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Clearing Lungs With ENAC Inhibition in Cystic Fibrosis (CLEAN-CF)

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ClinicalTrials.gov Identifier: NCT02343445
Recruitment Status : Completed
First Posted : January 22, 2015
Last Update Posted : August 9, 2016
Sponsor:
Collaborator:
Parion Sciences
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

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

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Study Type : Interventional  (Clinical Trial)
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 : April 2015
Actual Primary Completion Date : February 2016
Actual Study Completion Date : February 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: P-1037 in Hypertonic Saline
P-1037 Solution for Inhalation, 85 μg 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




Primary Outcome Measures :
  1. 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

  2. Safety (change in 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


Secondary Outcome Measures :
  1. 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

  2. Effect of P-1037 on other 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

  3. Effect of P-1037 on CFQ-R [ Time Frame: Day 15 ]
    CFQ-R on Day 15

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

  5. Effect of P-1037 on FEF25-75% [ Time Frame: Day 15 ]
    FEF25-75% from baseline (immediately before first dose) to same time of day on Day 15



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Ages Eligible for Study:   12 Years to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02343445


  Show 33 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Parion Sciences

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02343445     History of Changes
Other Study ID Numbers: PS-G201
First Posted: January 22, 2015    Key Record Dates
Last Update Posted: August 9, 2016
Last Verified: March 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
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Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pharmaceutical Solutions