A Phase 2 Multicenter, Randomized, Open-label Study to Evaluate the Pharmacokinetic, Safety and Efficacy of Peginterferon Alfa-2b Injection in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea.

Xiamen Amoytop Biotech Co., Ltd.8 个研究点分布在 1 个国家目标入组 27 人开始时间: 2024年8月29日最近更新: 2025年11月17日

适应症

干预措施

试验速览

阶段: 2 期
状态: 招募中
发起方: Xiamen Amoytop Biotech Co., Ltd.
入组人数: 27
试验地点: 8
主要终点: Maximum concentration (Cmax)

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

This is a multicenter, randomized, open-label Phase 2 clinical study. It is aimed to enroll 27 essential thrombocytopenia (ET) patients who are resistant to or intolerant of hydroxyurea(HU). Eligible patients will be randomized to receive either Peginterferon α-2b 135 mcg or Peginterferon α-2b 180 mcg at a ratio of 1:2, and all subjects will go through a target treatment period (Weeks 1 ~ Week 48), an extension treatment period (Weeks 49 ~ Week 96) and a follow-up period (Weeks 97 ~ Week 100). Pharmacokinetics, safety, efficacy will be evaluated.

研究设计

研究类型: Interventional
分配方式: Randomized
干预模型: Parallel
主要目的: Treatment
盲法: None

入排标准

年龄范围: 18 Years 至 —（Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Male or female subjects, aged greater or equal to 18 years old at screening;
•Subjects diagnosed as high-risk ET according to the World Health Organization (WHO) 2016 criteria：1) who is older than 60 years and JAK2V617F positive at screening, 2) or who previously suffered from disease-related thrombosis or hemorrhage;
•Subjects who have previously received HU for ET, and the time interval between the last HU dose and the first dose of the study drug should not be less than 7 days;
•Interferon treatment-naïve, and for those who have previously received interferon the the time interval between the last dose of interferon and randomization should not be less than 1 month;
•Patients with confirmed hydroxyurea resistance or intolerant, as at least one of the following criteria is met:
•Platelet count remain greater than 600×10^9 /L after at least 3 months of HU treatment at a dose ≥2g/d (dose ≥2.5 g/d if subject weight > 80 kg);
•Platelet count greater than 400*10^9/L while white blood cell (WBC) count lower than 2.5*10^9/L, or platelet count greater than 400*10^9 /L while hemoglobin lower than 100 g/L at any dose of HU;
•Presence of HU-related toxicities at any dose of HU: e.g. ulcers in legs, or any unacceptable skin mucosal manifestations or fever;
•Platelet counts > 450*10^9/L at screening;
•Neutrophil count ≥1.0*10^9/L at screening;
•Haemoglobin ≥11 g/dL at screening for males and 10 g/dL for females at screening;
•There is no serious function damage in liver and kidney: total bilirubin ≤1.5 upper limit of normal (ULN), alanine aminotransferase≤2.0 ULN, aspartate aminotransferase≤2.0 ULN, prothrombin time is prolonged by less than 4 seconds, Creatinine clearance ≥50 mL/min (according to Cockcroft-Gault formula) at screening;
•Both male and female subjects must agree take an appropriate contraceptive method, including:
•Male subjects: must agree to use reliable contraception from inform consent until 6 months following the last dose of the study drug.
•Female subjects: Must meet at least one of the following conditions:
•i) Women without childbearing potential; ii) Women of childbearing potential: no pregnant or breastfeed, negative in blood pregnancy test within 4 days prior to the first dosing, and must agree to use reliable contraception from inform consent until 6 months following the last dose of the study drug;
•Subjects understand the objective, characteristic, method and possible adverse reactions of the study, voluntarily participate in this study, and sign informed consent.

排除标准

•History of any other myeloproliferative tumors, or evidence of the presence of any other myeloproliferative tumors;
•Contraindications or hypersensitivities to interferons of any of its excipients;
•Severe medical conditions or serious comorbidities that the investigators determined could jeopardize the safety or protocol adherence, e.g. New York Heart Association [NYHA] Class III-IV, congestive heart failure, symptomatic arrhythmias，pulmonary hypertension;
•History of major organ transplantation;
•Documented autoimmune disease or history of autoimmune disease at screening, e.g. medication un-controlled thyroid dysfunction, autoimmune hepatitis, idiopathic thrombocytopenic purpura, scleroderma, psoriasis, or any autoimmune arthritis;
•Clinically significant pulmonary infiltration, infectious pneumonia, and non-infectious pneumonia at screening that, in the investigator's opinion, would jeopardize the safety of the subject or their compliance with the protocol;
•Infection with systemic clinical manifestations at screening, e.g., bacteria, fungi, human immunodeficiency virus, excluding hepatitis B and/or C;
•Evidence of severe retinopathy, e.g., cytomegalovirus retinitis, symptomatic macular degeneration, or clinically significant eye disease, e.g. due to diabetes mellitus or hypertension;
•Diagnosed clinically significant depression or a history of depression and, in the investigator's opinion, previous suicide attempts or at any risk of suicide at screening;
•Diagnosed clinically significant neurological disease or a history of clinically significant neurological disease, except for a history of stable cerebral thrombosis or cerebral hemorrhage;
•History of any malignancy within 5 years (except stage 0 chronic lymphocytic leukemia, basal cell carcinoma, squamous cell carcinoma, and superficial melanoma);
•A history of alcohol or drug abuse within 1 year;
•Have used any investigational drug within 4 weeks prior to first dose of investigational drug, or not recovered from the effects of prior investigational drug administration;
•Other situations that, in the investigator's opinion, not appropriate for inclusion.

研究组 & 干预措施

Peginterferon α-2b 135 mcg dose group

Experimental

干预措施: Peginterferon α-2b injection (Drug)

Peginterferon α-2b 180 mcg dose group

Experimental

干预措施: Peginterferon α-2b injection (Drug)