A Study of Ianalumab in Addition to Eltrombopag in Pediatric Patients With Primary ITP Who Failed Corticosteroids.

Официальное название

A Phase 2, Open-label, Single-arm Study of Ianalumab in Addition to Eltrombopag in Pediatric Patients With Primary Immune Thrombocytopenia Who Had an Insufficient Response to or Relapsed After First-line Corticosteroid Treatment (VAYHIT-P).

Обзор

The purpose of this study is to assess the efficacy, safety and pharmacokinetics (PK) of ianalumab (VAY736) in addition to eltrombopag treatment; and to inform the dose of ianalumab in pediatric patients (5 to \<18 years of age) with primary ITP who have had an insufficient response to or relapsed after first-line treatment with corticosteroids.

Подробное описание

The study will consist of 3 phases:

* Screening phase * Treatment phase * Follow-up phase consisting of Efficacy follow-up phase and Safety follow-up phase

Вмешательства

• Биопрепарат Ianalumab
  Liquid in a vial Concentrate for solution for infusion
• Препарат Eltrombopag
  Film-coated tablet and also Powder for oral suspension

Первичные конечные точки

• Time to treatment failure (TTF) [Срок оценки: enrolled until end of study (up to 39 months from the last patient enrolled)]

Вторичные конечные точки (12)

• Stable response rate at 6 months (SR-6) [Срок оценки: 6 months]
• Stable response rate at 12 months (SR-12) [Срок оценки: 12 months]
• Complete Response (CR) rate [Срок оценки: from enrollment until end of study (up to 39 months from the last patient enrolled)]
• Response Rate (RR) [Срок оценки: from enrollment until end of study (up to 39 months from the last patient enrolled)]
• Time to response [Срок оценки: from enrollment until end of study (up to 39 months from the last patient enrolled)]
• Time to complete response [Срок оценки: from enrollment until end of study (up to 39 months from the last patient enrolled)]
• Duration of response [Срок оценки: from enrollment until end of study (up to 39 months from the last patient enrolled)]
• Duration of complete response [Срок оценки: from enrollment until end of study (up to 39 months from the last patient enrolled)]
• Bleeding Events [Срок оценки: from enrollment until end of study (up to 39 months from the last patient enrolled)]
• Rescue medication [Срок оценки: from enrollment until end of study (up to 39 months from the last patient enrolled)]
• Rate of participants who successfully taper and discontinue eltrombopag - being treatment failure-free [Срок оценки: End of Week 24]
• Ianalumab serum concentration (Cmax) [Срок оценки: at pre-dose of each of the 4 dosing events and end-of-infusion and first and last dose]

Критерии участия

Критерии включения

• Signed informed consent and/or assent must be obtained from the participant and/or their parent(s)/legal guardian(s) before any study-related activity or assessment is performed. Informed consent must be obtained from participants once they reach the local legal age of adulthood during the study.
• Male or female patients aged 5 to <18 years on the day of signing informed assent/consent (as appropriate for age).
• A confirmed diagnosis of primary ITP, with insufficient response to, or relapse after a firstline corticosteroid therapy with or without IVIG.
• Patients with platelet count <30 G/L for whom eltrombopag is clinically indicated (per physician's discretion) and with no contraindications to receive eltrombopag.
• Patients who are up-to-date on childhood vaccinations as per the local recommended vaccination schedule.

Критерии исключения

• Patients with ITP who received previous second-line ITP treatments (other than corticosteroid therapy with or without IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (maximum one week) before or during screening are eligible.
• Patients with key renal or hepatic laboratory abnormalities.
• Patients with other hematologic diagnosis associated with cytopenias, including Evans Syndrome.
• Patients with current or history of life-threatening bleeding due to thrombocytopenia.
• Patients who are Human Immunodeficiency Virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg), or hepatitis B core antibody (HBcAb) positive.
• Patients with known active or uncontrolled infections requiring systemic treatment during the screening period or history of recurrent clinically significant infection.
• Patients with hepatic impairment (Child-Pugh score >5, or equivalent).
• Patients with concurrent bleeding disorders or coagulation disorders and/or receiving antiplatelet or anticoagulant medication.
• Female patients who are nursing or pregnant.

Other protocol-defined inclusion/exclusion may apply.

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

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07660172 · CVAY736T12201 · 2026-525995-25

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗