A phase 3 randomised, double-blind, controlled trial of inhaled 7% hypertonic saline versus 0.9% isotonic saline for 48 weeks in patients with cystic fibrosis at 3-6 years of age in parallel with the North American SHIP clinical trial

Cystic Fibrosis Foundation Therapeutics0 个研究点目标入组 120 人开始时间: 2015年11月5日最近更新: 2022年5月23日

适应症

试验速览

•1. Diagnosis of CF as evidenced by one or more clinical features consistent with the CF phenotype or positive CF newborn screen AND one or more of the following criteria:
•1.1. A documented sweat chloride = 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT)
•1.2. A documented genotype with two disease-causing mutations in the CFTR gene
•2. Informed consent by parent or legal guardian
•3. Age = 36 months and =72 months at screening visit
•4. Ability to comply with medication use, study visits and study procedures as judged by the site investigator
•5. Ability to cooperate with chest CT at the enrolment visit as determined by the lung function technician

•1. Chest CT within 8 months prior to the Screening visit
•2. Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate with onset within 3 weeks preceding screening or enrolment visit
•3. Acute wheezing at screening or enrolment visit
•4. Oxygen saturation < 95% (<90% in centres located above 4000 feet elevation) at screening or enrolment visit
•5. Other major organ dysfunction, excluding pancreatic dysfunction
•6. Physical findings that would compromise the safety of the participant or the quality of the study data as determined by site investigator
•7. Investigational drug use within 30 days prior to screening or enrolment visit
•8. Treatment with inhaled HS at any concentration within 30 days prior to screening or enrolment visit
•9. Start of any additional inhaled saline solution at any concentration, or other hydrating agent such as mannitol or mucolytic drug such as dornase alpha within 30 days prior or following the Screening or Enrolment visit
•10. Chronic lung disease not related to CF
•11. Inability to tolerate first dose of study treatment at the enrolment visit

发起方

Cystic Fibrosis Foundation Therapeutics