Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment

Официальное название

A Multi-center, Open-label, Single-arm Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment

Обзор

This is a multinational, open label, single arm study that will evaluate the impact of early multi-immune modulation with rilzabrutinib in adult ITP patients who failed first-line treatment. The study includes a screening period (up to 8 weeks), a primary analysis period (up to 28 weeks), a long-term extension period for selected participants (28 weeks) and a 24-week follow-up period only for eligible participants.

Вмешательства

• Препарат rilzabrutinib
  Pharmaceutical form:Tablet-Route of administration:Oral

Первичные конечные точки

• Durable platelet response [Срок оценки: Until Week 28]

Вторичные конечные точки (6)

• Overall platelet response [Срок оценки: Until Week 28]
• Duration of platelet response [Срок оценки: Until Week 28]
• Change from baseline in the immune thrombocytopenia bleeding scale (IBLS) score at the end of week 28 [Срок оценки: Until Week 28]
• Percentage of participants able to discontinue or reduce CS dose by at least 50% or to <5 mg/day (prednisone equivalent) from baseline at the end of week 28 [Срок оценки: Until Week 28]
• Frequency and severity of treatment emergent adverse events (TEAEs, including serious adverse events [SAEs], bleeding TEAEs, adverse event of special interest [AESI] and adverse events leading to discontinuation) [Срок оценки: Until Week 80]
• Percentage of participants with potential clinical significant abnormal (PCSA) [Срок оценки: Until Week 80]

Критерии участия

Критерии включения

• Male or female participants aged 18 years and older with a documented diagnosis of primary ITP in the medical history
• Participant received at least one course of first-line therapy and had a history of response while on treatment
• Participant has loss of response, relapse, or steroid dependency

Критерии исключения

• Participants with Secondary ITP
• Participants with Evans syndrome or history of myelodysplastic syndrome
• Participants with history of lymphoma, leukemia, or any malignancy within the past 5 years except for non-melanoma skin malignancy.
• Participants with history of solid organ transplant
• Participants with history of coagulation or bleeding disorders other than ITP, including genetic conditions, other than ITP
• Participant received advanced therapy for ITP or was splenectomized
• Pregnancy or nursing The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 12 центров

• Community Health Partners (CHP) - Community Medical Oncology Specialists (University Oncol — Clovis
• University of Southern California (USC) — Los Angeles
• Tulane University Health Sciences Center - Tulane Avenue-Investigational Site Number: 8400 — New Orleans
• Massachusetts General Hospital Cancer Center — Boston
• Beth Israel Deaconess Medical Center — Boston
• Mass General Brigham Cancer Institute — Danvers
• University of Michigan Health - Michigan Medicine - University Hospital-Investigational Si — Ann Arbor
• Mayo Clinic_Investigational Site Number: 8400009 — Rochester
• … и ещё 4 центра

Испания · 7 центров

• Investigational Site Number : 7240001 — Barcelona
• Investigational Site Number : 7240002 — Burgos
• Investigational Site Number : 7240007 — Madrid
• Investigational Site Number : 7240008 — Madrid
• Investigational Site Number : 7240005 — Murcia
• Investigational Site Number : 7240003 — San Cristóbal de La Laguna
• Investigational Site Number : 7240004 — Seville

Италия · 5 центров

• Investigational Site Number : 3800002 — Meldola
• Investigational Site Number : 3800004 — Milan
• Investigational Site Number : 3800006 — Naples
• Investigational Site Number : 3800007 — Torino
• Investigational Site Number : 3800003 — Vicenza

Франция · 4 центра

• Investigational Site Number: 2500003 — Créteil
• Investigational Site Number : 2500001 — Dijon
• Investigational Site Number : 2500002 — Pessac
• Investigational Site Number : 2500004 — Toulouse

Польша · 3 центра

• Investigational Site Number : 6160002 — Gdansk
• Investigational Site Number : 6160003 — Skorzewo
• Investigational Site Number : 6160004 — Słupsk

Венгрия · 2 центра

• Investigational Site Number : 3480001 — Budapest
• Investigational Site Number : 3480002 — Kaposvár

Австрия · 1 центр

• Investigational Site Number : 0400001 — Vienna

Чехия · 1 центр

• Investigational Site Number : 2030002 — Prague

Германия · 1 центр

• Investigational Site Number : 2760004 — Leipzig

Идентификаторы

NCT: NCT07007962 · LPS18573 · U1111-1320-4669

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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