A Phase IV Single Arm, Multicenter, Open-label Study Assessing Deep Molecular Response in Adult Patients With Newly Diagnosed Philadelphia Chromosome Positive CML in Chronic Phase After Two Years of Treatment With Nilotinib 300mg BID
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 171
- 试验地点
- 1
- 主要终点
- Percentage of Participants With Deep Molecular Response MR4.5 at 24 Months of Study Treatment
研究概览
简要总结
The main purpose of this study was to evaluate the rate of deep molecular response (MR4.5) after 24 months of therapy with nilotinib in newly diagnosed patients with chronic phase chronic myeloid leukemia (CML) using EUTOS (European Treatment and Outcome Study for CML)-standardized laboratories. All participants received nilotinib 300 mg twice daily (BID).
详细描述
This was a Phase IV open-label, multi-center, single-arm study in participants with newly diagnosed Philadelphia chromosome positive (Ph+) CML in chronic phase for who nilotinib is the appropriate treatment at the discretion of the investigator.
A screening period of 2 weeks was used to assess eligibility and to taper participants off disallowed medications. Participants whose eligibility was confirmed entered a 24 months treatment phase with nilotinib 300mg BID. Nilotinib was prescribed by the investigator according to the individual needs of the participants.
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 120 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with newly diagnosed (within 6 months) Philadelphia chromosome positive CML in chronic phase
- •Patients must be previously untreated for CML with the exception of 6 months treatment with hydroxyurea and a maximum of 6 weeks treatment with imatinib
- •Adequate end organ function
- •Normal serum levels ≥ lower limit of normal (LLN) of potassium, magnesium, total calcium corrected for serum albumin or phosphorus, or correctable to within normal limits with supplements, prior to the first dose of study medication.
排除标准
- •Known impaired cardiac function like long QT syndrome, history of myocardial infarction or unstable angina in the past 12 months.
- •Patients who are pregnant or breast feeding.
- •Other inclusion/exclusion criteria might apply
研究组 & 干预措施
Nilotinib
Participants with newly diagnosed CML in chronic phase received nilotinib 300 mg BID for 24 months
干预措施: Nilotinib (Drug)
结局指标
主要结局
Percentage of Participants With Deep Molecular Response MR4.5 at 24 Months of Study Treatment
时间窗: Month 24 and Month 21 (if assessment at Month 24 was missing)
Percentage of participants who were in deep molecular response MR4.5 (IS) at 24 months measured in a standardized EUTOS (European Treatment and Outcome Study for CML) MR4.5 laboratory. MR4.5 was defined as either (i) detectable disease ≤ 0.0032% BCR-ABL (fusion gene from breakpoint cluster region and Abelson genes) (IS) or (ii) undetectable disease in cDNA with 32000-99999 ABL1 transcripts or 77000-239999 glucuronidase beta (GUSB) transcripts. Responders: Participants with a MR4.5 at 24 months, or if the assessment at this time point was missing, with a MR4.5 at 21 months Non-responders: Participants dropping out early or not providing sufficient data for any other reason. Participants who achieved MR4.5 before 24 months, but was no longer in MR4.5 at 24 months or progressed (or was no longer in MR4.5 at 21 months if evaluation at 24 months was missing). Confidence intervals were calculated based on the Exact Clopper-Pearson method.
次要结局
- Percentage of Participants With Major Molecular Response (MMR) at 12 Months of Study Treatment(Month 12)
- Percentage of Participants With Complete Cytogenetic Response (CCyR) at 6 Months of Study Treatment(Month 6)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Global Health Status (GHS)/Quality of Life (QoL)(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Cognitive Functioning(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Percentage of Participants With MR4 at 24 Months of Study Treatment.(Month 24)
- Progression-free Survival(From date of start of treatment to first documented disease progression to AP/ BC or death, assessed up to 24 months)
- Time to Progression to AP/BC(From the date of start of study treatment to the date of earliest transformation to AP/BC or CML-related death, assessed up to 24 months)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Emotional Functioning(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Physical Functioning(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Role Functioning(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Impact on Worry/Mood(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Impact on Daily Life(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Body Image Problems(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Social Functioning(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Symptom Burden(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Satisfaction With Care and Information(Baseline, month 3, month 6, month 12, month 18 and month 24)
- Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Satisfaction With Social Life(Baseline, month 3, month 6, month 12, month 18 and month 24)