Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)
- Conditions
- Cystic Fibrosis
- Interventions
- Registration Number
- NCT02343445
- Lead Sponsor
- Parion Sciences
- Brief Summary
The goal of the study is to evaluate the safety and tolerability of P-1037 and to determine whether the combination of P-1037 with hypertonic saline or P-1037 alone has a greater effect on lung function in patients with CF than placebo (0.17% saline).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 142
- 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.
-
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description P-1037 in Hypertonic Saline (HS) Hypertonic Saline P-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID P-1037 in Saline Saline P-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID P-1037 in Saline P-1037 P-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID Saline Saline Placebo (0.17% saline) BID P-1037 in Hypertonic Saline (HS) P-1037 P-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID Hypertonic Saline Hypertonic Saline Hypertonic saline (4.2% saline) BID
- Primary Outcome Measures
Name Time Method Safety (number of adverse events of P-1037 in treatment groups) Day 15 number of adverse events of P-1037 in treatment groups
Safety [change in forced expiratory volume in one second (FEV1) from predosing to 1 hour post dosing] Day 1 change in FEV1 from predosing to 1 hour post dosing with P-1037 in 4.2 % hypertonic saline and P-1037 in 0.17% saline
- Secondary Outcome Measures
Name Time Method 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) Day 15 change in FVC from baseline (immediately before first dose) to same time of day on Day 15
Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%) Day 15 FEF25-75% from baseline (immediately before first dose) to same time of day on Day 15
Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R) Day 15 CFQ-R on Day 15
Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15) Day 15 FEV1 on Day 15
Absolute change in FEV1 Day 15 change in FEV1, from baseline (immediately before first dose) to same time of day on Day 15
Trial Locations
- Locations (33)
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Washington University
🇺🇸Saint Louis, Missouri, United States
Albany Medical College
🇺🇸Albany, New York, United States
Toledo Hospital
🇺🇸Toledo, Ohio, United States
Univ of Texas Health Science Center
🇺🇸Tyler, Texas, United States
University of Virginia Childrens
🇺🇸Charlottesville, Virginia, United States
Clinical Research of Charlotte
🇺🇸Charlotte, North Carolina, United States
University of Wisconsin UW Hospital
🇺🇸Madison, Wisconsin, United States
Anderson Pharmaceutical Research
🇺🇸Anderson, South Carolina, United States
UT Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Chicago Cystic Fibrosis Institute
🇺🇸Glenview, Illinois, United States
Maine Medical Center
🇺🇸Portland, Maine, United States
UMass Memorial medical Center
🇺🇸Worcester, Massachusetts, United States
Wayne State University/Harper University Hospital
🇺🇸Detroit, Michigan, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
University of Miami Adult CF Center
🇺🇸Miami, Florida, United States
Santiago Reyes
🇺🇸Oklahoma City, Oklahoma, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Alamo Clinical Research Associates
🇺🇸San Antonio, Texas, United States
Pediatric Pulmonology/Cystic Fibrosis
🇺🇸Somerville, New Jersey, United States
Dartmouth Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
Rutgets-Robert Wood Johnson Medical School
🇺🇸New Brunswick, New Jersey, United States
Long Island Jewish Medical Center
🇺🇸New Hyde Park, New York, United States
Univ of Florida Dept of Medicine
🇺🇸Gainesville, Florida, United States
Central Florida Pulmonary Group, PA
🇺🇸Orlando, Florida, United States
New Lung Associates
🇺🇸Tampa, Florida, United States
Tulane University Health Sciences Center
🇺🇸New Orleans, Louisiana, United States
University of North Carolina
🇺🇸Chapel Hill, North Carolina, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Austin Children's Chest Associates
🇺🇸Austin, Texas, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States