A Phase 1/2, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults With Immune Thrombocytopenia
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 68
- 试验地点
- 81
- 主要终点
- 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
研究概览
简要总结
The purpose of the phase 1 part of this study was 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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Basic Science
- 盲法
- Double (Participant, Investigator)
盲法说明
Phase 1-Open label, Phase 2-Double-blind
入排标准
- 年龄范围
- 18 Years 至 64 years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 at least 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
排除标准
- •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)
研究组 & 干预措施
Placebo Phase 2
Placebo administered orally
干预措施: Placebo (Drug)
Pirtobrutinib Phase 2
Pirtobrutinib administered orally
干预措施: Pirtobrutinib (Drug)
Pirtobrutinib Phase 1
Pirtobrutinib administered orally
干预措施: Pirtobrutinib (Drug)
结局指标
主要结局
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
时间窗: 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
时间窗: Baseline Up to Week 4
DLTs of Pirtobrutinib
Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Vital Signs: Blood Pressure, Pulse Rate, and Body Temperature
时间窗: Baseline Up to Week 16
Blood Pressure, Pulse Rate, and Body Temperature
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)
时间窗: Baseline Up to Week 16
Hematology, Clinical Chemistry, Urinalysis, Pregnancy, Hepatitis Serology and Cytomegalovirus (CMV)
Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Electrocardiograms (ECGs): ECG QT Interval
时间窗: Baseline Up to Week 16
ECG QT Interval
Phase 2-Efficacy of Pirtobrutinib Versus Placebo
时间窗: Baseline Up to Week 24
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
次要结局
- Phase 1-Preliminary Efficacy of Pirtobrutinib(Day 1 Up to Week 12)
- Phase 1-Evaluate the Extent of Disease Control(Day 1 Up to Week 12)
- Phase 1: Pharmacokinetics (PK) of Pirtobrutinib(Baseline Up to Week 16)
- Phase 2-Assess Additional Efficacy of Pirtobrutinib Versus Placebo(Week 14 Up to Week 24)
- Phase 2-Evaluate the Extent of Disease Control of Pirtobrutinib Versus Placebo(Baseline Up to Week 24)
- Phase 2-Describe the Use of Rescue Medications of Pirtobrutinib Versus Placebo(Baseline Up to Week 24)
- Phase 2-Describe the PK of Pirtobrutinib(Week 16 Up to Week 40)
研究者
Lilly Clinical Trials information desk
Scientific
Eli Lilly & Co.