Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)
CLEAN-CF
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)
1 other identifier
interventional
142
1 country
33
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Apr 2015
Shorter than P25 for phase_2
33 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 13, 2015
CompletedFirst Posted
Study publicly available on registry
January 22, 2015
CompletedStudy Start
First participant enrolled
April 1, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
February 1, 2016
CompletedJuly 26, 2021
July 1, 2021
10 months
January 13, 2015
July 22, 2021
Conditions
Outcome Measures
Primary Outcomes (2)
Safety (number of adverse events of P-1037 in treatment groups)
number of adverse events of P-1037 in treatment groups
Day 15
Safety [change in forced expiratory volume in one second (FEV1) from predosing to 1 hour post dosing]
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
Day 1
Secondary Outcomes (5)
Absolute change in FEV1
Day 15
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
Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)
Day 15
Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15)
Day 15
Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%)
Day 15
Study Arms (4)
P-1037 in Hypertonic Saline (HS)
EXPERIMENTALP-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID
P-1037 in Saline
EXPERIMENTALP-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID
Saline
PLACEBO COMPARATORPlacebo (0.17% saline) BID
Hypertonic Saline
SHAM COMPARATORHypertonic saline (4.2% saline) BID
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Parion Scienceslead
- Vertex Pharmaceuticals Incorporatedcollaborator
Study Sites (33)
Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
Univ of Florida Dept of Medicine
Gainesville, Florida, 32610, United States
University of Miami Adult CF Center
Miami, Florida, 33136, United States
Central Florida Pulmonary Group, PA
Orlando, Florida, 32803, United States
New Lung Associates
Tampa, Florida, 33606, United States
Chicago Cystic Fibrosis Institute
Glenview, Illinois, 60025, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Tulane University Health Sciences Center
New Orleans, Louisiana, 70112, United States
Maine Medical Center
Portland, Maine, 04102, United States
Johns Hopkins University
Baltimore, Maryland, 21287, United States
UMass Memorial medical Center
Worcester, Massachusetts, 01655, United States
Wayne State University/Harper University Hospital
Detroit, Michigan, 48201, United States
Washington University
St Louis, Missouri, 63110, United States
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756, United States
Rutgets-Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901, United States
Pediatric Pulmonology/Cystic Fibrosis
Somerville, New Jersey, 08876, United States
Albany Medical College
Albany, New York, 12208, United States
Long Island Jewish Medical Center
New Hyde Park, New York, 11042, United States
University of North Carolina
Chapel Hill, North Carolina, 27517, United States
Clinical Research of Charlotte
Charlotte, North Carolina, 28277, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Toledo Hospital
Toledo, Ohio, 43606, United States
Santiago Reyes
Oklahoma City, Oklahoma, 73112, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Anderson Pharmaceutical Research
Anderson, South Carolina, 29621, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
Austin Children's Chest Associates
Austin, Texas, 78723, United States
UT Southwestern Medical Center
Dallas, Texas, 75390, United States
Alamo Clinical Research Associates
San Antonio, Texas, 78212, United States
Univ of Texas Health Science Center
Tyler, Texas, 75708, United States
University of Virginia Childrens
Charlottesville, Virginia, 22908, United States
University of Wisconsin UW Hospital
Madison, Wisconsin, 53792, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Karl Donn
Parion Sciences
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 13, 2015
First Posted
January 22, 2015
Study Start
April 1, 2015
Primary Completion
February 1, 2016
Study Completion
February 1, 2016
Last Updated
July 26, 2021
Record last verified: 2021-07