A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
- Conditions
- Interventions
- Registration Number
- NCT06721013
- Lead Sponsor
- Eli Lilly and Company
- Brief Summary
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.
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- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 58
- 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
- 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)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Pirtobrutinib Phase 1 Pirtobrutinib Pirtobrutinib administered orally Pirtobrutinib Phase 2 Pirtobrutinib Pirtobrutinib administered orally Placebo Phase 2 Placebo Placebo administered orally
- Primary Outcome Measures
Name Time Method Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Vital Signs: Blood Pressure, Pulse Rate, and Body Temperature Baseline Up to Week 16 Blood Pressure, Pulse Rate, and Body Temperature
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 Baseline Up to Week 4 A summary of TEAEs and SAEs regardless of causality, will be reported in the Reported Adverse Events module
Phase 1-Dose Limiting Toxicity (DLT) of Pirtobrutinib Baseline Up to Week 4 DLTs of Pirtobrutinib
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), Human Immunodeficiency Virus (HIV) Baseline Up to Week 16 Hematology, Clinical Chemistry, Urinalysis, Pregnancy, Hepatitis Serology and Cytomegalovirus (CMV), Human Immunodeficiency Virus (HIV)
Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Electrocardiograms (ECGs): ECG QT Interval Baseline Up to Week 16 ECG QT Interval
Phase 2-Efficacy of Pirtobrutinib Versus Placebo Baseline Up to Week 24 Platelet response rate defined as the proportion of participants with greater than or equal to 2 consecutive platelet counts greater than or equal to 50 thousand per microliter (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 within 4 week...
- Secondary Outcome Measures
Name Time Method Phase 2-Evaluate the Extent of Disease Control of Pirtobrutinib Versus Placebo Baseline Up to Week 24 Number of cumulative weeks with platelet counts greater than or equal to 50 k/μL and greater than or equal to 100 k/μL
Phase 1-Preliminary Efficacy of Pirtobrutinib Day 1 Up to Week 12 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.
Phase 1-Evaluate the Extent of Disease Control Day 1 Up to Week 12 Number of cumulative weeks with platelet counts greater than or equal to 50 k/μL by Week 12
Phase 1: Pharmacokinetics (PK) of Pirtobrutinib Baseline Up to Week 16 PK: Plasma Concentrations of Pirtobrutinib
Phase 2-Evaluate Long-term Efficacy of Pirtobrutinib Versus Placebo Week 14 Up to Week 24 Durable response rate defined as a platelet count of at least 50 k/μL on at least 4 of the 6 consecutive biweekly visits between Weeks 14-24 with no use of rescue therapy
Phase 2-Describe the Use of Rescue Medications of Pirtobrutinib Versus Placebo Baseline Up to Week 24 Proportion of participants requiring rescue therapy
Phase 2-Describe the PK of Pirtobrutinib Week 16 Up to Week 40 PK: Plasma Concentrations of Pirtobrutinib
Trial Locations
- Locations (1)
MedStar Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States