Skip to main content
OpenTrials
Recruiting

NCT Number: NCT06721013

A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Immune Thrombocytopenia (ITP) Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Nanfang Hospital of Southern Medical University, Guangzhou, China

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for 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 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

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)

Treatment and study plan

Pirtobrutinib

Drug

Administered orally

Placebo

Drug

Administered orally

Primary outcomes

  1. 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

    Time frame: Baseline Up to Week 4

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

  2. Phase 1-Dose Limiting Toxicity (DLT) of Pirtobrutinib

    Time frame: Baseline Up to Week 4

    DLTs of Pirtobrutinib

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

    Time frame: Baseline Up to Week 16

    Blood Pressure, Pulse Rate, and Body Temperature

  4. 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)

    Time frame: Baseline Up to Week 16

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

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

    Time frame: Baseline Up to Week 16

    ECG QT Interval

  6. Phase 2-Efficacy of Pirtobrutinib Versus Placebo

    Time frame: 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

Secondary outcomes

  1. Phase 1-Preliminary Efficacy of Pirtobrutinib

    Time frame: 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.

  2. Phase 1-Evaluate the Extent of Disease Control

    Time frame: Day 1 Up to Week 12

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

  3. Phase 1: Pharmacokinetics (PK) of Pirtobrutinib

    Time frame: Baseline Up to Week 16

    PK: Plasma Concentrations of Pirtobrutinib

  4. Phase 2-Assess Additional Efficacy of Pirtobrutinib Versus Placebo

    Time frame: Week 14 Up to Week 24

    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

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

    Time frame: Baseline Up to Week 24

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

  6. Phase 2-Evalulate the Extent of Disease Control of Pirtobrutinib Versus Placebo

    Time frame: Baseline Up to Week 24

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

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

    Time frame: Baseline Up to Week 24

    Proportion of participants requiring rescue therapy

  8. Phase 2-Describe the PK of Pirtobrutinib

    Time frame: Week 16 Up to Week 40

    PK: Plasma Concentrations of Pirtobrutinib

Study contacts

Contact information is provided by the study sponsor or research team.

Physicians interested in becoming principal investigators please contact

CONTACT

[email protected]

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

CONTACT

[email protected]

1-317-615-4559

Sponsors and collaborators

Lead sponsor

Eli Lilly and Company

Industry

Registry information

Official study title

A Phase 1/2, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults With Immune Thrombocytopenia

Important dates

Study start
2025
Primary completion
2028
Study completion
2029
First posted
Dec 6, 2024
Registry last updated
Jun 25, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.