A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia

Официальное название

A Phase 1/2, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults With Immune Thrombocytopenia

Обзор

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.

Вмешательства

• Препарат Pirtobrutinib
  Administered orally
• Препарат Placebo
  Administered orally

Первичные конечные точки

• 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 [Срок оценки: Baseline Up to Week 4]
• Phase 1-Dose Limiting Toxicity (DLT) of Pirtobrutinib [Срок оценки: 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 [Срок оценки: 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) [Срок оценки: Baseline Up to Week 16]
• Phase 1-Number of Participants with Treatment-Related Adverse Events as Assessed by Electrocardiograms (ECGs): ECG QT Interval [Срок оценки: Baseline Up to Week 16]
• Phase 2-Efficacy of Pirtobrutinib Versus Placebo [Срок оценки: Baseline Up to Week 24]

Вторичные конечные точки (8)

• Phase 1-Preliminary Efficacy of Pirtobrutinib [Срок оценки: Day 1 Up to Week 12]
• Phase 1-Evaluate the Extent of Disease Control [Срок оценки: Day 1 Up to Week 12]
• Phase 1: Pharmacokinetics (PK) of Pirtobrutinib [Срок оценки: Baseline Up to Week 16]
• Phase 2-Assess Additional Efficacy of Pirtobrutinib Versus Placebo [Срок оценки: Week 14 Up to Week 24]
• Phase 2-Evaluate the Extent of Disease Control of Pirtobrutinib Versus Placebo [Срок оценки: Baseline Up to Week 24]
• Phase 2-Evalulate the Extent of Disease Control of Pirtobrutinib Versus Placebo [Срок оценки: Baseline Up to Week 24]
• Phase 2-Describe the Use of Rescue Medications of Pirtobrutinib Versus Placebo [Срок оценки: Baseline Up to Week 24]
• Phase 2-Describe the PK of Pirtobrutinib [Срок оценки: Week 16 Up to Week 40]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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)

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Последовательный дизайн

Маскирование

Двойное слепое

Основная цель

Фундаментальное исследование

Центры проведения

США · 11 центров

• USC Norris Comprehensive Cancer Center — Los Angeles
• Stanford University — Stanford
• MedStar Georgetown University Hospital — Washington D.C.
• University of Miami Hospital and Clinics Sylvester Comprehensive Cancer Center — Miami
• Bleeding and Clotting Disorders Institute — Peoria
• Ochsner Clinic Foundation — New Orleans
• Mayo Clinic — Rochester
• Clinical Research Alliance — Westbury
• … и ещё 3 центра

Польша · 5 центров

• Pratia Onkologia Katowice — Katowice
• Pratia MCM Krakow — Krakow
• Aidport sp z o.o. — Skorzewo
• MICS Centrum Medyczne Torun — Torun
• Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu — Wroclaw

South Korea · 5 центров

• Pusan National University Hospital — Busan
• Seoul National University Hospital — Seoul
• Severance Hospital, Yonsei University Health System — Seoul
• Asan Medical Center — Seoul
• The Catholic University of Korea, Seoul St. Mary's Hospital — Seoul

Великобритания · 5 центров

• Bristol Haematology and Oncology Centre — Bristol
• St James's University Hospital — Leeds
• Leicester Royal Infirmary — Leicester
• Royal London Hospital — London
• Hammersmith Hospital — London

Китай · 4 центра

• Nanfang Hospital of Southern Medical University — Гуанчжоу
• Qilu Hospital of Shandong University — Цзинань
• Hematology Hospital of the Chinese Academy of Medical Sciences — Тяньцзинь
• Union Hospital Tongji Medical College Huazhong University of Science and Technology — Ухань

Италия · 4 центра

• Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi IRCCS — Bologna
• Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico — Milan
• Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Roma
• Azienda Sanitaria Universitaria Giuliano Isontina (ASU GI) — Trieste

Испания · 4 центра

• Hospital Clinic de Barcelona — Barcelona
• Hospital Universitario de Burgos — Burgos
• Hospital General Universitario Morales Meseguer — Murcia
• Clinica Universidad de Navarra — Pamplona

Франция · 3 центра

• Hôpital Henri Mondor — Créteil
• CHU Dijon - Hopital du Bocage — Dijon
• CHU Bordeaux - Hôpital Haut-Lévêque — Pessac

Норвегия · 3 центра

• Haukeland University Hospital — Bergen
• Sykehuset Ostfold, Kalnes — Grålum
• St. Olavs Hospital Hf, Universitetssykehuset i Trondheim — Trondheim

Дания · 1 центр

• OUH — Odense C

Идентификаторы

NCT: NCT06721013 · 27294 · J2N-MC-JZNZ

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗