Not recruitingRare disease

Study on the Safety and Effectiveness of Rilzabrutinib for Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia (ITP)

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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), which is a disorder that can lead to easy or excessive bruising and bleeding due to low levels of platelets in the blood. The trial is testing a medication called Rilzabrutinib, also known by its code name PRN1008. This medication is taken orally in the form of a tablet. The purpose of the study is to evaluate the effectiveness and safety of Rilzabrutinib in treating adults and adolescents who have persistent or chronic ITP.

Participants in the study will be randomly assigned to receive either Rilzabrutinib or a placebo, which looks like the real medication but does not contain the active ingredient. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo during the initial phase. The study will last for a total of 24 weeks, with an additional open-label extension where all participants may receive Rilzabrutinib. The main goal is to see if Rilzabrutinib can help maintain a stable platelet count without the need for additional rescue treatments.

Throughout the study, participants will have regular check-ups to monitor their platelet levels and overall health. The trial aims to provide valuable information on how well Rilzabrutinib works in managing ITP and its safety profile. This research could potentially lead to new treatment options for those living with this condition.

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
Initial treatment phase
The trial begins with a 24-week period where you will receive either the study medication, rilzabrutinib, or a placebo. The placebo looks like the study medication but does not contain the active ingredient.
You will take the medication orally in the form of a tablet. The dosage is 400 mg, and it is taken as directed by the study team.
Step 2
Monitoring and assessment
Throughout the 24-week period, your platelet levels will be regularly monitored. The goal is to achieve a platelet count of at least 50,000 per microliter for at least 8 out of the last 12 weeks without needing additional treatment.
Your health will be assessed through various tests to ensure the safety and effectiveness of the treatment. This includes monitoring for any side effects or changes in your condition.
Step 3
Open-label extension
After the initial 24-week period, there is an option to enter an open-label extension phase. In this phase, all participants will receive rilzabrutinib regardless of their initial group assignment.
This phase allows for continued monitoring and assessment of the medication's long-term effects and safety.
Step 4
Final evaluation
At the end of the study, a final evaluation will be conducted to assess the overall effectiveness and safety of the treatment.
This includes a comprehensive review of your health status, platelet counts, and any side effects experienced during the trial.

Who can join the trial?

7 criteria

Patients must have **primary Immune Thrombocytopenia (ITP)** for more than 6 months if they are aged 12 to under 18 years, or more than 3 months if they are 18 years or older.
Patients should have previously responded to treatments like **IVIg/anti-D** or **corticosteroids (CSs)**, but the response did not last. They must also have issues with standard ITP treatments, such as intolerance, insufficient response, or contraindications.
During the screening period, patients must have an average of two **platelet counts** taken at least 5 days apart that are less than 30,000 per microliter, and no single count should be more than 35,000 per microliter. Pediatric patients must also need treatment for ITP as assessed by the doctor.
Patients must have adequate **blood, liver, and kidney function**. This includes a certain level of **neutrophils** (a type of white blood cell), **AST/ALT** (liver enzymes), **albumin** (a protein in the blood), and **bilirubin** (a substance made by the liver). The **glomerular filtration rate** (a measure of kidney function) must be above 50.
Patients must have a **hemoglobin** level greater than 9 grams per deciliter within one week before starting the study. Hemoglobin is a protein in red blood cells that carries oxygen.
All patients must use **contraception** according to local regulations if they are participating in the study.
Patients must be able to provide written **informed consent** or assent, and if they are minors, consent must also be obtained from their guardian. They must agree to follow the study's schedule of assessments.

Who cannot join the trial?

6 criteria

Patients who have a different condition than Immune Thrombocytopenia (ITP) cannot participate. ITP is a condition where the body has a low number of platelets, which are cells that help the blood to clot.
Patients who are not within the specified age range cannot participate. The age range includes children, teenagers, and adults.
Patients who are not willing to avoid using other treatments that could interfere with the study cannot participate. This means they should not use any other treatment that could affect their platelet count during the study.
Patients who are not able to follow the study procedures or attend the required visits cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have any other medical condition that the study doctors think might make it unsafe for them to participate cannot join the study.

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Verified sites

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Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Responsive
Madrid, Spain
Semmelweis University
Responsive
Budapest, Hungary
Sykehuset Oestfold HF Kalnes
Responsive
Graalum, Norway

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Investigated drugs

Rilzabrutinib (PRN1008) is an oral medication being studied for its effectiveness and safety in treating adults and adolescents with persistent or chronic immune thrombocytopenia (ITP). The trial aims to see how well this medication can help maintain a stable platelet count in patients who have not responded well to other treatments.

Rilzabrutinib

What is already known about the treatment

Rilzabrutinib – Rilzabrutinib is administered orally and is currently being studied in a Phase 3 clinical trial for its effectiveness and safety in treating Immune Thrombocytopenia (ITP), a condition characterized by low platelet counts. The medication is still under investigation, and its full approval for medical use is pending further results. It is primarily indicated for patients with persistent or chronic ITP who have not responded to other treatments. Rilzabrutinib works by inhibiting Bruton's tyrosine kinase, a protein involved in the immune response, which helps to increase platelet counts. It belongs to the pharmacological class of kinase inhibitors.

Investigated diseases

Immune Thrombocytopenia (ITP) – Immune Thrombocytopenia 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 bleeding varies among individuals, and some may experience no symptoms at all. The progression of ITP can be unpredictable, with periods of remission and relapse.