Rilzabrutinib for Immune Thrombocytopenic Purpura

(LUNA 4 Trial)

Recruiting at 1 trial location

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Sanofi

Disqualifiers: Secondary ITP, Evans syndrome, malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called rilzabrutinib for individuals with immune thrombocytopenic purpura (ITP), a condition where the immune system attacks and destroys platelets, causing easy bruising and bleeding. The researchers aim to determine if early treatment with rilzabrutinib benefits those who did not respond well to their initial ITP treatment. This single-group study provides the same treatment to all participants. Suitable candidates are adults who have previously tried standard ITP treatments without lasting success and are now experiencing a relapse or dependence on steroids. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that rilzabrutinib is likely to be safe for humans?

Research has shown that rilzabrutinib is safe for people with immune thrombocytopenic purpura (ITP). Studies indicate that it helps increase platelet counts, reducing the need for other treatments and lowering the risk of bleeding. Importantly, no reports of serious side effects, such as severe bleeding, clotting problems, or infections, have emerged. This makes rilzabrutinib a promising option for those who haven't had success with other treatments. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ITP?

Rilzabrutinib is unique because it targets Bruton's tyrosine kinase (BTK), a key player in the immune system that's not directly targeted by current treatments for Immune Thrombocytopenic Purpura (ITP), like corticosteroids or IVIG. This mechanism allows rilzabrutinib to potentially modulate the immune response more precisely, reducing the destruction of platelets. Researchers are excited because this could mean fewer side effects and a more effective treatment for patients who don't respond well to existing therapies.

What evidence suggests that rilzabrutinib might be an effective treatment for ITP?

Research has shown that rilzabrutinib, the investigational treatment in this trial, may help treat immune thrombocytopenia (ITP). In earlier studies, patients unresponsive to other treatments experienced quick and lasting increases in platelet counts, crucial for managing ITP. These studies also found that rilzabrutinib reduced the need for extra emergency medication and bleeding episodes, common issues for ITP patients. Additionally, patients reported feeling less tired during treatment. Overall, about 40% of patients showed significant improvements, and the treatment was generally safe. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

Adults over 18 with primary Immune Thrombocytopenic Purpura (ITP) who didn't respond well to their first treatment can join. They should have had some response to previous treatments but now show loss of response, relapse, or dependency on steroids.

Inclusion Criteria

Key

I am 18 or older with primary immune thrombocytopenia.

I have responded positively to my first cancer treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 8 weeks

Primary Analysis

Participants receive rilzabrutinib and are monitored for platelet response

up to 28 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

Long-term Extension

Selected participants continue treatment with rilzabrutinib

28 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Rilzabrutinib

Trial Overview The trial is testing Rilzabrutinib, an oral medication for ITP patients who failed first-line therapy. It's a single-arm study where all participants receive the drug, including initial and long-term phases followed by a follow-up period.

How Is the Trial Designed?

Group I: rilzabrutinib Experimental Treatment 1 Intervention

400 mg BID

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

1. ashpublications.org ashpublications.org/blood/article/145/24/2914/536104/Safety-and-efficacy-of-rilzabrutinib-vs-placebo-in

Safety and efficacy of rilzabrutinib vs placebo in adults with ... Rilzabrutinib in patients who failed multiple previous ITP therapies showed rapid and durable platelet response, reduced rescue medication and bleeding, ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S0006497125006081

Safety and efficacy of rilzabrutinib vs placebo in adults with ... Rilzabrutinib had rapid and durable platelet response, reduced rescue use and bleeding, improved physical fatigue, and favorable safety.

3. sanofi.com sanofi.com/en/media-room/press-releases/2024/2024-12-07-16-30-00-2993342

Press Release: ASH: rilzabrutinib demonstrated significant ... Rilzabrutinib significantly reduced the need for rescue therapy by 52% compared to placebo (p=0.0007); Significant and clinically meaningful ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39844469/

Efficacy and Safety Results With Rilzabrutinib, an Oral ... Rilzabrutinib continues to demonstrate durable platelet responses with favorable safety profile in previously treated ITP patients.

5. nejm.org nejm.org/doi/full/10.1056/NEJMoa2110297

Rilzabrutinib, an Oral BTK Inhibitor, in Immune ... Overall, treatment with oral rilzabrutinib led to rapid and durable clinical activity in 40% of these patients with immune thrombocytopenia, who ...

6. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/full/10.1002/ajh.27539

Efficacy and Safety Results With Rilzabrutinib, an Oral ... There was no treatment-related grade ≥ 2 bleeding/thrombotic events/infections, serious AE, or death. Rilzabrutinib continues to demonstrate ...