NCT02343445.
| Study name | Clearing lungs with ENAC inhibition in cystic fibrosis (CLEAN‐CF) |
| Methods | Blinded (participant, care provider, investigator, outcomes assessor) RCT. Parallel design. 3 arms. Duration: 15 days. Multicentre (33 locations) in the USA. |
| Participants | Actual enrollment: 142 participants, both genders. Inclusion criteria: aged 12 years or older; diagnosis of CF as determined by the 1997 CF consensus criteria; non‐smoker; FEV1 at Screening Visit 1 between 40% and 90%; stable regimen of CF medications and chest physiotherapy for the 28 days prior to screening; willing to discontinue use of HS for the duration of the trial; clinically stable for at least 2 weeks; all females of child‐bearing potential must have a negative serum pregnancy test and if sexually active must agree to practice a highly effective form of contraception throughout the trial and for 28 days after the last dose of trial medication. Exclusion criteria: history of any organ transplantation or any significant disease or disorder; use of diuretics (including amiloride) or renin‐angiotensin antihypertensive drugs or trimethoprim in the 28 days prior to Screening; history of significant intolerance to inhaled HS, as determined by the investigator; known hypersensitivity to the trial drug or amiloride; any clinically significant laboratory abnormalities at Screening Visit 1 as judged by the investigator (or any of the following: potassium ≥ 5 mEq/L; abnormal renal function; abnormal liver function, defined as ≥ 3 x upper limit of normal; haemoglobin level < 10.0 g/dL); female who is pregnant or lactating; history of sputum or throat swab culture yielding Burkholderia species or Mycobacterium abscessus within 2 years of screening; previous participation in an investigational trial involving administration of any investigational compound or use of an investigational device with 28 days prior to Screening; currently being treated with any ivacaftor containing regimen. |
| Interventions |
Intervention 1: P‐1037 solution for inhalation, 85 μg (28.3 μg/mL) in HS (4.2%). Intervention 2: P‐1037 solution for inhalation, 85 μg (28.3 μg/mL) in 0.17% saline. Control: placebo (0.17% saline). All treatments were inhaled 2x daily. |
| Outcomes |
Primary outcomes Adverse events related to P‐1037 in treatment groups. FEV1 (change from pre‐dosing to 1 hour post‐dosing). Secondary outcomes FEV1 (absolute change from baseline to Day 15). FVC (absolute change from baseline to Day 15). CFQ‐R. FEF25%-75% (absolute change from baseline to Day 15). |
| Starting date | Trial start date: April 2015. Primary Completion Date: February 2016. Study Completion Date: February 2016. |
| Contact information | Vertex Pharmaceuticals Incorporated. |
| Notes | Sponsors and Collaborators: Vertex Pharmaceuticals Incorporated, Parion Sciences. |
CF: cystic fibrosis CFQ‐R: cystic fibrosis questionnaire ‐ revised DNase: dornase alfa ENAC: epithelial sodium channels FEF25%-75%: mid‐peak forced expiratory flow rate FEV1: forced expiratory volume in one second FVC: forced vital capacity HS: hypertonic saline IPD: individual patient data IV: intravenous QoL: quality of life RCT: randomised controlled trial