Romiplostim N01 for Platelet Recovery in Adult Hematologic Malignancies Patients Undergoing UCBT

Efficacy and Safety of a Long-acting Thrombopoietin Receptor Agonist for Platelet Recovery in Adult Hematologic Malignancies Patients Undergoing Umbilical Cord Blood Transplantation

To evaluate the efficacy and safety of Romiplostim N01 for platelet recovery after umbilical cord blood transplantation (UCBT) in patients with hematological malignancies.

Study Overview

• Status: Recruiting
• Conditions: Platelet Recovery After Umbilical Cord Blood Transplantation
• Intervention / Treatment: Drug: Romiplostim N01

Detailed Description

Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is an important means of curing a wide range of hematologic diseases, and good hematopoietic and immune reconstitution after transplantation is one of the most important factors determining the success of the transplantation.Persistent thrombocytopenia after Allo-HSCT is one of the common post-transplantation complications.This is an investigator-initiated, single-center, single-arm, Phase II clinical trial to evaluate the efficacy and safety of Romiplostim N01 for platelet recovery after UCBT in patients with hematologic tumors. The study was conducted in patients ≥18 years of age with a diagnosis of hematological neoplasia who underwent UCBT. Subjects will be screened and receive roprostin N01 from +1 to +28 days after UCBT after signing an informed consent form, and the study will focus on the cumulative PLT implantation rate at 28 days after transplantation as the primary efficacy measure. The study is planned to enroll 34 patients.

• Study Type: Interventional
• Enrollment (Estimated): 34
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Xiaoyu Zhu, Ph.D; Phone Number: 15255456091; Email: xiaoyuz@ustc.edu.cn
• Study Contact Backup: Name: Wanjie Wang, M.D; Phone Number: 18855195535; Email: wangwanjie113@mail.ustc.edu.cn

Study Locations

• China
  • Anhui
    • Hefei, Anhui, China, 230036
      • Recruiting
      • The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
      • Contact: Xiaoyu Zhu, Ph.D; Phone Number: 15255456091; Email: xiaoyuz@ustc.edu.cn
      • Contact: Wanjie Wang, M.D; Phone Number: 18855195535; Email: wangwanjie113@mail.ustc.edu.cn
      • Contact: Xiaoyu Zhu, ph.D

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age ≥12 years old, male or female;
• Patients diagnosed with hematological malignancies and undergoing UCBT;
• ECOG score ≤2;
• Voluntary participation in this clinical trial, patients fully understand the trial content and sign the informed consent

Exclusion Criteria:

• Pregnant or lactating women；
• Known allergy to Romiplostim N01;
• A history of severe thrombotic events or known risk factors for thrombosis or active thromboembolism requiring anticoagulation;
• A history of platelet dysfunction or bleeding prone disease or severe bleeding (requiring more than 2 units of red blood cell infusion or a hematocrit drop of ≥10%) within 7 days prior to screening;
• Chronic active hepatitis B and C;
• Repeat or multiple transplantation or multiple organ transplantation;
• HIV positive, EBV-DNA positive, CMV-DNA positive;
• Have severe infection or complicated with serious heart, liver, lung, kidney, neurological or metabolic diseases;
• A serious adverse event (Level 4 or 5) as defined by the Standard General Terminology for Adverse Events Version 4.0 occurred during the pre-treatment period;
• Participants participate in another clinical study with any exploratory drug or device within 30 days prior to baseline visit; Observational studies are allowed;
• Subjects with cognitive impairment or uncontrolled mental illness;
• Subjects and/or authorized family members to refuse treatment with Romiplostim N01;
• Those who are judged by the researchers as not suitable for inclusion (such as accompanying medical history, which may affect the safety of the subjects or it is estimated that the treatment cannot be adhered to due to financial problems).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Romiplostim N01	Drug: Romiplostim N01; Romiplostim N01 was administered subcutaneously once weekly from +1 day to +28 days after UCBT at a recommended starting dose of 5µg/Kg (maximum single dose not to exceed 250µg) for a total of 4 administrations, with the option of continuation of dosing/switching/discontinuing after +28 days, depending on the patient's platelet profile.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time of platelet engraftment	Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10^9/L.	28 days
The cumulative incidence of platelet engraftment at 28 days after transplantation	Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10^9/L.	28 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The cumulative incidence of platelet recovery rate at 28 days after transplantation	Platelet recovery was defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥50×10^9/L.	28 days
Total platelet transfusion during 4 weeks of treatment	Total platelet transfusion during 4 weeks of treatment	28 days
Megakaryocyte levels in bone marrow smears 4 weeks after treatment	Megakaryocyte levels in bone marrow smears 4 weeks after treatment	4 weeks
Time of platelet recovery	The Median time of platelet count ≥20×10^9/L, 50×10^9/L, 100×10^9/L without platelet transfusions	28 days
Cumulative implantation rate of the granulocyte lineage at 28 days post-transplantation	Absolute neutrophil count (ANC) ≥0.5×10^9/L for 3 consecutive days	28 days

Collaborators and Investigators

• Sponsor: Anhui Provincial Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2024
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): December 1, 2026

Study Registration Dates

• First Submitted: November 13, 2024
• First Submitted That Met QC Criteria: November 15, 2024
• First Posted (Estimated): November 18, 2024

Study Record Updates

• Last Update Posted (Estimated): November 18, 2024
• Last Update Submitted That Met QC Criteria: November 15, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Hematologic Diseases; Hematologic Neoplasms
• Other Study ID Numbers: Romiplostim N01-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No