Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)

• Register: ClinicalTrials.gov
• Last refreshed on: 2 August 2021
• Main ID: NCT02343445
• Date of registration: 13/01/2015
• Prospective Registration: Yes
• Primary sponsor: Parion Sciences
• Public title: Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF) CLEAN-CF
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of P-1037 Solution for Inhalation in Patients With Cystic Fibrosis (CF)
• Date of first enrolment: April 2015
• Target sample size: 142
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02343445
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Karl Donn
• Affiliation: Parion Sciences

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female, 12 years of age or older.

- Diagnosis of cystic fibrosis as determined by the 1997 CF Consensus criteria (NIH
Consensus Statement, 1997)

- 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. Must be willing to discontinue use of hypertonic saline for the duration of
the study.

- 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 study and for 28 days after the last dose of study 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 hypertonic saline, as determined by the
Investigator

- Known hypersensitivity to the study drug or amiloride

- Any clinically significant laboratory abnormalities at Screening Visit 1 as judged by
the investigator, or any of the following:

- Potassium = 5 milliequivalent per Liter (mEq/L)

- Abnormal renal function

- Abnormal liver function, defined as = 3 x upper limit of normal (ULN)

- Hemoglobin 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

- Has previously participated 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



Age minimum: 12 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: P-1037

Drug: Saline

Drug: Hypertonic Saline

Primary Outcome(s)

Safety (number of adverse events of P-1037 in treatment groups) [Time Frame: Day 15]

Safety [change in forced expiratory volume in one second (FEV1) from predosing to 1 hour post dosing] [Time Frame: Day 1]

Secondary Outcome(s)

Effect of P-1037 on other forced vital capacity (FVC) measures (change in FVC from baseline (immediately before first dose) to same time of day on Day 15) [Time Frame: Day 15]

Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15) [Time Frame: Day 15]

Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%) [Time Frame: Day 15]

Absolute change in FEV1 [Time Frame: Day 15]

Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R) [Time Frame: Day 15]

Secondary ID(s)

PS-G201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Vertex Pharmaceuticals Incorporated