Romiplostim N01 for Platelet Recovery After Haploidentical HSCT

NCT07321626 | Recruiting Phase 1

• 1 other identifier: ZJU-HSCT-ROMN01
• Study Type: interventional
• Target: 130
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Acute Myeloid Leukemia; MDS (Myelodysplastic Syndrome); Delayed Platelet Engraftment; HSCT

Outcome Measures

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.

  Within 21 days after transplantation

Secondary Outcomes (6)

• Time to Platelet Engraftment

  Up to 60 days after transplantation

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

  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

Other Outcomes (3)

• 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

• Incidence of Adverse Events (AEs)

  From initiation of Romiplostim N01 to 100 days after transplantation

Study Arms (2)

Romiplostim N01 Treatment Arm

EXPERIMENTAL

Drug: Romiplostim N01; Other: Standard Supportive Care

Standard Care Control Arm

ACTIVE COMPARATOR

Other: Standard Supportive Care

Interventions

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.

Romiplostim N01 Treatment Arm

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.

Romiplostim N01 Treatment Arm; Standard Care Control Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• First Affiliated Hospital of Zhejiang University: lead

Study Sites (1)

The First Affiliated Hospital, College of Medicine, Zhejiang University

Hangzhou, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Anemia, Refractory, with Excess of Blasts

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia, Refractory; Anemia; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 23, 2025
• First Posted: January 7, 2026
• Study Start: June 1, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: January 28, 2026

Record last verified: 2025-06

Locations

CN (1)