Eltrombopag Plus Telitacicept vs. Eltrombopag Alone for Steroid-refractory/Relapsed ITP

Официальное название

Eltrombopag Combined With Telitacicept Versus Eltrombopag Monotherapy for the Treatment of Immune Thrombocytopenia Refractory or Relapsed to Corticosteroid Therapy: A Randomized Exploratory, Controlled, Open-label, Phase II Clinical Study

Обзор

Background: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Corticosteroids are first-line therapy, but about one-third of patients relapse or are refractory. Eltrombopag (a TPO-RA) promotes platelet production, yet some patients show no response or relapse upon discontinuation. Telitacicept, a TACI-Fc fusion protein, dual-targets BAFF and APRIL, inhibiting B cell and plasma cell function and reducing autoantibody production, potentially providing synergistic immunomodulatory benefit. Objective: To evaluate the sustained response rate of eltrombopag plus telitacicept vs. eltrombopag alone in patients with steroid-refractory/relapsed ITP. Design: Randomized, open-label, controlled, exploratory Phase II study. 40 patients planned (20 combination, 20 monotherapy). Combination group: eltrombopag + telitacicept . Monotherapy group: eltrombopag alone for 12 weeks. Monotherapy patients with no response after 4 weeks may cross over to the combination group. After 12 weeks, treatment is stopped and patients are followed until Week 24. Primary endpoint: Proportion of patients maintaining platelet count ≥30×10⁹/L with no bleeding at 24 weeks post-treatment. Secondary endpoints include platelet response rates during 12 weeks, safety, bleeding events, etc. Expected results: The combination group is expected to have a significantly higher proportion of patients achieving the primary endpoint without increased adverse events. Population: Age ≥18, diagnosed ITP ≥3 months, baseline platelets \<30×10⁹/L, prior corticosteroid failure. Safety: Monitoring for bleeding, infection, thrombosis, cytopenia, etc., with dose adjustment/cessation as per protocol. Conclusion: This study explores whether dual-targeting (platelet production + autoimmune suppression) with eltrombopag and telitacicept can provide more durable remission for steroid-refractory/relapsed ITP patients.

Вмешательства

• Препарат Eltrombopag
  eltrombopag plus telitacicept vs. eltrombopag alone

Первичные конечные точки

• Sustained Response Comparison [Срок оценки: Within 24weeks of first dose]

Вторичные конечные точки (9)

• Platelet Response Within 12 Weeks [Срок оценки: Within 12 weeks of first dose]
• Platelet ≥50×10⁹/L at Week 12 [Срок оценки: Within 12 weeks of first dose]
• Platelet ≥100×10⁹/L at Week 12 [Срок оценки: Within 12 weeks of first dose]
• Time to First Platelet Response [Срок оценки: Within 12 weeks of first dose]
• Proportion of Days with Platelet ≥30×10⁹/L [Срок оценки: Within 12 weeks of first dose]
• Platelet Response After Crossover (Monotherapy Group) [Срок оценки: With in 24 weeks of first dose]
• Safety and Tolerability [Срок оценки: Within 24 weeks of first dose]
• Time to Rescue Therapy or Platelet Decline [Срок оценки: Within 24 weeks of first dose]
• Proportion Requiring Rescue Therapy [Срок оценки: Within 24 weeks of first dose]

Критерии участия

Критерии включения

• Age ≥18 years, regardless of sex.
• Clinically diagnosed with immune thrombocytopenia for at least 3 months prior to enrollment. Platelet count <30×10⁹/L within 48 hours before the first dose of study drug.
• Previous failure (ineffective, unable to maintain response, or relapse) to first-line standard corticosteroid therapy for ITP as recommended by guidelines.
• Any prior emergency treatment for ITP (e.g., corticosteroids, platelet transfusion, intravenous immunoglobulin) must have been completed at least 2 weeks before the first dose.
• Patients receiving maintenance corticosteroid therapy must be on a stable dose for at least 2 weeks prior to the first dose; patients receiving immunosuppressants (e.g., azathioprine, danazol, cyclosporine A, tacrolimus, sirolimus, etc.) must be on a stable dose for at least 4 weeks prior to the first dose; anti-CD20 antibody therapy must have been completed >3 months prior.

Understand the study procedures and voluntarily provide written informed consent.

Критерии исключения

• Received anti-CD20 antibody therapy within 3 months.
• Uncontrolled primary disease of vital organs, such as malignant tumors, liver failure, heart failure, renal failure, etc.
• Positive for HIV.
• Uncontrolled active viral or bacterial infections, including positive for hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus, or syphilis.
• Extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.
• Currently have cardiac disease requiring treatment, arrhythmia, or hypertension poorly controlled as judged by the investigator.
• Patients with thrombotic diseases such as pulmonary embolism, thrombosis, atherosclerosis, etc.
• Patients with mental disorders who are unable to give informed consent or undergo study procedures and follow-up normally.
• Patients whose toxic symptoms from prior treatment before enrollment have not yet resolved.
• Other serious diseases that may limit the patient's participation in this study (e.g., poorly controlled diabetes; severe cardiac insufficiency; myocardial infarction, unstable arrhythmia, or unstable angina within the past 6 months; gastric ulcer; active autoimmune disease, etc.).
• Pregnant women, suspected pregnancy (positive urinary human chorionic gonadotropin pregnancy test at screening), or lactating patients.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Ethics Committee of Blood disease hospital, Chinese Academy of Medical Sciences — Тяньцзинь

Идентификаторы

NCT: NCT07687134 · TACI-ITP-2026

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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