Not recruitingRare disease

Study of Rilzabrutinib for Adults with Relapsed Immune Thrombocytopenia

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Immune Thrombocytopenia (ITP). ITP is a disorder where the immune system mistakenly attacks and destroys platelets, which are cells in the blood that help with clotting. This can lead to easy or excessive bruising and bleeding. The treatment being investigated in this study is a medication called Rilzabrutinib, which is taken orally in the form of a tablet. Rilzabrutinib is a type of drug known as a BTK inhibitor, which means it works by blocking a specific protein in the body that is involved in the immune response.

The purpose of the study is to explore the safety and effectiveness of Rilzabrutinib in adults with ITP who have not responded well to previous treatments. The study is divided into two parts. In the first part, different doses of Rilzabrutinib will be tested to find the most suitable dose. In the second part, the focus will be on a specific dose to see how well it works over a longer period. Participants will take the medication and have regular check-ups to monitor their platelet levels and any side effects they might experience.

Throughout the study, researchers will pay close attention to how the participants' platelet counts change and whether they experience any side effects from the medication. The goal is to determine if Rilzabrutinib can help increase platelet counts and reduce bleeding symptoms in people with ITP. Participants will be monitored closely to ensure their safety and to gather information on how well the treatment works over time.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, eligibility is confirmed based on age (18 to 80 years) and diagnosis of immune-related thrombocytopenia (ITP).
Step 2
Part A: dose-finding phase
In this phase, the focus is on understanding the safety and tolerability of up to four different dose levels of rilzabrutinib.
Rilzabrutinib is taken orally in the form of a tablet.
The clinical activity of these doses is also explored, particularly in patients whose condition has relapsed or not responded to previous treatments.
Monitoring includes tracking any adverse events and changes in platelet counts.
Step 3
Part B: fixed-dose phase
This phase involves taking a fixed dose of 400 mg of rilzabrutinib twice daily (BID).
The aim is to further assess the safety and effectiveness of this dose.
The response of platelet counts is evaluated over the first 8 weeks to determine the primary outcome.
Additional monitoring includes the number of weeks with increased platelet counts and any need for rescue medication.
Step 4
Monitoring and evaluation
Throughout the study, regular assessments are conducted to monitor platelet counts and any treatment-emergent adverse events.
The study aims to identify the proportion of patients achieving specific platelet count thresholds without the use of rescue medication.
The study is expected to conclude by December 31, 2025.

Who can join the trial?

3 criteria

Patients must be **male or female** and between **18 to 80 years old**.
Patients must have **Immune Thrombocytopenia (ITP)**, which is a condition where the immune system attacks and destroys platelets in the blood. Platelets are cells that help with blood clotting.
Patients can have either **primary** ITP, which means it occurs on its own, or **secondary** ITP, which means it is caused by another condition or factor.

Who cannot join the trial?

10 criteria

Patients who have a known allergy or severe reaction to the study medication or similar drugs.
Patients who are currently participating in another clinical trial or have recently participated in one.
Patients with a history of certain serious medical conditions that could interfere with the study, such as severe heart, liver, or kidney problems.
Patients who are pregnant or breastfeeding.
Patients who have a history of drug or alcohol abuse that could affect their ability to follow the study procedures.
Patients who have received certain treatments or medications that could interfere with the study results.
Patients who have an active infection that requires treatment.
Patients who have a condition that affects their immune system, making them more vulnerable to infections.
Patients who have a mental health condition that could affect their ability to understand or follow the study instructions.
Patients who have had a recent major surgery or are planning to have surgery during the study period.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Responsive
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, The Netherlands
Verified
Fakultni Nemocnice Brno
Brno, Czechia
Verified
Vseobecna Fakultni Nemocnice V Praze
Prague, Czechia

Trial locations

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Investigated drugs

Rilzabrutinib (PRN1008) is an oral medication being studied for its potential to help patients with immune thrombocytopenia (ITP), a condition where the immune system attacks and destroys platelets in the blood. This medication works by inhibiting a protein called BTK, which plays a role in the immune system's response. The trial aims to find the right dose of rilzabrutinib that is both safe and effective for patients who have not responded well to previous treatments. The study will also look at how well the medication can increase platelet counts in these patients.

Rilzabrutinib

What is already known about the treatment

Rilzabrutinib – Rilzabrutinib is an oral medication administered in tablet form, designed to treat patients with immune thrombocytopenia, a condition characterized by low platelet counts. It is currently being studied in clinical trials to assess its safety, tolerability, and effectiveness. The main therapeutic indication for rilzabrutinib is for patients who have relapsed or have not responded adequately to previous treatments for immune thrombocytopenia. At the molecular level, rilzabrutinib works by inhibiting Bruton's tyrosine kinase (BTK), which plays a role in the immune system's signaling pathways, potentially reducing the destruction of platelets. It is classified pharmacologically as a BTK inhibitor.

Investigated diseases

Immune Thrombocytopenia – Immune Thrombocytopenia (ITP) is a disorder characterized by a low platelet count, which can lead to easy or excessive bruising and bleeding. The condition occurs when the immune system mistakenly attacks and destroys platelets, which are essential for normal blood clotting. ITP can be acute, often resolving within six months, or chronic, lasting longer and requiring ongoing management. Symptoms may include petechiae, which are small red or purple spots on the skin, nosebleeds, and bleeding gums. The severity of symptoms can vary widely among individuals, with some experiencing mild symptoms and others facing more significant bleeding issues. The progression of ITP can be unpredictable, with periods of remission and relapse.