A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Study to Assess Efficacy and Safety of Ropeginterferon Alfa-2b (P1101) in Adult Patients With Pre-fibrotic/Early Primary Myelofibrosis (PMF) or Overt PMF at Low or Intermediate-1 Risk According to DIPSS Plus

PharmaEssentia Japan KK0 个研究点目标入组 20 人开始时间: 待定最近更新: 2025年3月24日

适应症

试验速览

阶段: 不适用
状态: 招募中
发起方: PharmaEssentia Japan KK
入组人数: 20
主要终点: Clinically relevant complete hematologic response (CrCHR)

研究概览研究设计入排标准结局指标研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional
分配方式: Randomized Controlled Trial
干预模型: Parallel Assignment
主要目的: Treatment Purpose
盲法: Double Blind

入排标准

年龄范围: 18age old over 至 No limit（—）
性别: All

入选标准

•1.Male or female patients aged >=18 years at the time of signing the informed consent form;
•2.Patients with pre-fibrotic/early PMF (Pre-PMF) or overt primary myelofibrosis at low to intermediate-1 risk according to DIPSS plus, diagnosed according to WHO 2016 or 2022 classification;
•3.With good liver function at screening, which is defined as total bilirubin =<1.5 x upper limit of normal (ULN), international normalized ratio (INR) =<1.5 x ULN, albumin >3.5 g/dL, alanine aminotransferase (ALT) =<2.0 x ULN, and aspartate aminotransferase (AST) =<2.0 x ULN;
•4.Hgb >=10.0 g/dL at screening;
•5.Neutrophil count >=1.0 x 10^9/L at screening;
•6.Creatinine clearance rate >=30 mL/min at screening (according to the Cockcroft-Gault formula);
•7.Females of childbearing potential, as well as all women <2 years after the onset of menopause, must agree to use an acceptable form of birth control until 60 days following the last dose of the study drug, and females must agree to not breastfeed during the study;
•8.Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.

排除标准

•1.Any known contraindications to interferon alfa or hypersensitivity to interferon alfa
•2.Patients with prior interferon therapy having poor tolerability or lack of efficacy to the previous interferon therapy per investigator's judgement;
•3.Patients with an ongoing cytoreduction (e.g., HU or IFN-alfa) at the time of screening if, in the Investigator's opinion, randomizing them into the placebo arm will lead to immediate rebound increase of peripheral blood counts and thus may jeopardize their health status;
•4.With severe or serious diseases that, in the Investigator's opinion, may affect the patient's participation in this study;
•5.History of major organ transplantation;
•6.Pregnant or breastfeeding women;
•7.Patients with any other diseases that will affect the study results or may weaken the compliance to protocol per the Investigator's judgment;
•8.Use any investigational drug <4 weeks prior to the first dose of study drug, or not recovered from effects of prior administration of any investigational drug.
•9.Eligible for JAK inhibitor therapy at screening.

结局指标

主要结局

Clinically relevant complete hematologic response (CrCHR)

时间窗: 56 weeks

The CrCHR is defined as: - Platelet count =<400 x 10^9/L, and - White blood cells (WBC) =<10 x 10^9/L, and - Peripheral blood: Hemoglobin (Hgb) >10.0 g/dL, and - Absence of major thrombotic events, and - No progression to secondary acute myeloid leukemia (AML).

Symptom endpoint

时间窗: 56 or possibly 80 weeks

The symptom endpoint is defined as: - No progression on clinical symptoms based on the MFSAF Total Symptom Score (TSS) v4.0; no progression on clinical symptoms defined as: - If baseline TSS score =<10, TSS score stays =<10; or - If baseline TSS score >10, no increase >50% in TSS score.

次要结局

未报告次要终点

研究者

发起方

PharmaEssentia Japan KK

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