A Multi-center, Open-label, Pharmacokinetic Study of Oral Nilotinib in Pediatric Patients With Newly Diagnosed Chronic Phase (CP) Ph+ CML, With CP or Accelerated Phase (AP) Ph+ CML Resistant/Intolerant to Imatinib and/or Dasatinib, or With Refractory/Relapsed Ph+ ALL
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Summary of Nilotinib Steady-state PK Parameters: AUCss
研究概览
简要总结
This study will assess the pharmacokinetics of nilotinib in Ph+ CML pediatric patients that are newly diagnosed or resistant or intolerant to imatinib or dasatinib or refractory or relapsed Ph+ ALL compared to the adult populations. It will also evaluate safety and activity of nilotinib as secondary objectives.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have one of the following: newly diagnosed CP Ph+CML, CP or AP resistant/ intolerant to imatinib and/or dasatinib, or Ph+ ALL either relapsed after or refractory to standard therapy
- •adequate renal, hepatic and pancreatic function
排除标准
- •patients receiving therapy with strong CYP3A4 inhibitors and/or inducers and treatments cannot be stopped or changed to a different medication at least 14 days prior to starting study drug
- •patients receiving therapy with any medications with a known risk or possible risk to prolong the QT interval and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug.
- •gastrointestinal impairment or disease that may interfere with drug absorption
- •liver, pancreatic or severe renal disease unrelated to disease under study
- •impaired cardiac function
- •patients who received dasatinib within 3 days of starting study drug
- •patients who received imatinib within 5 days of starting study drug
- •patients receiving hydroxyurea or corticosteroids that has not been discontinued at least 1 week after initiation of nilotinib
- •patients who received hematopoietic growth factors within 7 days of starting study drug or Pegfilgrastim (Neulasta®) within 14 days of starting study drug
- •patients with Stem Cell Transplant (SCT) or Rescue without TBI: Evidence of active graft vs. host disease and < 3 months since SCT
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Group 1
1 year to < 10 years pediatric patients with newly diagnosed CP-Ph+ CML, or CP or AP-Ph+ CML resistant/intolerant to imatinib and/or dasatinib, or relapsed/refractory Ph+ ALL (acute lymphoblastic leukemia) treated at the proposed dose of 230 mg/m2 bid.
干预措施: Nilotinib (Drug)
Group 2
>= 10 years to <18 years pediatric patients with newly diagnosed CP-Ph+ CML, or CP or AP-Ph+ CML resistant/intolerant to imatinib and/or dasatinib, or relapsed/refractory Ph+ ALL (acute lymphoblastic leukemia) treated at the proposed dose of 230 mg/m2 bid.
干预措施: Nilotinib (Drug)
结局指标
主要结局
Summary of Nilotinib Steady-state PK Parameters: AUCss
时间窗: Cycle 1 Day 8 - Cycle 1 Day 28
The steady-state PK profiles of nilotinib in pediatric patients were estimated using trough sampling following multiple 230 mg/m2 bid doses
Summary of Nilotinib Non-compartmental PK Parameters: Tmax
时间窗: Cycle 1 Day 1
The full PK profiles of nilotinib in pediatric patients were assessed using serial sampling following a single 230 mg/m2 dose on Cycle 1 Day 1.
Summary of Nilotinib Non-compartmental PK Parameters: AUClast (Last = 24h)
时间窗: Cycle 1 Day 1
The full PK profiles of nilotinib in pediatric patients were assessed using serial sampling following a single 230 mg/m2 dose on Cycle 1 Day 1.
Summary of Nilotinib Non-compartmental PK Parameters: AUC0-12h
时间窗: Cycle 1 Day 1
The full PK profiles of nilotinib in pediatric patients were assessed using serial sampling following a single 230 mg/m2 dose on Cycle 1 Day 1.
Summary of Nilotinib Non-compartmental PK Parameters: Cmax
时间窗: Cycle 1 Day 1
The full PK profiles of nilotinib in pediatric patients were assessed using serial sampling following a single 230 mg/m2 dose on Cycle 1 Day 1.
Summary of Nilotinib Steady-state PK Parameters: CLF (Body Surface Area (BSA) Adjusted)
时间窗: Cycle 1 Day 8 - Cycle 1 day 28
The steady-state PK profiles of nilotinib in pediatric patients were estimated using trough sampling following multiple 230 mg/m2 bid doses
Summary of Nilotinib Steady-state PK Parameters: Cmin
时间窗: Cycle 1 Day 8 - Cycle 1 Day 28
The full PK profiles of nilotinib in pediatric patients were assessed using serial sampling following a single 230 mg/m2 dose.
次要结局
- Number of Ph+ CML Participants With Confirmed Complete Hematologic Response (CHR)(minimum of 12 cycles (28 days per cycle))
- Number of Ph+ CML Participants With Major Molecular Response (MMR)(minimum of 12 cycles (28 days per cycle))
- Number of Ph+ CML Participants With Cytogenic Response(minimum of 12 cycles (28 days per cycle))
- Efficacy Endpoints for Ph+ ALL Patients(minimum of 12 cycles (28 days per cycle))