NCT01493414已完成3 期
An Open-label, Multicenter, Expanded Access Study of INC424 for Patients With Primary Myelofibrosis (PMF) or Post Polycythemia Myelofibrosis (PPV MF) or Post-essential Thrombocythemia Myelofibrosis (PET-MF).
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 2,233
- 试验地点
- 1
- 主要终点
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 5 Years
研究概览
简要总结
The primary objective of this study was to collect additional safety of INC424 in patients with Primary Myelofibrosis, Post Polycythemia Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis, who either received prior treatment with commercially available agents or who have never received treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 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
- •Patients must not be eligible for another ongoing INC424 clinical trial.
- •Patients must be diagnosed with PMF, PPV MF or PET-MF, according to the 2008 revised International Standard Criteria, irrespective of JAK2 mutation status..
- •Patients with PMF requiring therapy must be classified as high risk (3 prognostic factors) OR intermediate risk level 2 (2 prognostic factors, no more), OR intermediate risk level 1 (1 prognostic factor, no more) with an enlarged spleen (assessment to occur at the Screening Visit).
- •The prognostic factors, defined by the International Working Group are:
- •Age > 65 years;
- •Presence of constitutional symptoms (weight loss, fever, night sweats);
- •Marked anemia (Hgb < 10g/dL)*;
- •Leukocytosis (history of white blood cell (WBC) > 25 x109/L);
- •Circulating blasts > 1%. * A hemoglobin value < 10 g/dL must be demonstrated during the Screening Visit for patients who are not transfusion dependent. Patients receiving regular transfusions of packed red blood cells will be considered to have hemoglobin < 10 g/dL for the purpose of evaluation of risk factors.
- •Patients with Intermediate-1 disease and splenomegaly must have a palpable spleen measuring 5 cm or greater from the costal margin to the point of greatest splenic protrusion.
- •Patients must have a peripheral blood blast count of < 10%.
- •Patients with an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or
- •Fedratinib pretreated patients with documented complete physical examination including full neurologic examination and cardiology assessment, thiamine level testing, and MRI of the brain if indicated based on signs or symptoms. Patients pretreated with fedratinib should have completed or be receiving thiamine supplementation according to the investigator's instructions.
排除标准
- •Patients eligible for hematopoietic stem cell transplantation (suitable candidate and a suitable donor is available).
- •Patients with history of malignancy in past 3 years except for treated, early-stage squamous or basal cell carcinoma in situ.
- •Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral INC424 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection).
- •Patients with cardiac disease which in the Investigator's opinion may jeopardize the safety of the patient or the compliance with the protocol.
- •Patients with currently uncontrolled or unstable angina, rapid or paroxysmal atrial fibrillation or recent (approximately 6 months) myocardial infarction or acute coronary syndrome.
- •Patients with clinically significant bacterial, fungal, parasitic or viral infection which require therapy. Patients with acute bacterial infections requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed.
- •Patients with known active hepatitis A, B, C or who are HIV-positive.
- •Patients with inadequate bone marrow reserve at the Baseline visit as demonstrated by:
- •Absolute neutrophil count (ANC) ≤ 1000/µL.
- •Platelet count < 50,000/µL without the assistance of growth factors, thrombopoietic factors or platelet transfusions.
- •Patients with any history of platelet counts < 50,000/µL or ANC < 500/µL except during treatment for a myeloproliferative disorder or treatment with cytotoxic therapy for any other reason.
- •In the case of ruxolitinib pretreated patients, ruxolitinib primary resistant patients defined as:
- •No spleen reduction within the first 12 weeks after front line therapy with ruxolitinib.
- •No reduction in symptoms within the first 12 weeks after first-line treatment with ruxolitinib.
- •In the case of ruxolitinib pretreated patients, patients discontinuing ruxolitinib due to a Grade 4 Adverse event (AE) related or suspected to be related to ruxolitinib.
研究组 & 干预措施
INC424
Experimental
5 - 25 mg twice a day (BID)
干预措施: INC424 (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 5 Years
时间窗: Baseline up to approximately 5 years
An adverse event (AE) is any untoward medical occurrence in a clinical trial participant regardless of causal relationship to study drug and regardless whether study drug has been administered. A serious adverse event (SAE) is any untoward medical occurrence or effect that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability / incapacity, is a congenital anomaly / birth defect or is medically important due to other reasons than the above mentioned criteria. A non-serious AE is any AE that does not meet the criteria above.
次要结局
- Percentage of Participants With at Least 50% Reduction in Spleen Length(Baseline up to approximately 5 years)
- Number of Participants With Best Overall Response (BOR) up to 5 Years According to Spleen Length(Baseline up to approximately 5 years)
- Change in Functional Assessment of Cancer Therapy (FACT-TOI, FACT-G) and FACT-Lymphoma (FACT-Lym) Total Scores Measured at Baseline and Week 48(Baseline and Week 48)
- Change in Eastern Cooperative Oncology Group (ECOG) Performance Status From Baseline to Worst Post-baseline ECOG Status up to 5 Years(Baseline up to approximately 5 years)
- Time to First Improvement in FACT-Lym, FACIT-Fatigue Score and ECOG Performance Status(Baseline up to approximately 5 years)
- Medical Resource Utilization up to 5 Years(Baseline up to approximately 5 years.)
研究者
研究点 (1)
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