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

NCT ID: NCT02343445

Last Updated: 2021-07-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

142 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-04-30

Study Completion Date

2016-02-29

Brief Summary

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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

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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P-1037 in Hypertonic Saline (HS)

P-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID

Group Type EXPERIMENTAL

P-1037

Intervention Type DRUG

P-1037 is a novel ENaC inhibitor

Hypertonic Saline

Intervention Type DRUG

4.2% saline solution

P-1037 in Saline

P-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID

Group Type EXPERIMENTAL

P-1037

Intervention Type DRUG

P-1037 is a novel ENaC inhibitor

Saline

Intervention Type DRUG

0.17% saline solution

Saline

Placebo (0.17% saline) BID

Group Type PLACEBO_COMPARATOR

Saline

Intervention Type DRUG

0.17% saline solution

Hypertonic Saline

Hypertonic saline (4.2% saline) BID

Group Type SHAM_COMPARATOR

Hypertonic Saline

Intervention Type DRUG

4.2% saline solution

Interventions

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P-1037

P-1037 is a novel ENaC inhibitor

Intervention Type DRUG

Hypertonic Saline

4.2% saline solution

Intervention Type DRUG

Saline

0.17% saline solution

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role collaborator

Parion Sciences

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Karl Donn

Role: STUDY_CHAIR

Parion Sciences

Locations

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Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status

Univ of Florida Dept of Medicine

Gainesville, Florida, United States

Site Status

University of Miami Adult CF Center

Miami, Florida, United States

Site Status

Central Florida Pulmonary Group, PA

Orlando, Florida, United States

Site Status

New Lung Associates

Tampa, Florida, United States

Site Status

Chicago Cystic Fibrosis Institute

Glenview, Illinois, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Tulane University Health Sciences Center

New Orleans, Louisiana, United States

Site Status

Maine Medical Center

Portland, Maine, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

UMass Memorial medical Center

Worcester, Massachusetts, United States

Site Status

Wayne State University/Harper University Hospital

Detroit, Michigan, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States

Site Status

Rutgets-Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

Site Status

Pediatric Pulmonology/Cystic Fibrosis

Somerville, New Jersey, United States

Site Status

Albany Medical College

Albany, New York, United States

Site Status

Long Island Jewish Medical Center

New Hyde Park, New York, United States

Site Status

University of North Carolina

Chapel Hill, North Carolina, United States

Site Status

Clinical Research of Charlotte

Charlotte, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Toledo Hospital

Toledo, Ohio, United States

Site Status

Santiago Reyes

Oklahoma City, Oklahoma, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Anderson Pharmaceutical Research

Anderson, South Carolina, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Austin Children's Chest Associates

Austin, Texas, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

Alamo Clinical Research Associates

San Antonio, Texas, United States

Site Status

Univ of Texas Health Science Center

Tyler, Texas, United States

Site Status

University of Virginia Childrens

Charlottesville, Virginia, United States

Site Status

University of Wisconsin UW Hospital

Madison, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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PS-G201

Identifier Type: -

Identifier Source: org_study_id

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