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

• Sponsor: Parion Sciences (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02343445
• Collaborator: Vertex Pharmaceuticals Incorporated (Industry)
• Enrollment: 142
• Locations: 33
• Arms: 4
• Duration (Months): 10.1
• Patients Per Site: 4.3
• Patients Per Site Per Month: 0.4

Study Details

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).

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: P-1037; Drug: Hypertonic Saline; Drug: Saline	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 142 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• 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 Start Date: Apr 1, 2015
• Actual Primary Completion Date: Feb 1, 2016
• Actual Study Completion Date: Feb 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
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

Outcome Measures

Primary Outcome Measures

1. Safety (number of adverse events of P-1037 in treatment groups) [Day 15]

   number of adverse events of P-1037 in treatment groups

2. Safety [change in forced expiratory volume in one second (FEV1) from predosing to 1 hour post dosing] [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

Secondary Outcome Measures

1. Absolute change in FEV1 [Day 15]

   change in FEV1, from baseline (immediately before first dose) to same time of day on Day 15

2. 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) [Day 15]

   change in FVC from baseline (immediately before first dose) to same time of day on Day 15

3. Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R) [Day 15]

   CFQ-R on Day 15

4. Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15) [Day 15]

   FEV1 on Day 15

5. Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%) [Day 15]

   FEF25-75% from baseline (immediately before first dose) to same time of day on Day 15

Eligibility Criteria

Criteria

• Ages Eligible for Study: 12 Years to 80 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

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

Sponsors and Collaborators

• Parion Sciences
• Vertex Pharmaceuticals Incorporated

Investigators

• Study Chair: Karl Donn, Parion Sciences

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Parion Sciences
• ClinicalTrials.gov Identifier: NCT02343445
• Other Study ID Numbers: PS-G201
• First Posted: Jan 22, 2015
• Last Update Posted: Jul 26, 2021
• Last Verified: Jul 1, 2021

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Additional relevant MeSH terms: Cystic Fibrosis; Fibrosis

Study Results

No Results Posted as of Jul 26, 2021