LAM Pilot Study With Imatinib Mesylate
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 2
- 主要终点
- Serum VEGF-D
研究概览
简要总结
This is a phase 1 clinical trial comparing imatinib mesylate to placebo for individuals with lymphangioleiomyomatosis (LAM).
详细描述
This is a double blind, adjusted parallel design, randomized clinical trial comparing imatinib mesylate 400 mg daily or matching placebo on the primary outcome of log transformed serum VEGF-D level in patients with LAM.
Sirolimus using patients will have co-administration of Imatinib mesylate or placebo for 28 days prior to sirolimus discontinuation.
The duration of 400 mg imatinib mesylate or placebo will be 56 days, a dose reduction is allowed for toxicity.
The primary endpoint will be the change in the log transformed VEGF-D one month after monotherapy imatinib mesylate or placebo.
Total trial duration is 2 months of drug administration.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Definite or Probable LAM
- •FVC or Postbronchodilator FEV1 <90% predicted
排除标准
- •Current or planned pregnancy or lactation
- •Unwillingness to discontinue sirolimus
- •Change in the dose or use of sirolimus within the past month
- •Inability to perform spirometry
- •Allergy or intolerance of albuterol and/or ipratropium
- •Other serious illness that would impact the outcome of the study including cancer that has not received curative therapy, 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), uncontrolled diabetes, chronic renal disease, chronic liver disease, or active uncontrolled infection
- •Current lung transplant
- •Known diagnosis of human immunodeficiency virus (HIV) infection
- •Current cigarette smoking
- •Required use of warfarin, ketoconazole, itraconazole, clarithromycin, or rifampin during the 2 months of the study.
- •Unwillingness to avoid grapefruit juice or St. Johns Wort during the study.
- •Planned surgery during the 2 months of the study.
- •Patient with any significant history of non-compliance to medical regimens or with inability to grant reliable informed consent.
- •Patient has received and other investigational agents within 28 days of first day of study drug dosing.
研究组 & 干预措施
Imatinib Mesylate 400mg capsule
56 days of Imatinib mesylate 400 mg oral daily with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction to 200 mg daily is allowed for toxicity.
干预措施: Imatinib Mesylate 400Mg Capsule (Drug)
Placebo Capsule
56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity.
干预措施: Placebo - Capsule (Drug)
结局指标
主要结局
Serum VEGF-D
时间窗: Before and 1 month after initiation of monotherapy imatinib mesylate or placebo
Change in the square root of the intrasubject plasma VEGF-D
次要结局
- Adverse Events(3 months)
研究者
Charlie Strange
Professor of Pulmonary and Critical Care Medicine
Medical University of South Carolina