JPRN-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.0 个研究点目标入组 29 人开始时间: 2020年2月28日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 29
研究概览
简要总结
This phase 2 study demonstrated ropeginterferon alfa-2b to be a safe and efficacious treatment option in Japanese patients with PV, regardless of patients' estimated risk of thrombosis.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 20age old 至 ot applicable（—）
- 性别
- All
入选标准
- •1. Male or female patients more than or equal to 20 years old
- •2. Patients diagnosed with PV according to the WHO 2008 or WHO 2016 criteria
- •3. 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
- •4. Total HU treatment duration shorter than 3 years (cumulatively) at screening
- •5. 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)
- •6. 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
- •7. Hemoglobin (HGB) more than or equal to 10 g/dL at screening
- •8. Neutrophil count more than or equal to 1,500/microliter at screening
- •9. Serum creatinine less than or equal to 1.5 x ULN at screening
- •10. 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).
- •11. 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
- •12. 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
- •1. Patients with symptomatic splenomegaly
- •2. Previous use of IFN-alpha for any indication
- •3. Any contraindications or hypersensitivity to interferon-alfa
- •4. Co-morbidity with severe or serious conditions which may impact patient participation in the study in investigator's opinion
- •5. History of major organ transplantation
- •6. Pregnant or lactating females
- •7. 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
- •7-1. 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)
- •7-3. Clinically relevant pulmonary infiltrates and pneumonitis at screening, patients with a history of interstitial pulmonary disease
- •7-4. Active infections with systemic manifestations (e.g., bacterial, fungal, hepatitis B [HBV], hepatitis C [HCV], or human immunodeficiency virus [HIV]) at screening)
- •7-5. 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.
- •7-6. Uncontrolled depression
- •7-7. Previous suicide attempts or at any risk of suicide at screening
- •8. Uncontrolled diabetes mellitus (HbA1c level of more than 7% at baseline)
- •9. History of any malignancy within for the past 5 years
- •10. History of alcohol or drug abuse within the last year
- •11. History or evidence of post polycythemia vera-myelofibrosis (PPV-MF), essential thrombocythemia, or any non-PV MPN
- •12. Presence of circulating blasts in the peripheral blood within the last 3 months
- •13. 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
研究者
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