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NCT Number: NCT07321626

Romiplostim N01 for Platelet Recovery After Haploidentical HSCT

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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Key information

Conditions

Acute Myeloid Leukemia Anemia Anemia, Refractory Anemia, Refractory, with Excess of Blasts Bone Marrow Diseases Delayed Platelet Engraftment HSCT Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute MDS (Myelodysplastic Syndrome) Myelodysplastic Syndromes Neoplasms Neoplasms by Histologic Type

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

The First Affiliated Hospital, College of Medicine, Zhejiang University

Hangzhou, China

Location status: Recruiting

Location contact

Yi Luo, MD

CONTACT

[email protected]

86-1366660

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

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.

Primary outcomes

  1. Cumulative Platelet Engraftment Rate by Day +21 after Haploidentical HSCT

    Time frame: Within 21 days after transplantation

    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.

Secondary outcomes

  1. Time to Platelet Engraftment

    Time frame: Up to 60 days after transplantation

    The number of days from transplantation to the first of 7 consecutive days with a platelet count >20 × 10⁹/L without transfusion support.

  2. Proportion of Patients Achieving Platelet Counts ≥50 × 10⁹/L and ≥100 × 10⁹/L

    Time frame: Up to 60 days after transplantation

  3. 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 treatment

  4. Total Platelet Transfusion Volume

    Time frame: Up to 60 days after transplantation

  5. Proportion of Participants with Erythroid Response during the 4-Week Treatment Period

    Time frame: Within 4 weeks after initiation of Romiplostim N01 treatment

  6. Proportion of Participants with Neutrophil Response during the 4-Week Treatment Period

    Time frame: Within 4 weeks after initiation of Romiplostim N01 treatment

Other outcomes

  1. Incidence of Thrombotic or Thromboembolic Events

    Time frame: Within 4 weeks after initiation of Romiplostim N01 treatment

  2. Incidence of Treatment-Related Adverse Events (TRAEs) and Serious Adverse Events (SAEs)

    Time frame: From initiation of treatment up to 100 days after transplantation

  3. Incidence of Adverse Events (AEs)

    Time frame: From initiation of Romiplostim N01 to 100 days after transplantation

Sponsors and collaborators

Lead sponsor

First Affiliated Hospital of Zhejiang University

Other

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Jan 7, 2026
Registry last updated
Jan 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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