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

July 22, 2021 updated by: Parion Sciences

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)

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
142

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital of Los Angeles
    • Florida
      • Gainesville, Florida, United States, 32610
        • Univ of Florida Dept of Medicine
      • Miami, Florida, United States, 33136
        • University of Miami Adult CF Center
      • Orlando, Florida, United States, 32803
        • Central Florida Pulmonary Group, PA
      • Tampa, Florida, United States, 33606
        • New Lung Associates
    • Illinois
      • Glenview, Illinois, United States, 60025
        • Chicago Cystic Fibrosis Institute
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Tulane University Health Sciences Center
    • Maine
      • Portland, Maine, United States, 04102
        • Maine Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins University
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
        • UMass Memorial Medical Center
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Wayne State University/Harper University Hospital
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Dartmouth Hitchcock Medical Center
    • New Jersey
      • New Brunswick, New Jersey, United States, 08901
        • Rutgets-Robert Wood Johnson Medical School
      • Somerville, New Jersey, United States, 08876
        • Pediatric Pulmonology/Cystic Fibrosis
    • New York
      • Albany, New York, United States, 12208
        • Albany Medical College
      • New Hyde Park, New York, United States, 11042
        • Long Island Jewish Medical Center
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27517
        • University of North Carolina
      • Charlotte, North Carolina, United States, 28277
        • Clinical Research of Charlotte
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
      • Toledo, Ohio, United States, 43606
        • Toledo Hospital
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73112
        • Santiago Reyes
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • South Carolina
      • Anderson, South Carolina, United States, 29621
        • Anderson Pharmaceutical Research
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Texas
      • Austin, Texas, United States, 78723
        • Austin Children's Chest Associates
      • Dallas, Texas, United States, 75390
        • UT Southwestern Medical Center
      • San Antonio, Texas, United States, 78212
        • Alamo Clinical Research Associates
      • Tyler, Texas, United States, 75708
        • Univ of Texas Health Science Center
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • University of Virginia Childrens
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • University of Wisconsin UW Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years to 80 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: 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
Drug: P-1037
P-1037 is a novel ENaC inhibitor
Drug: Hypertonic Saline
4.2% saline solution
Experimental: P-1037 in Saline
P-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID
Drug: P-1037
P-1037 is a novel ENaC inhibitor
Drug: Saline
0.17% saline solution
Placebo Comparator: Saline
Placebo (0.17% saline) BID
Drug: Saline
0.17% saline solution
Sham Comparator: Hypertonic Saline
Hypertonic saline (4.2% saline) BID
Drug: Hypertonic Saline
4.2% saline solution

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety (number of adverse events of P-1037 in treatment groups)
Time Frame: Day 15
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]
Time Frame: 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
Day 1

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Absolute change in FEV1
Time Frame: Day 15
change in FEV1, from baseline (immediately before first dose) to same time of day on Day 15
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)
Time Frame: Day 15
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)
Time Frame: Day 15
CFQ-R on Day 15
Day 15
Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15)
Time Frame: Day 15
FEV1 on Day 15
Day 15
Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%)
Time Frame: Day 15
FEF25-75% from baseline (immediately before first dose) to same time of day on Day 15
Day 15

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Parion Sciences

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Vertex Pharmaceuticals Incorporated

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Chair: Karl Donn, Parion Sciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 1, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 1, 2016

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 13, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 21, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 22, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 26, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 22, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • PS-G201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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