Romiplostim N01 for Platelet Recovery After Haploidentical HSCT
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorFirst Affiliated Hospital of Zhejiang University
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
None
Disqualifiers
None
Trial design
Design model
Parallel
Treatments tested in this trial
Romiplostim N01
DrugRomiplostim 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.
Standard Supportive Care
Other interventionParticipants 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.
Treatment groups
130 Participants
are divided into 2 treatment groupsGroup A: Romiplostim N01 Treatment ArmExperimental treatment 2 interventions
Group B: Standard Care Control ArmActive comparator 1 intervention
Trial outcomes
Primary outcomes
1
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.
Time frame
Within 21 days after transplantationSecondary outcomes
1
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.
Time frame
Up to 60 days after transplantation2
Proportion of Patients Achieving Platelet Counts ≥50 × 10⁹/L and ≥100 × 10⁹/L
Time frame
Up to 60 days after transplantation3
Median Time to Achieve Platelet Count ≥100 × 10⁹/L during the 4-Week Treatment Period
Time frame
Within 4 weeks after initiation of Romiplostim N01 treatment4
Total Platelet Transfusion Volume
Time frame
Up to 60 days after transplantationOther outcomes
1
Incidence of Thrombotic or Thromboembolic Events
Time frame
Within 4 weeks after initiation of Romiplostim N01 treatment2
Incidence of Treatment-Related Adverse Events (TRAEs) and Serious Adverse Events (SAEs)
Time frame
From initiation of treatment up to 100 days after transplantation3
Incidence of Adverse Events (AEs)
Time frame
From initiation of Romiplostim N01 to 100 days after transplantationSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov