Pirtobrutinib for Immune Thrombocytopenic Purpura

Recruiting at 3 trial locations

Overseen By There may be multiple sites in this clinical trial. 1-877-CTLILLY (1-877-285-4559) or

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

Trial Summary

What is the purpose of this trial?

The purpose of the phase 1 part of this study is to evaluate how well pirtobrutinib is tolerated and what side effects may occur. The phase 2 part of the study will further investigate efficacy and safety of multiple pirtobrutinib dosages versus placebo. The study drug will be administered orally in participants with Primary Immune Thrombocytopenia (ITP). Blood tests will be performed to check how much pirtobrutinib gets into the bloodstream and how long it takes the body to eliminate it. The study will last up to approximately 16 weeks for phase 1 dose-escalation and 28 weeks for phase 2 dose-optimization, excluding screening.

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.

What data supports the effectiveness of the drug Pirtobrutinib for treating Immune Thrombocytopenic Purpura?

While there is no direct data on Pirtobrutinib for Immune Thrombocytopenic Purpura, similar drugs like Rilzabrutinib, which also inhibit Bruton's tyrosine kinase, have shown promise in increasing platelet counts by reducing platelet destruction and antibody production in patients with this condition. ^{1 2 3 4 5}

How is the drug Pirtobrutinib different from other treatments for immune thrombocytopenic purpura?

Pirtobrutinib is unique because it is a Bruton tyrosine kinase inhibitor, which targets specific pathways involved in immune thrombocytopenic purpura by potentially reducing platelet destruction and autoantibody production. This approach is different from traditional treatments that focus on general immunosuppression or stimulating platelet production. ^{1 2 3 5 6}

Research Team

Contact Lilly at 1-800-LillyRx (1-800-545-5979)

Principal Investigator

Eli Lilly and Company

Eligibility Criteria

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

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.

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

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Rilzabrutinib versus placebo in adults and adolescents with persistent or chronic immune thrombocytopenia: LUNA 3 phase III study. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia. [2022]

3. Hungary pubmed.ncbi.nlm.nih.gov

Recent changes in paradigms in the management of adulthood chronic idiopathic thrombocytopenic purpura [2022]

4. China pubmed.ncbi.nlm.nih.gov

[Efficacy and safety of rituximab therapy for refractory/relapsing thrombotic thrombocytopenic purpura]. [2015]

5. England pubmed.ncbi.nlm.nih.gov

Fostamatinib: a review of its clinical efficacy and safety in the management of chronic adult immune thrombocytopenia. [2021]

6. England pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of immune thrombocytopenia in adults. [2021]