NCT02965326招募中不适用
Personalized Therapy of Cystic Fibrosis: Set-up of Response Markers
Hôpital Necker-Enfants Malades2 个研究点 分布在 1 个国家目标入组 75 人开始时间: 2016年5月1日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 75
- 试验地点
- 2
- 主要终点
- Forced Expiratory Volume in 1 second
研究概览
简要总结
The purpose of this study is to determine which biological marker, or association of biological markers, best predict clinical response of cystic fibrosis patients to CFTR modulators.
详细描述
This study is based upon the hypothesis that clinical response of cystic fibrosis patients to CFTR modulators is correlated to in vitro responses to these drugs of epithelial cells derived from the patients, as assessed by CFTR-dependent Chloride secretion. Epithelial cells will be derived either from nasal or rectal epithelia, and consist both of cultured cells and organoids. The drugs tested will be Ivacaftor, or Lumacaftor/Ivacaftor, according to patient's treatment. Results of these assays will be compared with response to treatment at 6 and 12 months, assessed by clinical response and in vivo assay of CFTR function.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Cystic fibrosis patients treated by CFTR modulators (Ivacaftor or the association Ivacaftor-Lumacaftor)
- •Cystic fibrosis patients non treated by CFTR modulators
- •Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations
排除标准
- •pregnant or lactating women
- •contraindication to nasal swab
- •contraindication to rectal biopsy
结局指标
主要结局
Forced Expiratory Volume in 1 second
时间窗: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
Respiratory Function test
次要结局
- abdominal ultrasonography(treatment initiation, 1 year and every year)
- Dynamometry(initiation , 1 month, 3 Months, 6 months and every 6 months)
- Sweat test(initiation ,1 month)
- Chest CT scan(initiation, 3 years and 5 years)
- calprotectin in sputum(initiation, 7 days, 1 month, 6 months, 1 year and every 6 months)
- IL-8 in sputum(initiation, 7 days, 1 month, 6 months, 1 year and every 6 months)
- Amylase in blood(initiation, 1 month, 3 Months, 6 months and every 6 months)
- proteomics of exhalate(initiation, 7 days, 1 month, 6 months, 1 year and every 6 months)
- proton density fat fraction(treatment initiation, 1 year)
- CFTR activity in intestinal epithelium/bicarbonate(initiation of treatment and repeated of cell culture failure)
- proteomics in blood(initiation, 7 days, 1 month, 6 months, 1 year and every year)
- metabolomics in blood(initiation, 7 days, 1 month, 6 months, 1 year and every year)
- metabolomics in urine(initiation, 7 days, 1 month, 6 months, 1 year and every year)
- Forced Expiratory Flow 25-75(initiation, 7 days, 1 month, 6 months, 1 year and every 6 months)
- Forced Vital Capacity(initiation, 7 days, 1 month, 6 months, 1 year and every 6 months)
- elastase in sputum(initiation, 7 days, 1 month, 6 months, 1 year and every 6 months)
- Calprotectin in blood(initiation, 1 month, 3 Months, 6 months and every 6 months)
- IL-8 in blood(initiation, 1 month, 3 Months, 6 months and every 6 months)
- sputum bronchial microorganism colonization(initiation , 1 month, 3 Months, 6 months and every 6 months)
- fecal elastase(initiation, at 6 months and every year)
- fecal calprotectin(initiation, at 6 months and every year)
- patient quality of life(initiation, 1 month, 6 months, 1 year and every 6 months)
- CFTR activity in nasal cells/bicarbonat(initiation of treatment and repeated if cell culture failure)
- Residual Volume(initiation, 7 days, 1 month, 6 months, 1 year and every 6 months)
- creatine phosphokinase in blood(initiation, 1 month, 3 Months, 6 months and every 6 months)
- tumor necrosis factor in sputum(initiation, 1 month, 3 Months, 6 months and every 6 months)
- liver function test in blood(initiation, 1 month, 3 Months, 6 months and every 6 months)
- liver function test/SGPT in blood(initiation, 1 month, 3 Months, 6 months and every 6 months)
- liver function test/bilirubin in blood(initiation, 1 month, 3 Months, 6 months and every 6 months)
- liver function test, gammaGT in blood(initiation, 1 month, 3 Months, 6 months and every 6 months)
- metabolomics of sweat(initiation, 7 days, 1 month, 6 months, 1 year and every 6 months)
- glycemic Holter(initiation, 1 year and every year)
- CFTR activity in nasal cells/chloride(initiation of treatment and repeated if cell culture failure)
- proteomic in urine(initiation, 7 days, 1 month, 6 months, 1 year and every year)
- Exhaled air composition(initiation, 7 days, 1 month, 6 months, 1 year and every year)
- Impedancemetry(initiation , 1 month, 3 Months, 6 months and every 6 months)
- proteomics of sweat(initiation, 7 days, 1 month, 6 months, 1 year and every 6 months)
- Lung MRI(initiation, at 1 year and every year)
- osteodensitometry(initiation, 1 year, and every year)
- CFTR activity in intestinal epithelium/chloride(initiation of treatment and repeated of cell culture failure)
- sweat evaporimetry(initiation and 1 month)
- Elasto MRI(initiation and 1 year)
- Lung Clearance Index(initiation, 6 months, 1 year and every 6 months)
研究者
Isabelle Sermet-Gaudelus
Professor
Hôpital Necker-Enfants Malades
研究点 (2)
Loading locations...
相似试验
招募中
不适用
Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 TrialsCystic FibrosisNCT04580368Children's Hospital Medical Center, Cincinnati50
已完成
不适用
Detection of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Activity in Rectal Tissues From Human SubjectsCystic FibrosisNCT01078740CF Therapeutics Development Network Coordinating Center57
已完成
不适用
Cystic Fibrosis and Frailtyh What's in Common?Frailty SyndromeCystic FibrosisNCT06020547Federico II University139
终止
不适用
Diagnosis and Treatment of Patients With Cystic FibrosisLiver Disease or AbnormalitiesCystic FibrosisPancreatic Insufficiency (Exocrine and Endocrine)DiabetesNCT00001223National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)79
已完成
不适用
HFNT During Exercise in CFCystic FibrosisNCT03965832The Leeds Teaching Hospitals NHS Trust23