Clinical trial NCT02343445: Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)
Official 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cystic Fibrosis
Lead sponsor: Parion Sciences
Sponsor class: INDUSTRY
Collaborators: name: Vertex Pharmaceuticals Incorporated
First posted: 2015-01-22
Enrollment: 142
Sex: ALL
Minimum age (years): 12
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility 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

Primary outcomes:
measure: Safety (number of adverse events of P-1037 in treatment groups)
timeFrame: Day 15
measure: Safety [change in forced expiratory volume in one second (FEV1) from predosing to 1 hour post dosing]
timeFrame: Day 1
Secondary outcomes:
measure: Absolute change in FEV1
timeFrame: Day 15
measure: 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)
timeFrame: Day 15
measure: Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)
timeFrame: Day 15
measure: Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15)
timeFrame: Day 15
measure: Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%)
timeFrame: Day 15
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Gainesville
country: United States
facility: Univ of Florida Dept of Medicine
state: Florida
city: Miami
country: United States
facility: University of Miami Adult CF Center
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary Group, PA
state: Florida
city: Tampa
country: United States
facility: New Lung Associates
state: Florida
city: Glenview
country: United States
facility: Chicago Cystic Fibrosis Institute
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: New Orleans
country: United States
facility: Tulane University Health Sciences Center
state: Louisiana
city: Portland
country: United States
facility: Maine Medical Center
state: Maine
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Worcester
country: United States
facility: UMass Memorial medical Center
state: Massachusetts
city: Detroit
country: United States
facility: Wayne State University/Harper University Hospital
state: Michigan
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: New Brunswick
country: United States
facility: Rutgets-Robert Wood Johnson Medical School
state: New Jersey
city: Somerville
country: United States
facility: Pediatric Pulmonology/Cystic Fibrosis
state: New Jersey
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: New Hyde Park
country: United States
facility: Long Island Jewish Medical Center
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Charlotte
country: United States
facility: Clinical Research of Charlotte
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Toledo
country: United States
facility: Toledo Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: Santiago Reyes
state: Oklahoma
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Anderson
country: United States
facility: Anderson Pharmaceutical Research
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Austin
country: United States
facility: Austin Children's Chest Associates
state: Texas
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: San Antonio
country: United States
facility: Alamo Clinical Research Associates
state: Texas
city: Tyler
country: United States
facility: Univ of Texas Health Science Center
state: Texas
city: Charlottesville
country: United States
facility: University of Virginia Childrens
state: Virginia
city: Madison
country: United States
facility: University of Wisconsin UW Hospital
state: Wisconsin
Study officials:
affiliation: Parion Sciences
name: Karl Donn
role: STUDY_CHAIR
References: