NCT03688568撤回2 期
Phase 2 Study of Imatinib in Children With Neurofibromatosis and Airway Tumors
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Quantitative Functional Airway Response
研究概览
简要总结
The purpose of this study is to look at a subset of plexiform neurofibromas and determine if the airway tumors are more sensitive to imatinib therapy. Funding Source - FDA OOPD
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
- 6 Months 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged: > 6 months and < 12 years of age.
- •Diagnosis of neurofibromatosis type 1 (NF1).
- •Presence of symptomatic airway plexiform neurofibromas ; defined by abnormal sleep study or pulmonary function testing.
- •Patients must have measurable (> 1.5 cm in two dimensions or able to assess a minimum of 3 slices) disease by magnetic resonance imaging (MRI).
- •Patients must have a Karnofsky of > 70% or Lansky of > 50% and a life expectancy of > 2 months.
- •Adequate end organ function, defined as the following:
- •total bilirubin < 1.5 x ULN, SGOT and SGPT < 2.5 x UNL, creatinine < 1.5 x ULN, ANC > 1.5 x 109/L, platelets > 100 x 109/L.
- •Patients must be able to swallow whole pills or crushed pills in a soft food such as pudding or apple sauce; or have other GI access such as a G-tube.
- •Written, voluntary informed consent/assent.
排除标准
- •Patient has received any other investigational agents within 14 days of first day of study drug dosing.
- •Patient is < 5 years free of another primary malignancy except: if the other primary malignancy is not currently clinically significant nor requiring active intervention, or if other primary malignancy is a basal cell skin cancer or a cervical carcinoma in situ. Existence of any other malignant disease is not allowed.
- •Patient with Grade III/IV cardiac problems as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study)
- •Patient has a severe and/or uncontrolled medical disease (i.e., uncontrolled diabetes, chronic renal disease, or active uncontrolled infection).
- •Patient has a known brain metastasis. Non-specific CNS changes on MRI/CT characteristic of NF1 are allowed, but not known CNS malignancies requiring therapeutic intervention.
- •Patient has known chronic liver disease (i.e., chronic active hepatitis, and cirrhosis).
- •Patient has a known diagnosis of human immunodeficiency virus (HIV) infection.
- •Patient received chemotherapy within 4 weeks (6 weeks for nitrosourea or mitomycin-C) prior to study entry.
- •Patient previously received radiotherapy to > 25 % of the bone marrow
- •Patient had a major surgery within 2 weeks prior to study entry.
- •Patient/parent with any significant history of non-compliance to medical regimens or with inability to grant reliable informed consent.
- •Patients who have or anticipate receiving permanent (or semi-permanent) metallic structures attached to their body. (e.g., braces on teeth, body piercings), which their physicians believe will interfere with the MRI.
- •Patient has an unstable airway requiring more urgent intervention or deemed unable to travel due to unstable airway by referring MD.
研究组 & 干预措施
Imatinib Mesylate Arm
Experimental
Imatinib Mesylate, given daily orally, 55 mg PO BID, if tolerated for 2 weeks increase to 110 mg/m2 BID, and further increase to 165 and final dosage to 220 mg/m2 bid if tolerated. Can continue for 12 months.
干预措施: Imatinib Mesylate (Drug)
结局指标
主要结局
Quantitative Functional Airway Response
时间窗: 12 months
Sleep study or pulmonary function test
次要结局
- Radiologic response of tumor(12 months)
- Quality of Life Assessment(12 months)
- Cytokine Biomarker(12 months)
- Inflammatory Cell Biomarker(12 months)
研究者
Kent A Robertson
Associate Professor
Indiana University
研究点 (1)
Loading locations...
相似试验
终止
2 期
Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform NeurofibromasPlexiform NeurofibromasNCT02177825St. Justine's Hospital5
已完成
早期 1 期
Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasNeurofibromatosisNF1NeurofibromasNCT01275586Indiana University6
已完成
2 期
Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2Neurofibromatosis Type IINCT01419639NYU Langone Health10
已完成
2 期
Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform NeurofibromasPlexiform NeurofibromaNeurofibromatosis Type 1NCT03231306University of Alabama at Birmingham45
已完成
2 期
Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related TumorsNeurofibromatosis 2Vestibular SchwannomaNCT00973739NYU Langone Health21