Immune Thrombocytopenia (ITP)Phase 1/2NCT06721013
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
A Phase 1/2 study testing Pirtobrutinib in people with Immune Thrombocytopenia (ITP), run by Eli Lilly and Company at 43 sites in 10 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia (ITP)
- What is being tested?
- Pirtobrutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 68 participants
- Where
- 43 sitesin 10 countries
- Study dates
- 30 Jul 2025to Feb 2029
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
43 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or contact
Physicians interested in becoming principal investigators please contact contact
43 sites in 10 countries
United States10 sites, 10 recruiting
- Recruiting
Texas Oncology - Central South
Austin, Texas, 78705
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
- Recruiting
USC Norris Comprehensive Cancer Center
Los Angeles, California, 90033
- Not yet recruiting
University of Miami Hospital and Clinics Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
- Recruiting
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121
- Recruiting
Stanford University
Palo Alto, California, 94304
- Not yet recruiting
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61614
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
- Recruiting
Texas Oncology Gulf Coast
The Woodlands, Texas, 77380
- Recruiting
MedStar Georgetown University Hospital
Washington D.C., District of Columbia, 20007
Poland5 sites, 5 recruiting
- Recruiting
Pratia Onkologia Katowice
Katowice, 40-519
- Recruiting
Pratia MCM Krakow
Krakow, 30-727
- Recruiting
Aidport sp z o.o.
Skorzewo, 60-185
- Recruiting
MICS Centrum Medyczne Torun
Torun, 87-100
- Not yet recruiting
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
Wroclaw, 50-367
South Korea5 sites, 5 recruiting
- Recruiting
Pusan National University Hospital
Busan, 49241
- Recruiting
Seoul National University Hospital
Seoul, 03080
- Recruiting
Severance Hospital, Yonsei University Health System
Seoul, 03722
- Recruiting
Asan Medical Center
Seoul, 05505
- Recruiting
The Catholic University of Korea, Seoul St. Mary's Hospital
Seoul, 06591
United Kingdom5 sites, 5 recruiting
- Recruiting
Bristol Haematology and Oncology Centre
Bristol, BS2 8ED
- Recruiting
St James's University Hospital
Leeds, LS9 7TF
- Recruiting
Leicester Royal Infirmary
Leicester, LE1 5WW
- Not yet recruiting
Royal London Hospital
London, E1 1FR
- Not yet recruiting
Hammersmith Hospital
London, W12 0HS
China4 sites, 4 recruiting
- Not yet recruiting
Nanfang Hospital of Southern Medical University
Guangzhou, 510515
- Recruiting
Qilu Hospital of Shandong University
Jinan, 250012
- Not yet recruiting
Hematology Hospital of the Chinese Academy of Medical Sciences
Tianjin, 300020
- Not yet recruiting
Union Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan, 430022
Italy4 sites, 4 recruiting
- Recruiting
Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi IRCCS
Bologna, 40138
- Recruiting
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, 20122
- Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00168
- Recruiting
Azienda Sanitaria Universitaria Giuliano Isontina (ASU GI)
Trieste, 34129
Spain4 sites, 4 recruiting
- Recruiting
Hospital Clinic de Barcelona
Barcelona, 08036
- Recruiting
Hospital Universitario de Burgos
Burgos, 09006
- Recruiting
Hospital General Universitario Morales Meseguer
Murcia, 30008
- Recruiting
Clinica Universidad de Navarra
Pamplona, 31008
Norway3 sites, 3 recruiting
- Not yet recruiting
Haukeland University Hospital
Bergen, 5021
- Not yet recruiting
Sykehuset Ostfold, Kalnes
Grålum, 1714
- Not yet recruiting
St. Olavs Hospital Hf, Universitetssykehuset i Trondheim
Trondheim, 7030
France2 sites, 2 recruiting
- Recruiting
Hôpital Henri Mondor
Créteil, 94010
- Recruiting
CHU Dijon - Hopital du Bocage
Dijon, 21034
About this study
Official title: A Phase 1/2, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults 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.
- Conditions
- Immune Thrombocytopenia (ITP)
- Being tested
- Pirtobrutinib, Placebo
- Keywords
- Primary ITP
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PirtobrutinibDrug
Administered orally
PlaceboDrug
Administered orally
| Study group | Role | What they receive |
|---|---|---|
| Pirtobrutinib Phase 1 | Experimental | Pirtobrutinib administered orally Receives: Drug: Pirtobrutinib |
| Pirtobrutinib Phase 2 | Experimental | Pirtobrutinib administered orally Receives: Drug: Pirtobrutinib |
| Placebo Phase 2 | Placebo comparator | Placebo administered orally Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline Up to Week 4
Phase 1-Dose Limiting Toxicity (DLT) of Pirtobrutinib
DLTs of Pirtobrutinib
Measured: 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
Measured: 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)
Measured: 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
Measured: 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
Measured: Baseline Up to Week 24
Secondary outcomes (8)
Phase 1-Preliminary Efficacy of Pirtobrutinib
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.
Measured: Day 1 Up to Week 12
Phase 1-Evaluate the Extent of Disease Control
Number of cumulative weeks with platelet counts greater than or equal to 50 k/μL by Week 12
Measured: Day 1 Up to Week 12
Phase 1: Pharmacokinetics (PK) of Pirtobrutinib
PK: Plasma Concentrations of Pirtobrutinib
Measured: Baseline Up to Week 16
Phase 2-Assess Additional Efficacy of Pirtobrutinib Versus Placebo
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
Measured: Week 14 Up to Week 24
Phase 2-Evaluate the Extent of Disease Control of Pirtobrutinib Versus Placebo
Number of cumulative weeks with platelet counts greater than or equal to 50 k/μL
Measured: Baseline Up to Week 24
Phase 2-Evalulate the Extent of Disease Control of Pirtobrutinib Versus Placebo
Number of cumulative weeks with platelet counts greater than or equal to 100 k/μL
Measured: Baseline Up to Week 24
Phase 2-Describe the Use of Rescue Medications of Pirtobrutinib Versus Placebo
Proportion of participants requiring rescue therapy
Measured: Baseline Up to Week 24
Phase 2-Describe the PK of Pirtobrutinib
PK: Plasma Concentrations of Pirtobrutinib
Measured: Week 16 Up to Week 40
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2024
- Study start30 Jul 2025
- Primary completionFeb 2028 (estimated)
- Study completionFeb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- More trials run by Eli Lilly and CompanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- Phase 1-Open label Sequential, Phase 2-Double-blind Parallel
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 68 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eli Lilly and Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- 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), Study director, Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06721013
- Sponsor's protocol ID
- 27294
- Other IDs
- J2N-MC-JZNZ
- Status last verified
- Oct 2026
- First submitted
- 3 Dec 2024
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No