A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia

Phase 2RecruitingNCT06721013

Eli Lilly and Company58 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

BASIC_SCIENCE

Masking

DOUBLE

Enrollment

Conditions

Immune Thrombocytopenia (ITP)

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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)

Locations (45)

USC Norris Comprehensive Cancer Center

Los Angeles, California, United States

RECRUITING

Stanford University

Stanford, California, United States

NOT_YET_RECRUITING

MedStar Georgetown University Hospital

Washington D.C., District of Columbia, United States

RECRUITING

University of Miami Hospital and Clinics Sylvester Comprehensive Cancer Center

Miami, Florida, United States

Outcomes

Primary Outcomes

Phase 1-Number of Participants with One or More Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Event(s) (SAEs) Considered by the Investigator to be Related to Study Drug Administration

A summary of TEAEs and SAEs regardless of causality, will be reported in the Reported Adverse Events module

Time frame: Baseline Up to Week 4

Phase 1-Dose Limiting Toxicity (DLT) of Pirtobrutinib

DLTs of Pirtobrutinib

Time frame: Baseline Up to Week 4

Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Vital Signs: Blood Pressure, Pulse Rate, and Body Temperature

Blood Pressure, Pulse Rate, and Body Temperature

Time frame: Baseline Up to Week 16

Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Clinical Lab Tests: Hematology, Clinical Chemistry, Urinalysis, Pregnancy, Hepatitis Serology and Cytomegalovirus (CMV)

Hematology, Clinical Chemistry, Urinalysis, Pregnancy, Hepatitis Serology and Cytomegalovirus (CMV)

Time frame: Baseline Up to Week 16

Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Electrocardiograms (ECGs): ECG QT Interval

ECG QT Interval

Time frame: Baseline Up to Week 16

Phase 2-Efficacy of Pirtobrutinib Versus Placebo

Stable platelet response rate is defined as the proportion of participants achieving platelet count of greater than or equal to 50 thousand per microliter (k/μL) and on at least 4 of the 6 consecutive biweekly visits between weeks 14 and 24 in the absence of rescue therapy and prohibited concomitant medication that may impact efficacy

Time frame: Baseline Up to Week 24

Secondary Outcomes

Phase 1-Preliminary Efficacy of Pirtobrutinib

Platelet response rate defined as proportion of participants who achieve at least 2 consecutive platelet counts of greater than or equal to 50 k/μL and an increase from baseline of greater than or equal to 20 k/μL to any time during treatment without the use of rescue medication within 4 weeks prior to the latest elevated platelet count.

Time frame: Day 1 Up to Week 12

Phase 1-Evaluate the Extent of Disease Control

Number of cumulative weeks with platelet counts greater than or equal to 50 k/μL by Week 12

Time frame: Day 1 Up to Week 12

Phase 1: Pharmacokinetics (PK) of Pirtobrutinib

PK: Plasma Concentrations of Pirtobrutinib

Time frame: Baseline Up to Week 16

Phase 2-Assess Additional Efficacy of Pirtobrutinib Versus Placebo

Platelet response rate is defined as the proportion of participants with greater than or equal to 2 consecutive platelet counts greater than or equal to 50 k/μL and an increase of platelet count of greater than or equal to 20 k/μL from baseline to any time during treatment or follow-up without the use of rescue medication or prohibited concomitant medication that may impact efficacy within 4 weeks prior to the latest elevated platelet count

Time frame: Week 14 Up to Week 24

Phase 2-Evaluate the Extent of Disease Control of Pirtobrutinib Versus Placebo

Number of cumulative weeks with platelet counts greater than or equal to 50 k/μL and greater than or equal to 100 k/μL

Time frame: Baseline Up to Week 24

Phase 2-Describe the Use of Rescue Medications of Pirtobrutinib Versus Placebo

Proportion of participants requiring rescue therapy

Time frame: Baseline Up to Week 24

Phase 2-Describe the PK of Pirtobrutinib

PK: Plasma Concentrations of Pirtobrutinib

Time frame: Week 16 Up to Week 40

Central Contacts

Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or

CONTACT

1-317-615-4559 LillyTrials@Lilly.com

Physicians interested in becoming principal investigators please contact

CONTACT

clinical_inquiry_hub@lilly.com