A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia

NCT06721013 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: 27294J2N-MC-JZNZ
• Study Type: interventional
• Target: 58
• Locations: 10 countries
• Sites: 45

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.

Conditions

Immune Thrombocytopenia (ITP)

Outcome Measures

Primary Outcomes (6)

• 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

  Baseline Up to Week 4

• Phase 1-Dose Limiting Toxicity (DLT) of Pirtobrutinib

  DLTs of Pirtobrutinib

  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

  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)

  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

  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

  Baseline Up to Week 24

Secondary Outcomes (7)

• 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

Study Arms (3)

Pirtobrutinib Phase 1

EXPERIMENTAL

Pirtobrutinib administered orally

Drug: Pirtobrutinib

Pirtobrutinib Phase 2

EXPERIMENTAL

Pirtobrutinib administered orally

Drug: Pirtobrutinib

Placebo Phase 2

PLACEBO COMPARATOR

Placebo administered orally

Drug: Placebo

Interventions

Pirtobrutinib DRUG

Administered orally

Pirtobrutinib Phase 1; Pirtobrutinib Phase 2

Placebo DRUG

Administered orally

Placebo Phase 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Eli Lilly and Company: lead

Study Sites (45)

USC Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

RECRUITING

Stanford University

Stanford, California, 94305-5406, United States

NOT YET RECRUITING

MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States

RECRUITING

University of Miami Hospital and Clinics Sylvester Comprehensive Cancer Center

Miami, Florida, 33136, United States

NOT YET RECRUITING

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61614, United States

NOT YET RECRUITING

Ochsner Clinical Foundation

New Orleans, Louisiana, 70121, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

NOT YET RECRUITING

Clinical Research Alliance

Westbury, New York, 11590, United States

RECRUITING

Texas Oncology - Central South

Austin, Texas, 78758, United States

NOT YET RECRUITING

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Texas Oncology Gulf Coast

The Woodlands, Texas, 77380, United States

NOT YET RECRUITING

Nanfang Hospital of Southern Medical University

Guangzhou, 510515, China

NOT YET RECRUITING

Qilu Hospital of Shandong University

Jinan, 250012, China

NOT YET RECRUITING

Hematology Hospital of the Chinese Academy of Medical Sciences

Tianjin, 300020, China

NOT YET RECRUITING

Union Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, 430022, China

NOT YET RECRUITING

OUH

Odense C, 5000, Denmark

RECRUITING

Hôpital Henri Mondor

Créteil, 94010, France

RECRUITING

CHU Dijon - Hopital du Bocage

Dijon, 21034, France

RECRUITING

CHU Bordeaux - Hôpital Haut-Lévêque

Pessac, 33604, France

RECRUITING

Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi IRCCS

Bologna, 40138, Italy

RECRUITING

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

NOT YET RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, 00168, Italy

RECRUITING

Azienda Sanitaria Universitaria Giuliano Isontina (ASU GI)

Trieste, 34129, Italy

NOT YET RECRUITING

Haukeland University Hospital

Bergen, 5021, Norway

NOT YET RECRUITING

Sykehuset Ostfold, Kalnes

Grålum, 1714, Norway

NOT YET RECRUITING

St. Olavs Hospital Hf, Universitetssykehuset i Trondheim

Trondheim, 7030, Norway

NOT YET RECRUITING

Pratia Onkologia Katowice

Katowice, 40-519, Poland

RECRUITING

Pratia MCM Krakow

Krakow, 30-727, Poland

RECRUITING

Aidport sp z o.o.

Skorzewo, 60-185, Poland

RECRUITING

MICS Centrum Medyczne Torun

Torun, 87-100, Poland

RECRUITING

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu

Wroclaw, 50-367, Poland

NOT YET RECRUITING

Pusan National University Hospital

Busan, 49241, South Korea

RECRUITING

Seoul National University Hospital

Seoul, 03080, South Korea

RECRUITING

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

RECRUITING

Asan Medical Center

Seoul, 05505, South Korea

RECRUITING

The Catholic University of Korea, Seoul St. Mary's Hospital

Seoul, 06591, South Korea

RECRUITING

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

RECRUITING

Hospital Universitario de Burgos

Burgos, 09006, Spain

RECRUITING

Hospital General Universitario Morales Meseguer

Murcia, 30008, Spain

RECRUITING

Clinica Universidad de Navarra

Pamplona, 31008, Spain

RECRUITING

Bristol Haematology and Oncology Centre

Bristol, BS2 8ED, United Kingdom

RECRUITING

St James's University Hospital

Leeds, LS9 7TF, United Kingdom

RECRUITING

Leicester Royal Infirmary

Leicester, LE1 5WW, United Kingdom

RECRUITING

Royal London Hospital

London, E1 1FR, United Kingdom

NOT YET RECRUITING

Hammersmith Hospital

London, W12 0HS, United Kingdom

NOT YET RECRUITING

Related Links

• Lilly Trials

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

pirtobrutinib

Condition Hierarchy (Ancestors)

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

Study Officials

• Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 8 AM - 8 PM Eastern time (UTC/GMT - 5 hours, EST)

  Eli Lilly and Company

  STUDY DIRECTOR

Central Study 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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Phase 1-Open label, Phase 2-Double-blind
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Phase 1-Open label Sequential, Phase 2-Double-blind Parallel

Study Record Dates

• First Submitted: December 3, 2024
• First Posted: December 6, 2024
• Study Start: July 30, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): February 1, 2027
• Last Updated: April 17, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

ES (4); CN (4); DK (1); FR (3); GB (5); US (11); IT (4); NO (3); PL (5); KR (5)