A Study Of Pirtobrutinib In Participants With Immune Thrombocytopenia

Overview

About this study

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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

* Have a diagnosis of primary ITP, defined as isolated thrombocytopenia not associated with another known disease process
* Have documented history of response, defined as 2 or more platelet counts greater than or equal to 50,000/microliter (μL), to at least 1 prior line of therapy. Splenectomy is considered a line of therapy
* Have relapsed or treatment-resistant primary ITP, with no available therapies known to provide clinical benefit
* Have a platelet count less than 30,000/μL on 2 occasions more than 5 days apart in the 15 days before randomization
* Have adequate liver, renal, and hematologic functions as defined by a table
* Are willing to follow contraception requirements

Exclusion Criteria:


* Have a history of any thrombotic or embolic event within 12 months before screening
* Had a transfusion with blood or blood products or plasmapheresis within 14 days (Phase 1) or within 28 days (Phase 2) of randomization
* Have significant cardiovascular disease
* Have a diagnosis or history of hematologic malignancy
* Have hepatitis B virus (HBV) defined as positive for antigen of hepatitis B (HBsAg) or polymerase chain reaction (PCR) positive for HBV deoxyribonucleic acid (DNA)
* Have hepatitis C virus (HCV) defined as positive for anti-HCV antibodies and PCR positive for HCV ribonucleic acid (RNA)

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

 

Eligibility last updated 06/30/2025. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Alexandra Wolanskyj-Spinner, M.D.

Contact us for the latest status

Contact information:

Aaron Littlefield

5072932298

littlefield.aaron@mayo.edu

More information

Publications

Publications are currently not available