Pirtobrutinib for Immune Thrombocytopenic Purpura

Recruiting at 47 trial locations

Overseen By Physicians interested in becoming principal investigators please contact

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Eli Lilly and Company

Disqualifiers: Thrombotic events, Cardiovascular disease, Hematologic malignancy, Hepatitis B, others

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

58 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral treatment called pirtobrutinib for individuals with Primary Immune Thrombocytopenia (ITP), a condition characterized by low platelet counts, leading to easy bruising and bleeding. The trial's first phase evaluates the drug's tolerability and side effects. The second phase compares different doses of pirtobrutinib to a placebo to assess its efficacy and safety. Ideal participants have tried other ITP treatments but still have low platelet counts and no other conditions affecting their platelets. As a Phase 1 and Phase 2 trial, this research aims to understand the treatment's effects in people and measure its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking research.

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 pirtobrutinib is likely to be safe for humans?

Research has shown that pirtobrutinib has been tested for safety in other conditions. In a study involving 110 patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), most patients took the drug after trying at least two other treatments. The study found that pirtobrutinib was generally well-tolerated.

Common side effects included low levels of white blood cells (neutropenia), low levels of red blood cells (anemia), and lung infections (pneumonia). These side effects led to treatment discontinuation in a small number of cases.

Since this trial is in its early stages, the safety of pirtobrutinib in people with immune thrombocytopenia (ITP) is still under investigation. However, because pirtobrutinib is already approved for another condition, there is some confidence in its safety. It is important to discuss potential risks with the trial team. ^{1 2 3 4 5}

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

Pirtobrutinib is unique because it works by targeting Bruton's tyrosine kinase (BTK), an enzyme involved in the immune response, which is a different approach compared to traditional treatments for immune thrombocytopenic purpura (ITP) like corticosteroids or IVIG. This targeted action might offer a more direct way to manage the condition with potentially fewer side effects. Researchers are excited about pirtobrutinib because it could provide a new option for patients who do not respond well to existing therapies, offering hope for better management of ITP.

What evidence suggests that pirtobrutinib might be an effective treatment for immune thrombocytopenic purpura?

Research has shown that pirtobrutinib may help treat immune thrombocytopenia (ITP), a condition where the immune system mistakenly attacks platelets, leading to easy or excessive bruising and bleeding. Early studies suggest that pirtobrutinib can adjust B-cell activity, reducing the production of harmful antibodies that attack the body's own cells. In past studies, many participants experienced an increase in their platelet counts, with some studies showing a response rate of up to 40%. This trial will evaluate pirtobrutinib in two phases, with some participants receiving the active treatment and others receiving a placebo. Overall, these findings suggest that pirtobrutinib may help manage ITP by addressing its root causes. ^{1 3 6 7 8}

Who Is on the Research Team?

Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 8 AM - 8 PM Eastern time (UTC/GMT - 5 hours, EST)

Principal Investigator

Eli Lilly and Company

Are You a Good Fit for This Trial?

This trial is for individuals with Primary Immune Thrombocytopenia (ITP), a condition where the immune system destroys platelets. Participants must meet specific health criteria, which are not detailed here.

Inclusion Criteria

I have been diagnosed with ITP, a condition where my platelet count is low without any other known disease causing it.

My platelet counts improved to 50,000 or more after treatment.

My ITP has not improved with treatment and no other treatments are likely to help.

Exclusion Criteria

I have not had a blood transfusion or plasmapheresis within the last month.

I have or had blood cancer.

I have had a blood clot in the past year.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1 Treatment

Participants receive pirtobrutinib to evaluate tolerance and side effects

16 weeks

Regular visits for blood tests and monitoring

Phase 2 Treatment

Further investigation of efficacy and safety of multiple pirtobrutinib dosages versus placebo

28 weeks

Biweekly visits for efficacy assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Pirtobrutinib

Trial Overview The study tests pirtobrutinib's tolerability and side effects in phase 1, then compares its effectiveness and safety at various doses to a placebo in phase 2. It involves oral administration of the drug and regular blood tests over up to 16 weeks for phase 1 and an additional 28 weeks for phase 2.

How Is the Trial Designed?

Group I: Pirtobrutinib Phase 2 Experimental Treatment 1 Intervention

Pirtobrutinib administered orally

Group II: Pirtobrutinib Phase 1 Experimental Treatment 1 Intervention

Pirtobrutinib administered orally

Group III: Placebo Phase 2 Placebo Group 1 Intervention

Placebo administered orally

Pirtobrutinib is already approved in United States for the following indications:

Approved in United States as Jaypirca for:

• Mantle Cell Lymphoma
• Chronic Lymphocytic Leukemia
• Small Lymphocytic Lymphoma

Who Is Running the Clinical Trial?

Eli Lilly and Company

Lead Sponsor

Trials

2,708

Recruited

3,720,000+

Dr. Daniel Skovronsky

Eli Lilly and Company

Chief Medical Officer since 2018

MD from Harvard Medical School

David A. Ricks

Eli Lilly and Company

Chief Executive Officer since 2017

BSc from Purdue University, MBA from Indiana University

Published Research Related to This Trial

In a phase 1-2 clinical trial involving 60 patients with immune thrombocytopenia, rilzabrutinib demonstrated a platelet response in 40% of participants, indicating its efficacy in increasing platelet counts through dual mechanisms of action.

The treatment was well-tolerated, with only low-level, transient side effects and no serious bleeding or thrombotic events, suggesting a favorable safety profile for further testing at the identified effective dose of 400 mg twice daily.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia. Kuter, DJ., Efraim, M., Mayer, J., et al.[2022]

Fostamatinib is an oral spleen tyrosine kinase inhibitor that effectively increases platelet counts in adults with immune thrombocytopenia who did not respond adequately to previous treatments, as shown in two Phase III trials.

The treatment is generally well tolerated, with lower rates of severe bleeding-related side effects, although common adverse events include diarrhea, nausea, and liver function test elevations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of immune thrombocytopenia in adults. Moore, DC., Gebru, T., Muslimani, A.[2021]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06721013

NCT06721013 | A Study of Pirtobrutinib in Participants With ... A Phase 1/2, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults With Immune Thrombocytopenia

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12084790/

Efficacy and Safety of Syk and BTK Inhibitors in Immune ... The sustained response rate was 27% in G2 and 33% in G1, including two of the 13 patients who transitioned from G1 to G2. Of the 12 patients who ...

3. library.ehaweb.org library.ehaweb.org/eha/2025/eha2025-congress/4162765/catherine.broome.pirtobrutinib.in.adults.with.immune.thrombocytopenia.a.phase.html?f=listing%3D3%2Abrowseby%3D8%2Asortby%3D1%2Amedia%3D1

PIRTOBRUTINIB IN ADULTS WITH IMMUNE ... - EHA Library Preclinical data for pirtobrutinib in ITP models suggest that it effectively modulates B-cell activity and reduces autoantibody production, ...

4. withpower.com withpower.com/trial/phase-2-thrombocytopenia-2-2025-adc46

Pirtobrutinib for Immune Thrombocytopenic Purpura In a phase 1-2 clinical trial involving 60 patients with immune thrombocytopenia, rilzabrutinib demonstrated a platelet response in 40% of participants, ...

5. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-pirtobrutinib-for-adults-with-immune-thrombocytopenia/

Study on the Safety and Effectiveness of Pirtobrutinib for ... This clinical trial investigates the safety and effectiveness of Pirtobrutinib in treating adults with Immune Thrombocytopenia, a disorder characterized by ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2023/216059s001lbl.pdf

JAYPIRCA® (pirtobrutinib) tablets - accessdata.fda.gov BRUIN. The safety of JAYPIRCA was evaluated in the BRUIN trial in 110 patients with CLL/SLL, with 98% receiving at least two prior lines of systemic therapy ...

7. ctv.veeva.com ctv.veeva.com/study/a-study-of-pirtobrutinib-in-participants-with-immune-thrombocytopenia

A Study of Pirtobrutinib in Participants With Immune ... The purpose of the phase 1 part of this study is to evaluate how well pirtobrutinib is tolerated and what side effects may occur.

8. ema.europa.eu ema.europa.eu/en/documents/product-information/jaypirca-epar-product-information_en.pdf

ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS The most common adverse reactions (reported in more than 2 patients) leading to dose discontinuation are neutropenia (1.0 %), anaemia (1.0 %), pneumonia (0.9 %) ...