Safety and Efficacy of Pomalidomide in the Treatment of Refractory Cough in Patients With Idiopathic Pulmonary Fibrosis (IPF): A Pilot Study
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Outcome Measure: Cough related QOL as measured by Cough-Specific Quality of LIfe instrument (CQLQ)
研究概览
简要总结
The purpose of this study is to determine the safety and efficacy of pomalidomide over a 12 week duration in the treatment of chronic cough in patients with IPF as measured by a Cough Symptom Diary, Visual Analogue Scale for Cough Severity, Leicester Cough Questionnaire, St. George Respiratory Questionnaire, Cough-Specific Quality-of-Life Questionnaire, and adverse event reporting. There will be an option open to participants, who respond to treatment by meeting pre-determined criteria, to remain in the study for an additional 9 months or for a total of 54 weeks.
详细描述
Chronic cough is one of the most debilitating symptoms in patients with IPF. It is estimated that over 80% of patients with IPF experience clinically significant cough. The impact of chronic cough on QOL includes fatigue, embarrassment, anxiety, sleep disturbance, hoarseness, incontinence, dizziness, vomiting, rib fractures, and severe dyspnea. There is also some evidence that chronic cough may contribute to increased airway inflammation. In IPF patients, significant oxygen desaturation often accompanies coughing episodes leading to increased dyspnea, anxiety, and panic. Studies examining alternative therapeutic agents for cough in IPF patients are scarce. Cough in patients with IPF is typically refractory to traditional antitussive agents or corticosteroids.
Unfortunately, the side effects of corticosteroids combined with their lack of efficacy in prevention of disease progression in IPF makes this a less desired treatment option.
Although opiates can be effective in controlling cough, issues of sedation, respiratory depression and narcotic dependence are significant.
This is an open label, safety and efficacy pilot study of pomalidomide in 20 patients with IPF. This study will determine both the tolerability and feasibility of pomalidomide for the treatment of cough in this patient population.
Eligible patients will be selected from our Interstitial Lung Disease Clinic database which consists of patients with ILD seen in our clinic since about 2004 who have signed informed consent (previously approved by our Administrative Panel on Human Subjects in Medical Research, protocol 14054). Those patients with IPF who describe the severity of their cough as decreasing, or adversely affecting, their quality of life will be invited to participate in the study.
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
- •Understand and voluntarily sign an informed consent form.
- •>18 and < 75 years old at the time of signing the informed consent form.
- •Able to adhere to the study visit schedule and other protocol requirements.
- •Have a diagnosis of Idiopathic Pulmonary Fibrosis in accordance to American Thoracic Society guidelines.
- •Persistent cough:
- •as defined by a cough that adversely affects the patient's quality of life and has been present for at least 3 months.
- •Laboratory test results within these ranges:
- •Absolute neutrophil count >2 x 103/ul
- •Platelet count >100,000 /mm³
- •Serum creatinine < 2.0 mg/dL
- •Total bilirubin < 1.5 mg/dL
- •Diffusion capacity > 25%predicted
- •Forced vital capacity <80% predicted • AST (SGOT) and ALT (SGPT) < 2 x ULN
- •Able to take aspirin (81 or 325 mg) daily as prophylactic anti-coagulation (patients intolerant to ASA may use warfarin or low molecular weight heparin).
排除标准
- •Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
- •Females of child-bearing potential defined as a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).
- •Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- •Use of any other experimental drug or therapy within 28 days of baseline.
- •Known hypersensitivity to thalidomide or lenalidomide.
- •Any prior use of thalidomide, lenalidomide or pomalidomide (CC-4047).
- •Known positive for HIV or infectious hepatitis, type A, B or C.
- •History of deep venous thrombosis
- •History of pulmonary embolism
- •Use of the following anti-tussive agents must be discontinued 14 days prior to their baseline visit. -
- •narcotic anti-tussives
- •Treatment for infection or acute exacerbation within past 3 months
研究组 & 干预措施
Treatment
Open label
干预措施: pomalidomide (CC-4047 (Drug)
结局指标
主要结局
Outcome Measure: Cough related QOL as measured by Cough-Specific Quality of LIfe instrument (CQLQ)
时间窗: 12 weeks
次要结局
- Outcome Measure: Cough severity as measured by Leicester Cough Questionnaire (LCQ), Visual Analog Scale (VAS), St. George Respiratory Questionnaire (SGRQ)(12 wks)
研究者
Paul Mohabir
Principal Investigator
Stanford University