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

Cystic Fibrosis Clinical Trial

— CLEAN-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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02343445
• Other study ID #: PS-G201
• Status: Completed
• Phase: Phase 2
• Start date: April 2015
• Est. completion date: February 2016

Study information

• Verified date: July 2021
• Source: Parion Sciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 142
• Est. completion date: February 2016
• Est. primary completion date: February 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• P-1037
P-1037 is a novel ENaC inhibitor
• Hypertonic Saline
4.2% saline solution
• Saline
0.17% saline solution

Locations

Country	Name	City	State
United States	Albany Medical College	Albany	New York
United States	Anderson Pharmaceutical Research	Anderson	South Carolina
United States	Austin Children's Chest Associates	Austin	Texas
United States	Johns Hopkins University	Baltimore	Maryland
United States	University of North Carolina	Chapel Hill	North Carolina
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Clinical Research of Charlotte	Charlotte	North Carolina
United States	University of Virginia Childrens	Charlottesville	Virginia
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	UT Southwestern Medical Center	Dallas	Texas
United States	Wayne State University/Harper University Hospital	Detroit	Michigan
United States	Univ of Florida Dept of Medicine	Gainesville	Florida
United States	Chicago Cystic Fibrosis Institute	Glenview	Illinois
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Dartmouth Hitchcock Medical Center	Lebanon	New Hampshire
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	University of Wisconsin UW Hospital	Madison	Wisconsin
United States	University of Miami Adult CF Center	Miami	Florida
United States	Vanderbilt University Medical Center	Nashville	Tennessee
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	Tulane University Health Sciences Center	New Orleans	Louisiana
United States	Santiago Reyes	Oklahoma City	Oklahoma
United States	Central Florida Pulmonary Group, PA	Orlando	Florida
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	Maine Medical Center	Portland	Maine
United States	Washington University	Saint Louis	Missouri
United States	Alamo Clinical Research Associates	San Antonio	Texas
United States	Pediatric Pulmonology/Cystic Fibrosis	Somerville	New Jersey
United States	New Lung Associates	Tampa	Florida
United States	Toledo Hospital	Toledo	Ohio
United States	Univ of Texas Health Science Center	Tyler	Texas
United States	UMass Memorial medical Center	Worcester	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Parion Sciences	Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety (number of adverse events of P-1037 in treatment groups)	number of adverse events of P-1037 in treatment groups	Day 15
Primary	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	Absolute change in FEV1	change in FEV1, from baseline (immediately before first dose) to same time of day on Day 15	Day 15
Secondary	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)	change in FVC from baseline (immediately before first dose) to same time of day on Day 15	Day 15
Secondary	Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)	CFQ-R on Day 15	Day 15
Secondary	Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15)	FEV1 on Day 15	Day 15
Secondary	Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%)	FEF25-75% from baseline (immediately before first dose) to same time of day on Day 15	Day 15