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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
P-1037
Hypertonic Saline
Saline
Sponsored by
Parion Sciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

12 Years - 80 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's Hospital of Los Angeles
  • Univ of Florida Dept of Medicine
  • University of Miami Adult CF Center
  • Central Florida Pulmonary Group, PA
  • New Lung Associates
  • Chicago Cystic Fibrosis Institute
  • University of Kansas Medical Center
  • Tulane University Health Sciences Center
  • Maine Medical Center
  • Johns Hopkins University
  • UMass Memorial medical Center
  • Wayne State University/Harper University Hospital
  • Washington University
  • Dartmouth Hitchcock Medical Center
  • Rutgets-Robert Wood Johnson Medical School
  • Pediatric Pulmonology/Cystic Fibrosis
  • Albany Medical College
  • Long Island Jewish Medical Center
  • University of North Carolina
  • Clinical Research of Charlotte
  • Cincinnati Children's Hospital Medical Center
  • Toledo Hospital
  • Santiago Reyes
  • Children's Hospital of Pittsburgh of UPMC
  • Anderson Pharmaceutical Research
  • Medical University of South Carolina
  • Vanderbilt University Medical Center
  • Austin Children's Chest Associates
  • UT Southwestern Medical Center
  • Alamo Clinical Research Associates
  • Univ of Texas Health Science Center
  • University of Virginia Childrens
  • University of Wisconsin UW Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Placebo Comparator

Sham Comparator

Arm Label

P-1037 in Hypertonic Saline (HS)

P-1037 in Saline

Saline

Hypertonic Saline

Arm Description

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

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

Placebo (0.17% saline) BID

Hypertonic saline (4.2% saline) BID

Outcomes

Primary Outcome Measures

Safety (number of adverse events of P-1037 in treatment groups)
number of adverse events of P-1037 in treatment groups
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

Secondary Outcome Measures

Absolute change in FEV1
change in FEV1, from baseline (immediately before first dose) to same time of day on 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)
change in FVC from baseline (immediately before first dose) to same time of day on Day 15
Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)
CFQ-R on Day 15
Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15)
FEV1 on Day 15
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

Full Information

First Posted
January 13, 2015
Last Updated
July 22, 2021
Sponsor
Parion Sciences
Collaborators
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT02343445
Brief Title
Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)
Acronym
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)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2021
Overall Recruitment Status
Completed
Study Start Date
April 2015 (undefined)
Primary Completion Date
February 2016 (Actual)
Study Completion Date
February 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Parion Sciences
Collaborators
Vertex Pharmaceuticals Incorporated

4. Oversight

Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
142 (Actual)

8. Arms, Groups, and Interventions

Arm Title
P-1037 in Hypertonic Saline (HS)
Arm Type
Experimental
Arm Description
P-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID
Arm Title
P-1037 in Saline
Arm Type
Experimental
Arm Description
P-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID
Arm Title
Saline
Arm Type
Placebo Comparator
Arm Description
Placebo (0.17% saline) BID
Arm Title
Hypertonic Saline
Arm Type
Sham Comparator
Arm Description
Hypertonic saline (4.2% saline) BID
Intervention Type
Drug
Intervention Name(s)
P-1037
Intervention Description
P-1037 is a novel ENaC inhibitor
Intervention Type
Drug
Intervention Name(s)
Hypertonic Saline
Intervention Description
4.2% saline solution
Intervention Type
Drug
Intervention Name(s)
Saline
Intervention Description
0.17% saline solution
Primary Outcome Measure Information:
Title
Safety (number of adverse events of P-1037 in treatment groups)
Description
number of adverse events of P-1037 in treatment groups
Time Frame
Day 15
Title
Safety [change in forced expiratory volume in one second (FEV1) from predosing to 1 hour post dosing]
Description
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
Time Frame
Day 1
Secondary Outcome Measure Information:
Title
Absolute change in FEV1
Description
change in FEV1, from baseline (immediately before first dose) to same time of day on Day 15
Time Frame
Day 15
Title
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)
Description
change in FVC from baseline (immediately before first dose) to same time of day on Day 15
Time Frame
Day 15
Title
Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)
Description
CFQ-R on Day 15
Time Frame
Day 15
Title
Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15)
Description
FEV1 on Day 15
Time Frame
Day 15
Title
Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%)
Description
FEF25-75% from baseline (immediately before first dose) to same time of day on Day 15
Time Frame
Day 15

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Karl Donn
Organizational Affiliation
Parion Sciences
Official's Role
Study Chair
Facility Information:
Facility Name
Children's Hospital of Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Univ of Florida Dept of Medicine
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
University of Miami Adult CF Center
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Central Florida Pulmonary Group, PA
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
New Lung Associates
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Facility Name
Chicago Cystic Fibrosis Institute
City
Glenview
State/Province
Illinois
ZIP/Postal Code
60025
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Tulane University Health Sciences Center
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Facility Name
Maine Medical Center
City
Portland
State/Province
Maine
ZIP/Postal Code
04102
Country
United States
Facility Name
Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
UMass Memorial medical Center
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States
Facility Name
Wayne State University/Harper University Hospital
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Washington University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Dartmouth Hitchcock Medical Center
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
Facility Name
Rutgets-Robert Wood Johnson Medical School
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Facility Name
Pediatric Pulmonology/Cystic Fibrosis
City
Somerville
State/Province
New Jersey
ZIP/Postal Code
08876
Country
United States
Facility Name
Albany Medical College
City
Albany
State/Province
New York
ZIP/Postal Code
12208
Country
United States
Facility Name
Long Island Jewish Medical Center
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11042
Country
United States
Facility Name
University of North Carolina
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27517
Country
United States
Facility Name
Clinical Research of Charlotte
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28277
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Toledo Hospital
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43606
Country
United States
Facility Name
Santiago Reyes
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73112
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Anderson Pharmaceutical Research
City
Anderson
State/Province
South Carolina
ZIP/Postal Code
29621
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Austin Children's Chest Associates
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Facility Name
UT Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Alamo Clinical Research Associates
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78212
Country
United States
Facility Name
Univ of Texas Health Science Center
City
Tyler
State/Province
Texas
ZIP/Postal Code
75708
Country
United States
Facility Name
University of Virginia Childrens
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
University of Wisconsin UW Hospital
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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

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