Evaluation of the Accuracy and Reliability of B-Adrenergic Sweat Secretion Using an Evaporimeter to Assess CFTR Function in Cystic Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 2
- 主要终点
- Measurement of beta-adrenergic induced sweat rate using an evaporimeter can accurately and reliably determine different levels of CFTR dysfunction within a spectrum of patients expressing various degrees of CF disease.
研究概览
简要总结
The investigators hypothesize that measurement of beta adrenergic induced sweat rate using an evaporimeter can accurately and reliably determine different levels of CFTR dysfunction within a spectrum of patients expressing various degrees of cystic fibrosis disease.
The investigators overall goal is to determine whether the evaporimeter technique of measuring beta-adrenergic induced sweat rate is capable of accurately and reliably identifying different levels of CFTR dysfunction, as a prerequisite before advancing this technique as biomarker assay into clinical trials.
详细描述
Preliminary data show that following β-adrenergic stimulation, evaporimetry can reliably measure sweat secretion that is: highly reproducible in healthy controls; reduced by 50% in CF obligate heterozygotes; and absent in CF patients carrying severe mutations on both alleles. Further, test- retest experiments suggest good intra-individual reliability. All these features satisfy the required criteria for a biomarker assay that is capable of assessing small increments in CFTR function in vivo in clinical trials designed to assess the effectiveness of correctors and potentiators of CFTR channel activity. Therefore, to provide additional evidence of the value of this novel technique the investigators will determine the accuracy and reliability of evaporimetry to measure Beta-adrenergic induced sweating in subjects with a range of CFTR channel activity.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Male or female ages 18 years and older.
- •Subject with or without confirmed diagnosis of CF.
- •Written informed consent obtained from subject.
排除标准
- •Patients who are participating in clinical trials evaluating the safety, efficacy or clinical outcome of drugs that may alter the CFTR Cl channel function will be excluded.
- •Any subject with a known hypersensitivity to any agonist used in the study or subjects receiving any drug (e.g. theophylline, antihypertensive agent) that might interfere with the investigations.
- •Subjects with active dermatitis or other chronic skin condition such as psoriasis or a strong allergic history.
- •Patients with a history of cardiac disease (including arrhythmias or hypertrophic obstructive cardiomyopathy).
- •CF patients with severe malnutrition (BMI<18 kg/m2).
- •CF patients with severe lung disease (FEV1<25%).
- •Subject who has been treated for pulmonary exacerbation or other acute illness within one week of the study procedure.
- •Any medical condition that, in the opinion of the investigator, will interfere with accurate conduct of the study.
- •Subjects who are pregnant or lactating.
- •Subject who is not able to stop inhalation therapy of β -adrenergic drugs 12 hrs before starting each test.
结局指标
主要结局
Measurement of beta-adrenergic induced sweat rate using an evaporimeter can accurately and reliably determine different levels of CFTR dysfunction within a spectrum of patients expressing various degrees of CF disease.
时间窗: Up to 2 hours
The response patterns will be interpreted and classified by the study PI, as consistent or inconsistent to the published standards. The obtained measurements will be compared to the results obtained from a recently finished validation trial. According to the result of this trial the ranges were as followed: cholinergic response for all subjects 60±20 Evaporimeter units, beta-adrenergic response: Healthy control 51 ± 20 heterozygote -22 ±20, CF -1.4 ±2.
次要结局
未报告次要终点