A Study to Investigate the Safety and Efficacy of Pirtobrutinib in Adults with Immune Thrombocytopenia

Phase 1/2

Not yet recruiting

• Conditions: Immune Thrombocytopenia
• Interventions: Drug: Pirtobrutinib; Drug: Placebo

• Registration Number: 2024-518502-40-00
• Lead Sponsor: Eli Lilly & Co.

Brief Summary

Phase 1: To assess the safety profile and tolerability of pirtobrutinib in participants with ITP and select the doses for the Phase 2 part of this study

Phase 2: To evaluate the efficacy of pirtobrutinib versus placebo in participants with ITP

Detailed Description

Not available

Study Timeline

• Study First Posted: 2025-06-23
• Last Update Posted: 2025-06-25

Recruitment & Eligibility

• Status: Authorised, recruitment pending
• Sex: Not specified
• Target Recruitment: 13

Inclusion Criteria

Are 18 years of age or older

Have a confirmed diagnosis of primary ITP

Have documented history of response to at least 1 previous treatment for ITP

Have not responded to previous treatments for primary ITP

Exclusion Criteria

Have a history of any blood clots or blockages in their blood vessels in the last 12 months

Had a transfusion with blood or blood products or plasmapheresis within 14 days of the Phase 1 part of the study or within 28 days for the Phase 2 part of the study

Have a history of significant cardiovascular disease

Have a diagnosis or history of a blood-related cancer

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Pirtobrutinib Phase 1	Pirtobrutinib	Pirtobrutinib administered orally
Pirtobrutinib Phase 2	Pirtobrutinib	Pirtobrutinib administered orally
Placebo Phase 2	Placebo	Placebo administered orally

Primary Outcome Measures

Name	Time	Method
Phase 1: DLTs		Phase 1: DLTs
Phase 1: Other safety endpoints, including, but not limited to, TEAEs, SAEs, clinical laboratory tests, vital signs, and ECGs		Phase 1: Other safety endpoints, including, but not limited to, TEAEs, SAEs, clinical laboratory tests, vital signs, and ECGs
Phase 2: Stable platelet response rate is defined as the proportion of participants achieving platelet count of ≥50 k/μL 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		Phase 2: Stable platelet response rate is defined as the proportion of participants achieving platelet count of ≥50 k/μL 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 Outcome Measures

Name	Time	Method
Phase 1: Platelet response rate defined as proportion of participants who achieve at least 2 consecutive platelet counts of ≥50 k/μL and an increase from baseline of ≥20 k/μL to any time during treatment without the use of rescue medication or prohibited concomitant medication that may impact efficacy within 4 weeks prior to the latest elevated platelet count		Phase 1: Platelet response rate defined as proportion of participants who achieve at least 2 consecutive platelet counts of ≥50 k/μL and an increase from baseline of ≥20 k/μL to any time during treatment without the use of rescue medication or prohibited concomitant medication that may impact efficacy within 4 weeks prior to the latest elevated platelet count
Phase 1: Number of cumulative weeks with platelet counts ≥50 k/μL by Week 12		Phase 1: Number of cumulative weeks with platelet counts ≥50 k/μL by Week 12
Phase 1: Plasma concentrations of pirtobrutinib		Phase 1: Plasma concentrations of pirtobrutinib
Phase 2: Platelet response rate is defined as the proportion of participants with ≥2 consecutive platelet counts ≥50 k/μL and an increase of platelet count of ≥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		Phase 2: Platelet response rate is defined as the proportion of participants with ≥2 consecutive platelet counts ≥50 k/μL and an increase of platelet count of ≥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
Phase 2: Number of cumulative weeks with platelet counts ≥50 k/μL and ≥100 k/μL		Phase 2: Number of cumulative weeks with platelet counts ≥50 k/μL and ≥100 k/μL
Phase 2: Proportion of participants requiring rescue therapy		Phase 2: Proportion of participants requiring rescue therapy
Phase 2: Summary of safety data, including but not limited to the number and incidence of TEAEs, SAEs, and discontinuations due to AEs		Phase 2: Summary of safety data, including but not limited to the number and incidence of TEAEs, SAEs, and discontinuations due to AEs
Phase 2: Plasma concentrations of pirtobrutinib		Phase 2: Plasma concentrations of pirtobrutinib

Trial Locations

Locations (8)

Odense University Hospital; 🇩🇰 Odense C, Denmark

Centre Hospitalier Universitaire De Dijon; 🇫🇷 Dijon, France

Centre Hospitalier Universitaire De Bordeaux; 🇫🇷 Pessac, France

Assistance Publique Hopitaux De Paris; 🇫🇷 Creteil Cedex, France

Azienda Sanitaria Universitaria Giuliano Isontina; 🇮🇹 Trieste, Italy

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico; 🇮🇹 Milan, Italy

Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico; 🇮🇹 Bologna, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS; 🇮🇹 Rome, Italy

Odense University Hospital

🇩🇰Odense C, Denmark

Henrik Frederiksen

Site contact

+4521849307

henrik.frederiksen@rsyd.dk