Romiplostim N01 for Platelet Recovery After Haploidentical HSCT

NCT ID: NCT07321626

Last Updated: 2026-01-07

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 130 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-06-01
• Study Completion Date: 2027-12-31

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Romiplostim N01 Treatment Arm

Group Type: EXPERIMENTAL

Romiplostim N01

Intervention Type: 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

Intervention Type: 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.

Standard Care Control Arm

Group Type: ACTIVE_COMPARATOR

Standard Supportive Care

Intervention Type: 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.

Interventions

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.

Intervention Type: DRUG

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.

Intervention Type: OTHER

Eligibility Criteria

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 hepatic and renal function, defined as:

Serum creatinine ≤1.5 × upper limit of normal (ULN); Blood urea nitrogen (BUN) ≤1.5 × ULN; ALT ≤2 × ULN; 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

First Affiliated Hospital of Zhejiang University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

The First Affiliated Hospital, College of Medicine, Zhejiang University

Hangzhou, , China

Site Status: RECRUITING

Countries

China

Facility Contacts

Yi Luo, MD

Role: primary

luoyijr@zju.edu.com

86-1366660

Other Identifiers

ZJU-HSCT-ROMN01

Identifier Type: -

Identifier Source: org_study_id