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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Romiplostim N01 Treatment Arm | Experimental |
| |
| Standard Care Control Arm | Active Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Romiplostim N01 | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Cumulative Platelet Engraftment Rate by Day +21 after Haploidentical HSCT | Platelet engraftment is defined as achieving a sustained platelet count >20 × 10⁹/L for at least 7 consecutive days without platelet transfusion. The cumulative engraftment rate by day +21 post-transplant will be compared between the Romiplostim N01 treatment arm and the standard care control arm. | Within 21 days after transplantation |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Platelet Engraftment | The number of days from transplantation to the first of 7 consecutive days with a platelet count >20 × 10⁹/L without transfusion support. | Up to 60 days after transplantation |
| Proportion of Patients Achieving Platelet Counts ≥50 × 10⁹/L and ≥100 × 10⁹/L |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Thrombotic or Thromboembolic Events | Within 4 weeks after initiation of Romiplostim N01 treatment | |
| Incidence of Treatment-Related Adverse Events (TRAEs) and Serious Adverse Events (SAEs) | From initiation of treatment up to 100 days after transplantation |
Inclusion Criteria:
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.
Exclusion Criteria:
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.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital, College of Medicine, Zhejiang University | Recruiting | Hangzhou | China |
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| ID | Term |
|---|---|
| D015470 | Leukemia, Myeloid, Acute |
| D000754 | Anemia, Refractory, with Excess of Blasts |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| Standard Supportive Care | Other | 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. |
|
| Up to 60 days after transplantation |
| Median Time to Achieve Platelet Count ≥100 × 10⁹/L during the 4-Week Treatment Period | Within 4 weeks after initiation of Romiplostim N01 treatment |
| Total Platelet Transfusion Volume | Up to 60 days after transplantation |
| Proportion of Participants with Erythroid Response during the 4-Week Treatment Period | Within 4 weeks after initiation of Romiplostim N01 treatment |
| Proportion of Participants with Neutrophil Response during the 4-Week Treatment Period | Within 4 weeks after initiation of Romiplostim N01 treatment |
| Incidence of Adverse Events (AEs) | From initiation of Romiplostim N01 to 100 days after transplantation |
| D006402 |
| Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000753 | Anemia, Refractory |
| D000740 | Anemia |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |