What side effects are associated with this type of intervention?

Common treatments for Thrombocytopenic Purpura (ITP) include corticosteroids, intravenous immunoglobulins (IVIG), and thrombopoietin receptor agonists. Corticosteroids can cause side effects such as weight gain, hypertension, and increased risk of infections. IVIG may lead to headaches, fever, and allergic reactions. Thrombopoietin receptor agonists can result in fatigue, headache, and increased risk of blood clots. For treatments similar to Rilzabrutinib, which is a Bruton's Tyrosine Kinase (BTK) inhibitor, side effects may include bleeding, infections, and gastrointestinal issues. It is important to discuss these potential side effects with a healthcare provider to determine the best treatment plan.

What prior FDA approvals exist?

For the treatment of Thrombocytopenic Purpura, the FDA has approved several thrombopoietin receptor agonists (TPO-RAs) such as romiplostim, eltrombopag, and avatrombopag. These drugs stimulate platelet production and are used in patients with chronic immune thrombocytopenia (ITP). Romiplostim and eltrombopag are particularly notable for their efficacy in increasing platelet counts. Additionally, avatrombopag is approved for thrombocytopenic patients with chronic liver disease undergoing invasive procedures. While Rilzabrutinib, a BTK inhibitor, is still under investigation, it represents a novel approach targeting the underlying immune mechanisms of ITP.

What other types of research exist?

Promising novel alternatives to Rilzabrutinib for ITP patients include other BTK inhibitors such as Acalabrutinib and Zanubrutinib, which have shown efficacy in related hematologic conditions. Additionally, therapies like Eltrombopag and Romiplostim, which are thrombopoietin receptor agonists, continue to be effective options. Emerging treatments in clinical trials, such as Syk inhibitors (e.g., Fostamatinib), also offer potential new avenues for managing ITP.

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Bruton's Tyrosine Kinase (BTK) Inhibitor

Rilzabrutinib for Immune Thrombocytopenia (LUNA 3 Trial)

Phase 3

Recruiting

Research Sponsored by Principia Biopharma, a Sanofi Company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥1.5 X 10^9/L, AST/ALT ≤1.5 x upper limit of normal [ULN], albumin ≥3 g/dL, total bilirubin ≤1.5 x ULN [unless the patient has documented Gilbert syndrome], glomerular filtration rate >50 [Cockcroft and Gault method])

Must not have

Planned surgery in the time frame of the dosing period

History of solid organ transplant

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose

Awards & highlights

Pivotal Trial

Summary

This trial tests rilzabrutinib, a drug that may help people with chronic ITP who have very low platelet counts. The drug works by blocking a substance that causes the immune system to destroy platelets. Participants will take the drug for several months to see if it helps increase their platelet levels.

Who is the study for?

Adults and adolescents with persistent or chronic immune thrombocytopenia (ITP) who have a very low platelet count are eligible for this trial. They must not be pregnant, planning surgery soon, or have had certain treatments like blood transfusions recently. Participants need to agree to use contraception and cannot have other health conditions that might interfere with the study.

What is being tested?

The trial is testing Rilzabrutinib, a potential new treatment for ITP. Patients will either receive Rilzabrutinib or a placebo twice daily over several weeks to see if it helps increase their platelet counts compared to no active treatment.

What are the potential side effects?

While specific side effects of Rilzabrutinib aren't listed here, similar medications can cause issues like headaches, nausea, diarrhea, muscle pain, and could potentially affect liver function tests. Side effects vary from person to person.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My blood, liver, and kidney tests are within normal ranges.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am scheduled for surgery during the treatment period.

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I have had a solid organ transplant.

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My B-cell count is normal after rituximab treatment.

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I have been diagnosed with secondary immune thrombocytopenia.

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I haven't had cancer that needed chemo or surgery in the last 5 years, except for non-melanoma skin cancer.

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I have never taken rilzabrutinib.

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I have not received a live vaccine in the last 28 days and do not plan to during the study.

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I have been diagnosed with myelodysplastic syndrome.

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I have not had rituximab or a spleen removal in the last 3 months.

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I haven't taken any BTK inhibitors (except rilzabrutinib) in the last 30 days.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Durable platelet response during the last 6 weeks of the 24-week blinded treatment period (not for EU and UK)

Secondary study objectives

Change from baseline in disease-specific QoL as measured by the Kids' ITP Tools (ITP-KIT) score in pediatric participants

Change from baseline on the Symptoms, Bother and Activity domains of the ITP Patient Assessment Questionnaire (ITP-PAQ) in adult patients (≥18 years)

Frequency and severity of Treatment Emergent Adverse Events

+1 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: RilzabrutinibExperimental Treatment1 Intervention

Patients receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label period

Group II: PlaceboPlacebo Group1 Intervention

Patients receive matching placebo 400mg orally twice daily for up to 24 weeks

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Rilzabrutinib

2021

Completed Phase 2

~380

0 / 11Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Bruton's Tyrosine Kinase (BTK) inhibitors, such as Rilzabrutinib, work by blocking the BTK enzyme, which is essential in the signaling pathways of B-cells and other immune cells. This inhibition reduces the immune system's attack on platelets, thereby increasing platelet counts. Other common treatments for Thrombocytopenic Purpura (ITP) include corticosteroids, which suppress the overall immune response, and thrombopoietin receptor agonists like eltrombopag, which stimulate platelet production. Understanding these mechanisms is crucial for ITP patients as it aids in selecting the most appropriate treatment based on whether their condition is due to increased destruction or decreased production of platelets.