Clinical trial NCT06898983: Efficacy and Safety Study of Romiplostim N01 for Cancer Treatment-induced Thrombocytopenia (CTIT) in the Treatment of Leukemia
Official title: Efficacy and Safety Study of Romiplostim N01 for Cancer Treatment-induced Thrombocytopenia (CTIT) in the Treatment of Leukemia
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Cancer Treatment-induced Thrombocytopenia (CTIT)
Lead sponsor: Anhui Provincial Hospital
Sponsor class: OTHER_GOV
First posted: 2025-03-27
Enrollment: 97
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

Eligibility criteria: 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).

Primary outcomes:
measure: Platelet response at Week 2 (PLT ≥ 50×10⁹/L)
timeFrame: 2 weeks
Secondary outcomes:
measure: Early Platelet Response Rate (within 7 days)
timeFrame: 7 days
measure: Median time to platelet recovery without transfusion
timeFrame: 8 weeks
measure: Total platelet transfusion during treatment
timeFrame: 8 weeks
measure: Platelet nadir during treatment
timeFrame: 8 weeks
Other outcomes:
measure: Incidence of adverse events
timeFrame: Within 28 days after treatment discontinuation
measure: Incidence of bleeding events
timeFrame: Within 28 days after treatment discontinuation
measure: Survival outcomes
timeFrame: Within 28 days after treatment discontinuation
Locations:
city: Hefei
country: China
facility: The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
state: Anhui
Study officials:
affiliation: The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
name: Xiaoyu Zhu, Ph.D
role: PRINCIPAL_INVESTIGATOR
References: