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)
Key facts
- Where
- Florence, Genoa, Milan, Rome és Verona, OlaszországSee the 5 sites
- Phase
- 2. fázis
Where in Olaszország
- Azienda Ospedaliera Universitaria Meyer IRCCSFlorence, OlaszországNo longer taking part
- Giannina Gaslini Institute For Scientific Hospitalization And CareGenoa, OlaszországNo longer taking part
- Fondazione IRCCS Cà Granda Ospedale Maggiore PoliclinicoMilan, OlaszországNo longer taking part
- Ospedale Pediatrico Bambino GesùRome, OlaszországNo longer taking part
- Azienda Ospedaliera Universitaria Integrata VeronaVerona, OlaszországNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- University Medical Center Utrecht
- Trial code
- EU 2022-500410-26-01
- Record updated
- 2025. június 2.
- Original description
To evaluate the efficacy of DIR/POS/NES after 8 weeks compared to placebo in (a) CF patients with rare CFTR mutations and a high organoid response to DIR/POS/NES and (b) CF patients with rare CFTR mutations not pre-selected on organoid response.Read full original descriptionShow less
To evaluate the efficacy of DIR/POS/NES after 8 weeks compared to placebo in (a) CF patients with rare CFTR mutations and a high organoid response to DIR/POS/NES and (b) CF patients with rare CFTR mutations not pre-selected on organoid response.
Inclusion: 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.
Exclusion: Subject is currently taking or has taken a CFTR modulator within 28 days prior to Day 1.