Efficacy and Safety Study of Romiplostim N01 for Cancer Treatment-induced Thrombocytopenia (CTIT) in the Treatment of Leukemia

Evaluation of the Efficacy and Safety of Romiplostim N01 for the Treatment of Cancer Treatment-Induced Thrombocytopenia (CTIT) in Patients with Leukemia

Study Overview

• Status: Not yet recruiting
• Conditions: Cancer Treatment-induced Thrombocytopenia (CTIT)
• Intervention / Treatment: Drug: Romiplostim N01

Detailed Description

Cancer treatment-induced thrombocytopenia (CTIT) refers to a decrease in platelet count caused by antitumor therapies during cancer treatment. It is a common adverse effect of anticancer treatment, with a particularly high incidence in patients with hematologic malignancies. CTIT increases the risk of bleeding, may limit treatment options, and can ultimately compromise the effectiveness of cancer therapy and reduce long-term survival. Currently, aside from platelet transfusion, thrombopoietic agents are commonly used to manage CTIT. Studies have shown that Romiplostim demonstrates a response rate of up to 71% in patients with chemotherapy-induced thrombocytopenia from solid tumors, with 89% of patients avoiding the need for platelet transfusion, thereby significantly reducing the risk of bleeding. However, there is limited evidence and a lack of prospective clinical trials investigating the use of Romiplostim in leukemia patients with CTIT. This study aims to evaluate the efficacy and safety of Romiplostim in adult leukemia patients with CTIT, in order to provide new therapeutic options and strategies, and ultimately improve the quality of life for this patient population.

• Study Type: Interventional
• Enrollment (Estimated): 97
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Xiaoyu Zhu, Ph.D; Phone Number: 0551-62283347; Email: xiaoyuz@ustc.edu.cn
• Study Contact Backup: Name: Aijie Huang, M.D; Phone Number: 0551-62283347; Email: huangaijie@mail.ustc.edu.cn

Study Locations

• China
  • Anhui
    • Hefei, Anhui, China, 230036
      • The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
      • Contact: Aijie Huang, M.D; Phone Number: 0551-62283347; Email: huangaijie@mail.ustc.edu.cn
      • Contact: Xiaoyu Zhu, Ph.D; Phone Number: 0551-62283347; Email: xiaoyuz@ustc.cn

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Age between 18 and 75 years, inclusive, regardless of sex;
• Histologically or pathologically confirmed diagnosis of leukemia;
• Patients with cancer treatment-induced thrombocytopenia (CTIT) in patients with leukemia due to antitumor therapy, with platelet count < 50×10⁹/L;
• Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2;
• Subjects of childbearing potential agree to use reliable methods of contraception throughout the study period (including male or female condoms, contraceptive foam, gel, film, cream, suppository, abstinence, or intrauterine device);
• Patients considered by the investigator to potentially benefit from the study treatment;
• Voluntarily agrees to participate in the clinical trial, is fully informed of the study procedures, and has signed the written informed consent form.

Exclusion Criteria:

• Pregnant or breastfeeding women;
• Known hypersensitivity to Romiplostim N01;
• Presence of hematologic disorders other than cancer treatment-induced thrombocytopenia (CTIT) caused by leukemia treatment, including but not limited to primary immune thrombocytopenia, myeloproliferative disorders, multiple myeloma, or myelodysplastic syndromes;
• History of thrombocytopenia due to causes other than CTIT within 6 months prior to screening, including but not limited to chronic liver disease, hypersplenism, infections, or bleeding disorders;
• History of severe thrombotic events or known risk factors for thrombosis, or active thromboembolism requiring anticoagulation therapy;
• Severe bleeding within 2 weeks prior to screening (requiring more than 2 units of red blood cell transfusion or a sudden ≥10% drop in hematocrit);
• Use of thrombopoietin receptor agonists (e.g., eltrombopag), recombinant human thrombopoietin (rhTPO), or interleukin-11 (IL-11) within 1 month prior to screening;
• HIV infection;
• Chronic active hepatitis B or hepatitis C infection;
• Presence of severe infection or serious comorbidities involving the heart, liver, lungs, kidneys, nervous system, or metabolic diseases;
• Participation in any investigational drug or device clinical trial within 28 days prior to baseline visit;
• Subjects with cognitive impairment or uncontrolled psychiatric disorders;
• Refusal of the subject and/or legal representative to receive Romiplostim N01 treatment;
• Deemed unsuitable for enrollment by the investigator (e.g., comorbid conditions that may compromise subject safety or anticipated treatment non-adherence due to financial or other constraints).

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; Romiplostim N01 will be administered once weekly via subcutaneous injection upon the occurrence of Grade 3 thrombocytopenia (platelet count < 50×10⁹/L) following leukemia treatment. The recommended initial dose is 5 µg/kg, with a maximum single dose not exceeding 250 µg. A maximum of 8 doses may be administered during the treatment period.	Drug: Romiplostim N01; Romiplostim N01 will be administered via subcutaneous injection once weekly when patients develop Grade 3 thrombocytopenia (platelet count < 50×10⁹/L) following leukemia treatment. The recommended initial dose is 5 µg/kg, with a maximum single dose not exceeding 250 µg. Treatment may continue for up to 8 doses.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Platelet response at Week 2 (PLT ≥ 50×10⁹/L)	Proportion of patients achieving a platelet count ≥ 50×10⁹/L at 2 weeks after initiation of treatment.	2 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Early Platelet Response Rate (within 7 days)	Proportion of patients achieving a response within 7 days of treatment initiation (Response defined as: no requirement for platelet transfusion and either a platelet count increase to ≥ 50×10⁹/L, at least a two-fold increase from baseline, or a platelet count ≥ 100×10⁹/L.)	7 days
Median time to platelet recovery without transfusion	Median time to achieve platelet counts of ≥ 50×10⁹/L and ≥ 100×10⁹/L without platelet transfusion.	8 weeks
Total platelet transfusion during treatment	Total platelet transfusion during treatment	8 weeks
Platelet nadir during treatment	Nadir platelet count during the treatment period (from initiation to end of treatment)	8 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of adverse events	Incidence of adverse events	Within 28 days after treatment discontinuation
Incidence of bleeding events	Incidence of bleeding events (based on World Health Organization bleeding assessment scale)	Within 28 days after treatment discontinuation
Survival outcomes	Relapse-Free Survival (RFS), Overall Survival (OS), and Cumulative Incidence of Relapse (CIR)	Within 28 days after treatment discontinuation

Collaborators and Investigators

• Sponsor: Anhui Provincial Hospital
• Investigators: Principal Investigator: Xiaoyu Zhu, Ph.D, The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)

Study record dates

Study Major Dates

• Study Start (Estimated): March 31, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): February 28, 2027

Study Registration Dates

• First Submitted: March 21, 2025
• First Submitted That Met QC Criteria: March 21, 2025
• First Posted (Actual): March 27, 2025

Study Record Updates

• Last Update Posted (Actual): March 28, 2025
• Last Update Submitted That Met QC Criteria: March 27, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cytopenia; Hematologic Diseases; Blood Platelet Disorders; Thrombocytopenia
• Other Study ID Numbers: Romiplostim N01-002

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