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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Eli Lilly & Co.

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

28 Jul 2025 → ongoing

Decision date (initial)

2025-06-23

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Eli Lilly and Company

External identifiers

EU CT number

2024-518502-40-00

ClinicalTrials.gov

NCT06721013

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Dose response, Pharmacokinetic

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

Secondary objectives 8

1. Phase 1: To evaluate the preliminary efficacy of pirtobrutinib in participants with ITP
2. Phase 1: To evaluate the extent of disease control
3. Phase 1: To describe the pharmacokinetics of pirtobrutinib in participants with ITP
4. Phase 2: To assess additional efficacy of pirtobrutinib versus placebo in participants with ITP
5. Phase 2: To evaluate the extent of disease control of pirtobrutinib versus placebo in participants with ITP
6. Phase 2: To describe the use of rescue medications of pirtobrutinib versus placebo in participants with ITP
7. Phase 2: To assess the safety of pirtobrutinib in participants with ITP
8. Phase 2: To describe the pharmacokinetics of pirtobrutinib in participants with ITP

Conditions and MedDRA coding

Immune Thrombocytopenia

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Are 18 years of age or older
2. Have a confirmed diagnosis of primary ITP
3. Have documented history of response to at least 1 previous treatment for ITP
4. Have not responded to previous treatments for primary ITP

Exclusion criteria 4

1. Have a history of any blood clots or blockages in their blood vessels in the last 12 months
2. 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
3. Have a history of significant cardiovascular disease
4. Have a diagnosis or history of a blood-related cancer

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Phase 1: DLTs
2. Phase 1: Other safety endpoints, including, but not limited to, TEAEs, SAEs, clinical laboratory tests, vital signs, and ECGs
3. 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 endpoints 8

1. 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
2. Phase 1: Number of cumulative weeks with platelet counts ≥50 k/μL by Week 12
3. Phase 1: Plasma concentrations of pirtobrutinib
4. 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
5. Phase 2: Number of cumulative weeks with platelet counts ≥50 k/μL and ≥100 k/μL
6. Phase 2: Proportion of participants requiring rescue therapy
7. Phase 2: Summary of safety data, including but not limited to the number and incidence of TEAEs, SAEs, and discontinuations due to AEs
8. Phase 2: Plasma concentrations of pirtobrutinib

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Eli Lilly & Co.

Sponsor organisation

Eli Lilly & Co.

Address

1 Lilly Corporate Center

City

Indianapolis

Postcode

46285-0001

Country

United States

Scientific contact point

Organisation

Eli Lilly & Co.

Contact name

Lilly Clinical Trials information desk

Public contact point

Organisation

Eli Lilly & Co.

Contact name

Lilly Clinical Trials information desk

Third parties 4

Locations

6 EU/EEA countries · 20 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 97 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications