you don't have enough platelets, you might experience easy bruising, small red spots on your skin called petechiae, gum bleeding, or nosebleeds. This new medicine is given by mouth – it's a tablet or capsule.
The study has two main parts. The first part is about finding the right dose of the medicine. The researchers will start with a small amount and gradually increase it in different groups of people to see what dose works best and is safe. The second part of the study will then use the best dose found in the first part to see how well it helps more people with ITP. They want to know if it can consistently increase and keep platelet counts at a safe level for a longer time.
Throughout the study, the medical team will be closely watching for any side effects the medicine might cause. They also want to understand how the medicine moves through your body and how it affects your platelet count. The main aim is to find a safe and effective way to help people with ITP maintain healthy platelet levels and reduce their bleeding problems.
Key takeaways
- This study investigates a new oral medicine, Rilzabrutinib, for adults with ITP.
- It aims to find a safe and effective dose, and see if it helps increase platelet counts.
- Participation involves taking the medicine and regular clinic visits for blood tests and health checks.
- The study assesses safety (side effects) and how well the medicine works to reduce bleeding.
- Both men and women aged 18 and over with ITP may be eligible.
Who may be eligible?
This study is open to both men and women who are 18 years old or older. You would need to have been diagnosed with Immune Thrombocytopenia (ITP) and have experienced the condition coming back or not getting better with previous treatments.
To be considered for this study, your medical team would need to check your current health and medical history to make sure the study medicine would be safe for you. This involves making sure you don't have other health problems or are taking medicines that might react badly with the study drug.
Ultimately, the medical staff at the study site will be able to tell you if this particular study is a good fit for you based on all your individual circumstances.
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 18 years old or older?
- Have you been diagnosed with Immune Thrombocytopenia (ITP)?
- Has your ITP come back or not fully improved with previous treatments?
- Are you willing to take an oral medication daily?
- Can you attend regular clinic visits and have blood tests?
What does participation involve?
If you decide to take part in this study, you will be given the study medicine, Rilzabrutinib, to take by mouth. The study involves regular visits to the clinic, especially at the beginning, so that the doctors and nurses can closely monitor how you are doing. You will have blood tests during these visits to check your platelet count and to see how the medicine is affecting your body. They will also ask you about any side effects you might be experiencing.
Initially, you might have more frequent visits, and these might become less often as the study progresses. You will also be asked about any bleeding events you experience and how these impact your daily life. The total length of your participation in the study will depend on which part of the study you are in and how you respond to the treatment, but it will involve taking the study medicine regularly and attending follow-up appointments.
Potential risks and benefits
Locations (3)
- —UnverifiedCzechia
- —UnverifiedNetherlands
- —UnverifiedBulgaria
Common questions
What is Immune Thrombocytopenia (ITP)?
ITP is a condition where your body's immune system attacks and destroys its own platelets, which are important for blood clotting. This can cause bruising and bleeding.
What is Rilzabrutinib?
Rilzabrutinib is a new experimental medicine being tested for ITP. It is taken by mouth, like a tablet or capsule.
What does 'Phase 1/2 study' mean?
This means it's an early-stage study. Phase 1 focuses on safety and finding the right dose, while Phase 2 looks at how well the medicine works and continues to check for safety.
How long will I be in the study?
The exact length varies for each person and depends on the study part and your response. The study team will explain the expected duration if you join.
Will I know if I'm getting the actual medicine?
This study is 'open-label', which means both you and the study doctors will know you are receiving Rilzabrutinib. There is no placebo in this study.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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