Clinical trial • Phase I/II • Immunology|Rare Disease
PIRTOBRUTINIB for Immune Thrombocytopenia
Phase I/II trial of PIRTOBRUTINIB for Immune Thrombocytopenia.
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- Immune Thrombocytopenia
- Trial Stage
- Phase I/II
- Drug Modality
- Small molecule
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 28-02-2025
- First CTIS Authorization Date
- 23-06-2025
Trial design
Randomised, open-label, matching placebo tablets (placebo tablets). dose and schedule for placebo not specified; placebo is comparator to pirtobrutinib in phase 2.-controlled, adaptive Phase I/II trial across 20 sites in France, Norway, Italy and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Matching placebo tablets (Placebo tablets). Dose and schedule for placebo not specified; placebo is comparator to pirtobrutinib in Phase 2.
- Adaptive
- True, includes a Phase 1 open-label dose-escalation (dose-finding) part; dose-limiting toxicity (DLT) assessments are primary in Phase 1 and are used to select doses for Phase 2.
- Single Multiple Or Escalation Dose Combined
- Yes
- Target Sample Size
- 34
Eligibility
Recruits 34 Vulnerable population flag selected in the record (isVulnerablePopulationSelected = true). Specific consent/assent handling text is not provided in the JSON; multiple subject information and informed consent form documents (country- and language-specific ICFs and eConsent materials) are listed in the documents section..
- Vulnerable Population
- Vulnerable population flag selected in the record (isVulnerablePopulationSelected = true). Specific consent/assent handling text is not provided in the JSON; multiple subject information and informed consent form documents (country- and language-specific ICFs and eConsent materials) are listed in the documents section.
Inclusion criteria
- {"criterion_text":"- Are 18 years of age or older"}
- {"criterion_text":"- Have a confirmed diagnosis of primary ITP"}
- {"criterion_text":"- Have documented history of response to at least 1 previous treatment for ITP"}
- {"criterion_text":"- Have not responded to previous treatments for primary ITP"}
Exclusion criteria
- {"criterion_text":"- Have a history of any blood clots or blockages in their blood vessels in the last 12 months"}
- {"criterion_text":"- 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"}
- {"criterion_text":"- Have a history of significant cardiovascular disease"}
- {"criterion_text":"- Have a diagnosis or history of a blood-related cancer"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Phase 1: DLTs","definition_or_measurement_approach":""}
- {"endpoint_text":"- Phase 1: Other safety endpoints, including, but not limited to, TEAEs, SAEs, clinical laboratory tests, vital signs, and ECGs","definition_or_measurement_approach":"Safety assessed by treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), clinical laboratory tests, vital signs and electrocardiograms (ECGs)."}
- {"endpoint_text":"- 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","definition_or_measurement_approach":"Proportion of participants with platelet count ≥50 k/μL on at least 4 of 6 consecutive biweekly visits between Weeks 14 and 24, with no rescue therapy or prohibited concomitant medication that may impact efficacy."}
Secondary endpoints
- {"endpoint_text":"- 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","definition_or_measurement_approach":"Proportion achieving ≥2 consecutive platelet counts ≥50 k/μL and increase from baseline ≥20 k/μL during treatment without rescue medication or prohibited concomitant medication within 4 weeks prior to the latest elevated platelet count."}
- {"endpoint_text":"- Phase 1: Number of cumulative weeks with platelet counts ≥50 k/μL by Week 12","definition_or_measurement_approach":"Count of cumulative weeks with platelet count ≥50 k/μL up to Week 12."}
- {"endpoint_text":"- Phase 1: Plasma concentrations of pirtobrutinib","definition_or_measurement_approach":"Plasma concentration measurements of pirtobrutinib (PK assessment)."}
- {"endpoint_text":"- 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","definition_or_measurement_approach":"Proportion with ≥2 consecutive platelet counts ≥50 k/μL and increase ≥20 k/μL from baseline at any time during treatment or follow-up without rescue medication or prohibited concomitant medication within 4 weeks prior to the latest elevated count."}
- {"endpoint_text":"- Phase 2: Number of cumulative weeks with platelet counts ≥50 k/μL and ≥100 k/μL","definition_or_measurement_approach":"Count of cumulative weeks with platelet counts meeting thresholds (≥50 k/μL and ≥100 k/μL)."}
- {"endpoint_text":"- Phase 2: Proportion of participants requiring rescue therapy","definition_or_measurement_approach":"Proportion of participants who receive rescue therapy during the study."}
- {"endpoint_text":"- Phase 2: Summary of safety data, including but not limited to the number and incidence of TEAEs, SAEs, and discontinuations due to AEs","definition_or_measurement_approach":"Summary statistics of safety events including counts and incidence of TEAEs, SAEs and discontinuations due to adverse events."}
- {"endpoint_text":"- Phase 2: Plasma concentrations of pirtobrutinib","definition_or_measurement_approach":"Plasma concentration measurements of pirtobrutinib (PK assessment)."}
Recruitment
- Registry Or Advocacy Recruitment
- True, Patient Advocacy Group letters/materials are included (generic "Patient Advocacy Group" materials are listed in multiple country-specific recruitment packages).
- Digital Remote Recruitment
- True, eConsent (participant-facing landing pages and screenshots), online banner ads, social media clinical trial posts and other digital materials are included in the recruitment documents.
- Planned Sample Size
- 34
- Recruitment Window Months
- 23
- Consent Approach
- Informed consent is required from adult participants (inclusion requires participants to be 18 years or older). Multiple subject information and informed consent form documents are listed (country- and language-specific ICFs and eConsent materials for France, Italy, Norway, Poland, Spain, Denmark and other translations such as EN/ES/IT/FR/PL). eConsent materials and privacy/security documents are listed. Specific text of consent/assent processes is not present in the JSON, but ICF and eConsent documents are enumerated.
Methods
- Country-specific recruitment materials and channels are listed: Patient Banner Ads; Banner Ads; Social Media and Clinical Trial Posts; Small Print Ad; Large Print Ad; Newspaper Advertisement (small and large print); Patient Brochure; Patient Study Guide; Doctor-to-Patient Letter; Physician Referral Letter/Brochure; Patient Advocacy Group Letter; Participant ID Card; eConsent participant-facing landing pages and screenshots.
- Target audiences include patients with immune thrombocytopenia (ITP) and referring physicians/clinics; materials are localized by country (documents present for France, Italy, Norway, Poland, Spain and Denmark).
- eConsent and digital recruitment: eConsent landing pages, eConsent screenshots, privacy and security guides are included among the subject information materials.
Geography
- Total Number Of Sites
- 20
- Total Number Of Participants
- 30
France
- Earliest CTIS Part Ii Submission Date
- 16-05-2025
- Latest Decision Or Authorization Date
- 25-08-2025
- Processing Time Days
- 101
- Number Of Sites
- 3
- Number Of Participants
- 5
Sites
- Site Name
- Centre Hospitalier Universitaire De Bordeaux
- Department Name
- Médecine Interne – Maladies Infectieuses
- Principal Investigator Name
- Jean-François VIALLARD
- Principal Investigator Email
- jean-francois.viallard@chu-bordeaux.fr
- Contact Person Name
- Jean-François VIALLARD
- Contact Person Email
- jean-francois.viallard@chu-bordeaux.fr
- Site Name
- Centre Hospitalier Universitaire De Dijon
- Department Name
- Médecine Interne et Immunologie Clinique
- Principal Investigator Name
- Sylvain AUDIA
- Principal Investigator Email
- sylvain.audia@chu-dijon.fr
- Contact Person Name
- Sylvain AUDIA
- Contact Person Email
- sylvain.audia@chu-dijon.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Service de Médecine Interne
- Principal Investigator Name
- Marc MICHEL
- Principal Investigator Email
- marc.michel2@aphp.fr
- Contact Person Name
- Marc MICHEL
- Contact Person Email
- marc.michel2@aphp.fr
Norway
- Earliest CTIS Part Ii Submission Date
- 09-12-2025
- Latest Decision Or Authorization Date
- 12-12-2025
- Processing Time Days
- 3
- Number Of Sites
- 3
- Number Of Participants
- 6
Sites
- Site Name
- Helse Bergen HF
- Department Name
- Hematology
- Principal Investigator Name
- Galina Tsykunova
- Principal Investigator Email
- galina.tsykunova@helse-bergen.no
- Contact Person Name
- Galina Tsykunova
- Contact Person Email
- galina.tsykunova@helse-bergen.no
- Site Name
- St. Olavs Hospital HF
- Department Name
- Hematology
- Principal Investigator Name
- Petter Quist Paulsen
- Principal Investigator Email
- Petter.Quist-Paulsen@Stolav.no
- Contact Person Name
- Petter Quist Paulsen
- Contact Person Email
- Petter.Quist-Paulsen@Stolav.no
- Site Name
- Sykehuset Ostfold HF
- Department Name
- Head of Research
- Principal Investigator Name
- Waleed Ghanima
- Principal Investigator Email
- Waleed.Ghanima@so-hf.no
- Contact Person Name
- Waleed Ghanima
- Contact Person Email
- Waleed.Ghanima@so-hf.no
Italy
- Earliest CTIS Part Ii Submission Date
- 13-03-2025
- Latest Decision Or Authorization Date
- 02-12-2025
- Processing Time Days
- 264
- Number Of Sites
- 4
- Number Of Participants
- 7
Sites
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- Medical Area Dept. - SC of Hematology
- Principal Investigator Name
- Bruno Fattizzo
- Principal Investigator Email
- bruno.fattizzo@policlinico.mi.it
- Contact Person Name
- Bruno Fattizzo
- Contact Person Email
- bruno.fattizzo@policlinico.mi.it
- Site Name
- Azienda Sanitaria Universitaria Giuliano Isontina
- Department Name
- UCO of Hematology
- Principal Investigator Name
- Francesco Zaja
- Principal Investigator Email
- francesco.zaja@asugi.sanita.fvg.it
- Contact Person Name
- Francesco Zaja
- Contact Person Email
- francesco.zaja@asugi.sanita.fvg.it
- Site Name
- Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
- Department Name
- Dep. of Hematologic and Oncologic Diseases
- Principal Investigator Name
- Francesca Palandri
- Principal Investigator Email
- francesca.palandri@unibo.it
- Contact Person Name
- Francesca Palandri
- Contact Person Email
- francesca.palandri@unibo.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Institute of Hematology
- Principal Investigator Name
- Elena Rossi
- Principal Investigator Email
- elena.rossi@unicatt.it
- Contact Person Name
- Elena Rossi
- Contact Person Email
- elena.rossi@unicatt.it
Poland
- Earliest CTIS Part Ii Submission Date
- 11-06-2025
- Latest Decision Or Authorization Date
- 02-12-2025
- Processing Time Days
- 174
- Number Of Sites
- 4
- Number Of Participants
- 6
Sites
- Site Name
- Aidport Sp. z o.o.
- Department Name
- Aidport
- Principal Investigator Name
- Michał Kwiatek
- Principal Investigator Email
- michal.kwiatek@aidport.pl
- Contact Person Name
- Michał Kwiatek
- Contact Person Email
- michal.kwiatek@aidport.pl
- Site Name
- Medicover Integrated Clinical Services Sp. z o.o.
- Department Name
- MICS Centrum Medyczne Toruń
- Principal Investigator Name
- Dominik Chraniuk
- Principal Investigator Email
- d.chraniuk@naszlekarz.pl
- Contact Person Name
- Dominik Chraniuk
- Contact Person Email
- d.chraniuk@naszlekarz.pl
- Site Name
- Pratia Hematologia Sp. z o.o.
- Department Name
- Pratia Onkologia Katowice
- Principal Investigator Name
- Sebastian Grosicki
- Principal Investigator Email
- sgrosicki@wp.pl
- Contact Person Name
- Sebastian Grosicki
- Contact Person Email
- sgrosicki@wp.pl
- Site Name
- Pratia S.A.
- Department Name
- Pratia MCM Krakow
- Principal Investigator Name
- Wojciech Jurczak
- Principal Investigator Email
- wojciech.jurczak@pratia.com
- Contact Person Name
- Wojciech Jurczak
- Contact Person Email
- wojciech.jurczak@pratia.com
Spain
- Earliest CTIS Part Ii Submission Date
- 11-06-2025
- Latest Decision Or Authorization Date
- 13-11-2025
- Processing Time Days
- 155
- Number Of Sites
- 5
- Number Of Participants
- 5
Sites
- Site Name
- Hospital Clinic De Barcelona
- Department Name
- Hematology
- Principal Investigator Name
- Anna Gaya Valls
- Principal Investigator Email
- agayav@clinic.cat
- Contact Person Name
- Anna Gaya Valls
- Contact Person Email
- agayav@clinic.cat
- Site Name
- Hospital General Universitario Morales Meseguer
- Department Name
- Hematology
- Principal Investigator Name
- María Luisa Lozano Almela
- Principal Investigator Email
- mllozano@um.es
- Contact Person Name
- María Luisa Lozano Almela
- Contact Person Email
- mllozano@um.es
- Site Name
- Clinica Universidad De Navarra (Pamplona)
- Department Name
- Hematology
- Principal Investigator Name
- Carlos Grande García
- Principal Investigator Email
- cgrandeg@unav.es
- Contact Person Name
- Carlos Grande García
- Contact Person Email
- cgrandeg@unav.es
- Site Name
- Clinica Universidad De Navarra (Madrid)
- Department Name
- Hematology
- Principal Investigator Name
- Carlos Grande García
- Principal Investigator Email
- cgrandeg@unav.es
- Contact Person Name
- Carlos Grande García
- Contact Person Email
- cgrandeg@unav.es
- Site Name
- Hospital Universitario De Burgos
- Department Name
- Hematology
- Principal Investigator Name
- Tomás José González López
- Principal Investigator Email
- tjgonzalez@saludcastillayleon.es
- Contact Person Name
- Tomás José González López
- Contact Person Email
- tjgonzalez@saludcastillayleon.es
Sponsor
Primary sponsor
- Full Name
- Eli Lilly & Co.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- IQVIA Limited
- Responsibilities
- Multiple sponsor duties (codes 1,10,11,12,13,15 (site training),2,5,6,7) as listed in sponsorDuties
- Name
- Q Squared Solutions Limited
- Responsibilities
- Sponsor duty code 4
- Name
- Q Squared Solutions LLC
- Responsibilities
- Sponsor duty code 4
Third parties
- {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"code 4","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"codes 1,10,11,12,13,15 (site training),2,5,6,7","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Q Squared Solutions LLC","duties_or_roles":"code 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Oracle America Inc.","duties_or_roles":"code 7","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Pirtobrutinib
- Active Substance
- PIRTOBRUTINIB
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- prodAuthStatus: 1 (EU MP PRD10161913)
- Orphan Designation
- Yes
- Investigational Product Name
- Placebo tablets
- Modality
- Other
Related trials
Other published trials that may interest you.
- BELIMUMAB for Immune thrombocytopenia
- DAZODALIBEP for Sjögren's syndrome
- rilzabrutinib for Immune thrombocytopenic purpura | Immune Thrombocytopenia Purpura
- DONIDALORSEN for Hereditary angioedema
- ALLOGENEIC ADIPOCYTE-DERIVED MESENCHYMAL STROMAL CELLS TRANSDUCED WITH A LENTIVIRAL PROVIRUS VECTOR CONTAINING THE HUMAN CXCR4 AND IL-10 GENES for Acute graft-versus-host disease (aGVHD) | Steroid-refractory acute graft-versus-host disease | Ruxolitinib-refractory acute graft-versus-host disease