jRCT2080225099已完成2 期
Phase 2 Single Arm Study for Efficacy and Safety of Ropeginterferon alfa-2b for Japanese Polycythemia Vera (PV) Patients for Whom the Current Standard of Treatment is Difficult to Apply
PharmaEssentia Japan K.K.1 个研究点目标入组 30 人开始时间: 待定最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Proportion of subjects achieving durable phlebotomy-free complete hematological response (CHR) at Month 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 干预模型
- Open, Single arm, multi-center trial
- 主要目的
- Treatment Purpose
入排标准
- 年龄范围
- 20age old over 至 No limit（—）
- 性别
- All
入选标准
- •Male or female patients more than or equal to 20 years old
- •Patients diagnosed with PV according to the WHO 2008 or WHO 2016 criteria
- •PV patients for whom the current standard of treatment is difficult to apply. (Patients with a documented history of refractory to HU are excluded.)
- •Younger patients (long-term treatment is anticipated)
- •Patients who are categorized as low risk, but cytoreduction is recommended due to disease-related signs and symptoms (headache, dizziness, pruritus, night sweats, fatigue, erythromelalgia, vision disorders, scintillating scotoma, early satiety, abdominal distension).
- •Patients with HU intolerance
- •Total HU treatment duration shorter than 3 years (cumulatively) at screening
- •For cytoreduction naive patients only: PV in need of cytoreductive treatment, defined by fulfilling as one or more of the following criteria at baseline:
- •at least one previous well documented major cardiovascular PV-related event in the medical history
- •poor tolerance of phlebotomy (defined as a phlebotomy/ procedure-related adverse event causing significant adverse impact on the patient and limiting ability to apply phlebotomy with the intention to keep Hct less than45%)
- •frequent need of phlebotomy (more than one phlebotomy within last month prior entering the study)
- •platelet counts greater than 1,000,000/microliter (for two measurements within the month prior treatment start)
- •leukocytosis (WBC greater than 10,000/microliter for two measurements within the month prior treatment start)
- •Adequate hepatic function defined as bilirubin less than or equal to 1.5 x upper limit normal (ULN), international normalized ratio (INR) less than or equal to 1.5 x ULN, albumin more than 3.5 g/dL, alanine aminotransferase (ALT) less than or equal to 2.0 x ULN, aspartate aminotransferase (AST) less than or equal to 2.0 x ULN at screening
- •Hemoglobin (HGB) more than or equal to 10 g/dL at screening
- •Neutrophil count more than or equal to 1,500/microliter at screening
- •Serum creatinine less than or equal to 1.5 x ULN at screening
- •Hospital Anxiety and Depression Scale (HADS) score 0-7 on both subscales (Patients with a borderline of HADS score [score 7 but less than10] or patients with necessity [expected benefits are higher than the risks] based on investigators' discretion are required to receive following assessment by psychiatric specialist to confirm the eligibility for IFN-alpha therapy).
- •Males and females of childbearing potential, as well as all women less than 2 years after the onset of menopause, must agree to use an acceptable form of birth control until 28 days following the last dose of the study drug
- •Written informed consent obtained from the patient or the patient's legal representative, and ability for the patient to comply with the requirements of the study
排除标准
- •Patients with symptomatic splenomegaly
- •Previous use of IFN-alpha for any indication
- •Any contraindications or hypersensitivity to interferon-alfa
- •Co-morbidity with severe or serious conditions which may impact patient participation in the study in investigator's opinion
- •History of major organ transplantation
- •Pregnant or lactating females
- •Patients with any other medical conditions, which in the opinion of the Investigator would compromise the results of the study or may impair compliance with the requirements of the protocol
- •History or presence of thyroid dysfunction (clinical symptoms of hyper- or hypo-thyroidism) of the autoimmune origin, except late stages cases on the oral thyroid substitution therapy, where potential exacerbation under interferon therapy will not constitute any further harm to the patient
- •7-2.Documented autoimmune disease (e.g., hepatitis, idiopathic thrombocytopenic purpura [ITP], scleroderma, psoriasis, or any autoimmune arthritis)
- •Clinically relevant pulmonary infiltrates and pneumonitis at screening, patients with a history of interstitial pulmonary disease
- •Active infections with systemic manifestations (e.g., bacterial, fungal, hepatitis B [HBV], hepatitis C [HCV], or human immunodeficiency virus [HIV]) at screening)
- •Evidence of severe retinopathy (e.g., cytomegalovirus retinitis [CMV], macular degeneration) or clinically relevant ophthalmological disorder (due to diabetes mellitus or hypertension) based on the ophthalmological assessment by specialists.
- •Uncontrolled depression
- •Previous suicide attempts or at any risk of suicide at screening
- •Uncontrolled diabetes mellitus (HbA1c level of more than 7% at baseline)
- •History of any malignancy within for the past 5 years
- •History of alcohol or drug abuse within the last year
- •History or evidence of post polycythemia vera-myelofibrosis (PPV-MF), essential thrombocythemia, or any non-PV MPN
- •Presence of circulating blasts in the peripheral blood within the last 3 months
- •Use of any investigational drug(s), or investigational drug combinations less than 4 weeks prior to the first dose of study drug or not recovered from effects of prior administration of any investigational agent
结局指标
主要结局
Proportion of subjects achieving durable phlebotomy-free complete hematological response (CHR) at Month 12
时间窗: Month 12
The primary efficacy outcome measure is the proportion of subjects achieving durable phlebotomy-free complete hematologic response (CHR) at Month 12. Durable phlebotomy-free CHR is defined as any subject achieving phlebotomy-free CHR at Month 9 and maintaining the response up to Month 12. A responder in sense of a primary outcome measure is a subject who has met all the following criteria at the time points: - Hematocrit less than 45% phlebotomy-free (absence of phlebotomy during the previous 3 months) - Platelet count less than or equal to 400,000/microliter, - WBC count less than or equal to 10,000/microliter
次要结局
- Changes in Hct, WBC, Plt count and spleen size from baseline
- Proportion of subjects without thrombotic or hemorrhagic events
- Duration of response maintenance
- Time to requiring no phlebotomy
- Time required to first response
- PK of P1101
- Change of JAK2 V617F mutant allelic burden over time vs. baseline
研究者
研究点 (1)
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