Clinical trial NCT06721013: A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Official title: A Phase 1/2, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults With Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Immune Thrombocytopenia (ITP)
Lead sponsor: Eli Lilly and Company
Sponsor class: INDUSTRY
First posted: 2024-12-06
Enrollment: 58
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Phase 1-Open label Sequential, Phase 2-Double-blind Parallel
Primary purpose: BASIC_SCIENCE

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.

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.


Eligibility criteria: 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)

Primary outcomes:
measure: 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
timeFrame: Baseline Up to Week 4
measure: Phase 1-Dose Limiting Toxicity (DLT) of Pirtobrutinib
timeFrame: Baseline Up to Week 4
measure: Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Vital Signs: Blood Pressure, Pulse Rate, and Body Temperature
timeFrame: Baseline Up to Week 16
measure: 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)
timeFrame: Baseline Up to Week 16
measure: Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Electrocardiograms (ECGs): ECG QT Interval
timeFrame: Baseline Up to Week 16
measure: Phase 2-Efficacy of Pirtobrutinib Versus Placebo
timeFrame: Baseline Up to Week 24
Secondary outcomes:
measure: Phase 1-Preliminary Efficacy of Pirtobrutinib
timeFrame: Day 1 Up to Week 12
measure: Phase 1-Evaluate the Extent of Disease Control
timeFrame: Day 1 Up to Week 12
measure: Phase 1: Pharmacokinetics (PK) of Pirtobrutinib
timeFrame: Baseline Up to Week 16
measure: Phase 2-Assess Additional Efficacy of Pirtobrutinib Versus Placebo
timeFrame: Week 14 Up to Week 24
measure: Phase 2-Evaluate the Extent of Disease Control of Pirtobrutinib Versus Placebo
timeFrame: Baseline Up to Week 24
measure: Phase 2-Describe the Use of Rescue Medications of Pirtobrutinib Versus Placebo
timeFrame: Baseline Up to Week 24
measure: Phase 2-Describe the PK of Pirtobrutinib
timeFrame: Week 16 Up to Week 40
Other outcomes:
Locations:
city: Washington
country: United States
facility: MedStar Georgetown University Hospital
state: District of Columbia
city: Peoria
country: United States
facility: Bleeding and Clotting Disorders Institute
state: Illinois
city: Westbury
country: United States
facility: Clinical Research Alliance
state: New York
city: Houston
country: United States
facility: University of Texas MD Anderson Cancer Center
state: Texas
Study officials:
affiliation: Eli Lilly and Company
name: Contact Lilly at 1-800-LillyRx (1-800-545-5979)
role: STUDY_DIRECTOR
References: