Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel-Group Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia (ITP).

Researchers are investigating a new medicine, rilzabrutinib, for adults in the UK living with Immune Thrombocytopenia (ITP). ITP is a condition where the body mistakenly attacks its own platelets, which are essential for blood clotting. This study aims to see if rilzabrutinib can effectively and safely increase platelet counts to a healthy level and reduce bleeding. Participants will receive either the new medicine or a dummy treatment (placebo) for a period, followed by an option to continue with the active treatment. We want to understand how well the medicine works at raising platelet counts and if it improves daily life for people with ITP, while also carefully monitoring for any side effects.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Principia Biopharma Inc.

Enrolment target

Start

04 Apr 2024

Immune Thrombocytopenia (ITP)

What is this study about?

This clinical trial is designed for adults living with Immune Thrombocytopenia (ITP). ITP is a condition where your body's immune system mistakenly attacks and destroys your own platelets. Platelets are tiny cells in your blood that help it to clot. When you have ITP, you have too few platelets, which can lead to easy bruising, bleeding, and fatigue. The study is testing a new medication called rilzabrutinib. It's hoped that this medicine could help your body produce more platelets or stop your immune system from destroying them, ultimately improving your platelet count and overall health.

This is a 'Phase 3' study, which means it's one of the final steps before a new medicine might be approved for wider use. It's a key part of seeing if rilzabrutinib is effective and safe. To make sure results are fair, some participants will receive the active medicine, while others will receive a placebo – a dummy treatment that looks just like the real medicine but contains no active drug. Neither you nor your doctor will know which one you're receiving during the main part of the study. This helps researchers accurately measure the medicine's true effects.

The main goal of this study is to see if rilzabrutinib can help adults with ITP keep their platelet counts at a safe level (50,000 or more per microlitre) for most of a 24-week period, without needing additional emergency treatments for low platelets. Researchers will also be looking at other important factors, including how many weeks participants maintain a good platelet count, how long it takes to reach that count, whether they need urgent platelet-boosting treatments, and how the medicine affects their quality of life and any bleeding symptoms. Safety is always a top priority, so any side effects experienced by participants will be closely monitored and recorded.

Key takeaways

This study is for adults with Immune Thrombocytopenia (ITP).
It's testing a new medicine, rilzabrutinib, against a dummy treatment (placebo).
The main goal is to see if the medicine helps raise and maintain safe platelet counts.
Participation involves regular clinic visits, blood tests, and questionnaires.
Potential benefits include access to a new treatment; risks include possible side effects.
Your decision to participate is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is looking for adults who are 18 years old or older. Both men and women are welcome to take part. At this time, teenagers under 18 cannot join this specific trial.

To be considered, you must have persistent or chronic Immune Thrombocytopenia (ITP). This means you've been living with ITP for a certain period, and it hasn't resolved on its own, or treatments haven't kept your platelet count stable enough.

There will also be other specific medical criteria that your doctor will need to check to make sure the study is a safe and suitable option for you. These might include details about your current health, past ITP treatments, and other medications you might be taking.

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 persistent or chronic Immune Thrombocytopenia (ITP)?
Are you able to attend regular clinic appointments for blood tests and check-ups?
Are you willing to take a study medication, which could be the active drug or a placebo?
Are you comfortable filling out questionnaires about your health and quality of life?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be randomly assigned to either receive the new medicine, rilzabrutinib, or a placebo (dummy treatment) for about 24 weeks. This period is 'blinded,' meaning neither you nor your study doctor will know which one you're getting. You'll take the study medicine by mouth. Throughout these weeks, you'll have regular visits to the clinic for medical check-ups, blood tests to measure your platelet count and general health, and to discuss any symptoms or side effects you might be experiencing. We'll also ask you to fill out questionnaires about how you're feeling and how ITP impacts your daily life. After the initial 24 weeks, you might have the option to join an 'open-label' extension, where all participants receive the active rilzabrutinib, and everyone knows what treatment they are on. The total duration of your participation would depend on whether you join this extension phase.

Potential risks and benefits

Taking part in this study could offer several potential benefits. You might receive a new medicine that could improve your platelet count and quality of life. Even if you receive the placebo, you will still receive close medical attention and monitoring from a dedicated research team. However, there are potential risks, like any new medicine, rilzabrutinib may have side effects, which will be carefully monitored. The placebo might not improve your ITP, meaning you could continue to experience symptoms. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (7)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Hungary
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Unverified
Spain
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Unverified
Italy
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Unverified
Poland
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Unverified
Norway
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Unverified
France
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Unverified
Germany

Common questions

What is ITP?

ITP stands for Immune Thrombocytopenia. It's a condition where your body's immune system mistakenly attacks and destroys your own platelets, which are important for blood clotting.

What is rilzabrutinib?

Rilzabrutinib is a new medicine being tested. It's hoped to help increase platelet counts in people with ITP.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks exactly like the actual medicine but doesn't contain any active drug. It helps researchers compare the real medicine's effects accurately.

Will I know if I'm getting the real medicine?

During the first 24 weeks, neither you nor your doctor will know if you're receiving rilzabrutinib or the placebo. This is a common practice in research to ensure fair results.

How long will I be in the study?

The main part of the study lasts 24 weeks, but there might be an option to continue in an open-label extension phase, which could extend your participation.