Romiplostim N01 for Platelet Recovery After Haploidentical HSCT

Официальное название

Efficacy and Safety of Romiplostim N01 in Promoting Platelet Reconstruction After Haploidentical Allogeneic Stem Cell Transplantation in Patients With Hematologic Malignancies

Обзор

This is a prospective, randomized, controlled clinical study designed to evaluate the efficacy and safety of Romiplostim N01 in promoting platelet engraftment after haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) in patients with hematologic malignancies. A total of 130 patients who undergo haplo-HSCT for acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or other hematologic malignancies will be enrolled and randomized 1:1 into a treatment group and a control group. The treatment group will receive Romiplostim N01 subcutaneously once weekly at a starting dose of 5 µg/kg, with dose adjustments based on platelet counts (maximum 10 µg/kg), for up to 4 weeks or until platelet counts reach ≥100 × 10⁹/L. The control group will not receive rh-TPO or any thrombopoietin receptor agonist (TPO-RA) therapy. Supportive care including transfusions and growth factors (G-CSF, ESA) is allowed in both groups. The primary endpoint is the cumulative platelet engraftment rate by day +21 post-transplant, defined as sustained platelet counts \> 20 × 10⁹/L for at least 7 consecutive days without transfusion. Secondary endpoints include median time to platelet engraftment, median time to achieve platelet counts ≥ 50 × 10⁹/L and ≥ 100 × 10⁹/L, total platelet transfusion volume, erythroid and neutrophil responses within 4 weeks, and overall hematopoietic recovery. Safety endpoints include the incidence of adverse events, thromboembolic events, and treatment-related serious adverse events. The study aims to determine whether early administration of Romiplostim N01 can accelerate platelet recovery and reduce bleeding risk in patients undergoing haplo-HSCT, thereby improving post-transplant outcomes.

Вмешательства

• Препарат Romiplostim N01
  Romiplostim N01 is a thrombopoietin receptor agonist (TPO-RA) administered subcutaneously once weekly to promote platelet recovery after haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT). The starting dose is 5 µg/kg, adjusted up to 10 µg/kg according to platelet response. Treatment continues for up to 4 weeks or until platelet counts reach ≥100 × 10⁹/L without transfusion. Standard post-transplant supportive care is provided to all participants.
• Другое Standard Supportive Care
  Participants in the control arm will receive standard post-transplant supportive care, including transfusions, growth factors (G-CSF, ESA), and infection prophylaxis as clinically indicated, but will not receive Romiplostim or any other thrombopoietin receptor agonist.

Первичные конечные точки

• Cumulative Platelet Engraftment Rate by Day +21 after Haploidentical HSCT [Срок оценки: Within 21 days after transplantation]

Вторичные конечные точки (6)

• Time to Platelet Engraftment [Срок оценки: Up to 60 days after transplantation]
• Proportion of Patients Achieving Platelet Counts ≥50 × 10⁹/L and ≥100 × 10⁹/L [Срок оценки: Up to 60 days after transplantation]
• Median Time to Achieve Platelet Count ≥100 × 10⁹/L during the 4-Week Treatment Period [Срок оценки: Within 4 weeks after initiation of Romiplostim N01 treatment]
• Total Platelet Transfusion Volume [Срок оценки: Up to 60 days after transplantation]
• Proportion of Participants with Erythroid Response during the 4-Week Treatment Period [Срок оценки: Within 4 weeks after initiation of Romiplostim N01 treatment]
• Proportion of Participants with Neutrophil Response during the 4-Week Treatment Period [Срок оценки: Within 4 weeks after initiation of Romiplostim N01 treatment]

Критерии участия

Критерии включения

Patients with malignant hematologic diseases scheduled to undergo haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) Age ≥18 years, male or female. ECOG performance status 0-1. Estimated life expectancy >6 months. Adequate renal function, defined as:serum creatinine ≤1.5 × upper limit of normal (ULN);Blood urea nitrogen (BUN) ≤1.5 × ULN.

Adequate hepatic function, defined as:Alanine aminotransferase (ALT) ≤2 × ULN.Aspartate aminotransferase (AST) ≤2 × ULN.Total bilirubin ≤1.5 × ULN.

Ability to understand and sign informed consent, and willingness to comply with all study requirements.

Критерии исключения

Uncontrolled active infection or other active malignancy that could interfere with study participation.

Severe cardiovascular disease, including:New York Heart Association (NYHA) Class III-IV heart failure;Uncontrolled hypertension or hypotension;History of or high risk for thromboembolic events.

Receiving anticoagulation therapy for thrombotic events. Known hypersensitivity to romiplostim or similar agents. Use of rh-TPO or any thrombopoietin receptor agonist (TPO-RA) within 30 days prior to enrollment.

Participation in another interventional clinical study within 30 days prior to enrollment.

Any other condition that, in the investigator's judgment, makes the patient unsuitable for the study.

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

• The First Affiliated Hospital, College of Medicine, Zhejiang University — Ханчжоу

Идентификаторы

NCT: NCT07321626 · ZJU-HSCT-ROMN01

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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