Validation of respiratory epithelial functional assessment to predict clinical efficacy of Orkambi®. Pathway to personalized therapy in Cystic Fibrosis PREDICT-CF - PREDICT-CF

ASSISTANC-PUBLIQUE-HOPITAUX DE PARIS (AP-HP)0 个研究点目标入组 104 人开始时间: 2018年11月21日最近更新: 2019年2月28日

适应症

试验速览

暂无简介。

•- Homozygous F508del patient aged 12 years or older
•- Patient with an indication for Orkambi® treatment according to the marketing authorization application
•- Patient never received Orkambi® in the past
•- Signed Informed consent form by the patient (if aged = 18 years), or by parents / legal guardian and patient’s agreement (if aged < 18 years)
•- Patient affiliated to the health insurance system
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 52
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 50
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 2

•- Homozygous F508del patients who do not meet the treatment indications according to the marketing authorization application
•- Patients refusing Orkambi®
•- CF patients not homozygous for the p.Phe508del mutation
•- Active smoker
•- Severe nasal mucosa disrepair
•- Contraindications to xylocaine anesthesia,
•- Participation with another interventional study with drug

发起方

ASSISTANC-PUBLIQUE-HOPITAUX DE PARIS (AP-HP)