A Phase IIb, Multicentre, Randomised, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Subjects with Cystic Fibrosis Aged 18 Years or Older (CHOICES) - HIT-CF-001

niversity Medical Center Utrecht0 个研究点目标入组 52 人开始时间: 2023年12月22日最近更新: 2024年8月21日

适应症

试验速览

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•Male or female subjects who have completed the HIT-CF Organoid Study and are =18 years of age on the date of informed consent, Confirmed diagnosis of CF as follows: Sweat chloride value of =60 mmol/L based on quantitative pilocarpine iontophoresis (at screening) OR 2-CF causing mutations AND o chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities, Clinically stable CF disease in the opinion of the investigator with no significant changes in health status within 28 days prior to Day 1, FEV1 =40% to =90% predicted according to the Global Lung Function Initiative (GLI), Body mass index (BMI) =16 kg/m2 and =30 kg/m2, Non-smoker and non-tobacco user for a minimum of 30 days prior to screening, and subject agrees not to smoke or use tobacco for the duration of the study, Selected by an unblinded coordinating team based on organoid response or random selection

•Key exclusion criteria include: • Any CF patient meeting either of the following two criteria is defined as having a ‘common’ CFTR mutation(s): o At least one of the following mutations: F508del, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, A455E, 3849+10kbC>T; OR o A combination of any two of the following mutations: any nonsense mutation, 1717-1G>A, 621+1G>T, 3120+1G>A, 1898+1G->A, CFTRdele2,3, and 2183AA->G, Subject is currently taking or has taken a CFTR modulator within 28 days prior to Day 1

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