Cystic FibrosisPhase 2CLEAN-CFNCT02343445
Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)
A Phase 2 study testing P-1037, Hypertonic Saline and Saline in people with Cystic Fibrosis, run by Parion Sciences at 33 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 12 to 80 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- P-1037, Hypertonic Saline and Saline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 142 participants
- Where
- 33 sitesUnited States
- Study dates
- Apr 2015to Feb 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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)
Show all 13Show fewer
- 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
Trial locations and contacts
33 sites in 1 country.
33 sites in 1 country
United States33 sites
Albany Medical College
Albany, New York, 12208
Anderson Pharmaceutical Research
Anderson, South Carolina, 29621
Austin Children's Chest Associates
Austin, Texas, 78723
Johns Hopkins University
Baltimore, Maryland, 21287
University of North Carolina
Chapel Hill, North Carolina, 27517
Medical University of South Carolina
Charleston, South Carolina, 29425
Clinical Research of Charlotte
Charlotte, North Carolina, 28277
University of Virginia Childrens
Charlottesville, Virginia, 22908
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
UT Southwestern Medical Center
Dallas, Texas, 75390
Wayne State University/Harper University Hospital
Detroit, Michigan, 48201
Univ of Florida Dept of Medicine
Gainesville, Florida, 32610
Chicago Cystic Fibrosis Institute
Glenview, Illinois, 60025
University of Kansas Medical Center
Kansas City, Kansas, 66160
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
Children's Hospital of Los Angeles
Los Angeles, California, 90027
University of Wisconsin UW Hospital
Madison, Wisconsin, 53792
University of Miami Adult CF Center
Miami, Florida, 33136
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Rutgets-Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
Long Island Jewish Medical Center
New Hyde Park, New York, 11042
Tulane University Health Sciences Center
New Orleans, Louisiana, 70112
Santiago Reyes
Oklahoma City, Oklahoma, 73112
Central Florida Pulmonary Group, PA
Orlando, Florida, 32803
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Maine Medical Center
Portland, Maine, 04102
Alamo Clinical Research Associates
San Antonio, Texas, 78212
Pediatric Pulmonology/Cystic Fibrosis
Somerville, New Jersey, 08876
Washington University
St Louis, Missouri, 63110
New Lung Associates
Tampa, Florida, 33606
Toledo Hospital
Toledo, Ohio, 43606
Univ of Texas Health Science Center
Tyler, Texas, 75708
UMass Memorial medical Center
Worcester, Massachusetts, 01655
About this study
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)
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).
- Conditions
- Cystic Fibrosis
- Being tested
- P-1037, Hypertonic Saline, Saline
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
P-1037Drug
P-1037 is a novel ENaC inhibitor
Hypertonic SalineDrug
4.2% saline solution
SalineDrug
0.17% saline solution
| Study group | Role | What they receive |
|---|---|---|
| P-1037 in Hypertonic Saline (HS) | Experimental | P-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID Receives: Drug: P-1037; Drug: Hypertonic Saline |
| P-1037 in Saline | Experimental | P-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID Receives: Drug: P-1037; Drug: Saline |
| Saline | Placebo comparator | Placebo (0.17% saline) BID Receives: Drug: Saline |
| Hypertonic Saline | Sham comparator | Hypertonic saline (4.2% saline) BID Receives: Drug: Hypertonic Saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety (number of adverse events of P-1037 in treatment groups)
number of adverse events of P-1037 in treatment groups
Measured: Day 15
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
Measured: Day 1
Secondary outcomes (5)
Absolute change in FEV1
change in FEV1, from baseline (immediately before first dose) to same time of day on Day 15
Measured: 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
Measured: Day 15
Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)
CFQ-R on Day 15
Measured: 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
Measured: 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
Measured: Day 15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2015 (estimated)
- Study startApr 2015
- Primary completionFeb 2016
- Study completionFeb 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 142 participants (actual)
Sponsor and investigators
- Lead sponsor
- Parion Sciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Vertex Pharmaceuticals Incorporated
- Responsible party
- Sponsor
- Investigators
- Karl Donn, Study chair, Parion Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02343445
- Sponsor's protocol ID
- PS-G201
- Acronym
- CLEAN-CF
- Status last verified
- Jul 2021
- First submitted
- 13 Jan 2015
- Last updated
- 26 Jul 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02343445
- Acronym / sponsor ID
- CLEAN-CF / PS-G201
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 142 participants (actual)
- Lead sponsor
- Parion SciencesIndustry (pharma, biotech, devices)
- Collaborators
- Vertex Pharmaceuticals Incorporated
- Study start
- Apr 2015
- Primary completion
- Feb 2016 (actual)
- Study completion
- Feb 2016 (actual)
- First posted
- 22 Jan 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| P-1037 in Hypertonic Saline (HS) | Experimental | Drug: P-1037, Drug: Hypertonic SalineP-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID |
| P-1037 in Saline | Experimental | Drug: P-1037, Drug: SalineP-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID |
| Saline | Placebo comparator | Drug: SalinePlacebo (0.17% saline) BID |
| Hypertonic Saline | Sham comparator | Drug: Hypertonic SalineHypertonic saline (4.2% saline) BID |
Who may qualify
- Ages
- 12 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (13 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| P-1037P-1037 is a novel ENaC inhibitor | Drug | P-1037 in Hypertonic Saline (HS), P-1037 in Saline |
| Hypertonic Saline4.2% saline solution | Drug | Hypertonic Saline, P-1037 in Hypertonic Saline (HS) |
| Saline0.17% saline solution | Drug | P-1037 in Saline, Saline |
Primary outcome
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
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
5 secondary outcomes
Absolute change in FEV1
Time frame: 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
Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)
Time frame: 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
Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%)
Time frame: Day 15
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2021.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Karl Donn study chair
Parion Sciences
33 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing P-1037, Hypertonic Saline and Saline in people with Cystic Fibrosis, run by Parion Sciences at 33 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 33 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety (number of adverse events of P-1037 in treatment groups) (time frame: Day 15). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jul 2021.