Study of Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis
Purpose
Study RIN-PF-301 is designed to evaluate the superiority of inhaled treprostinil against placebo for the change in absolute forced vital capacity (FVC) from baseline to Week 52.
Conditions
- Idiopathic Pulmonary Fibrosis
- Interstitial Lung Disease
Eligibility
- Eligible Ages
- Over 40 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Subject gives voluntary informed consent to participate in the study. 2. Subject is ≥40 years of age, inclusive, at the time of signing informed consent. 3. The subject has a diagnosis of IPF based on the 2018 ATS/ERS/JRS/ALAT Clinical Practice Guideline (Raghu 2018) and confirmed by central review of high-resolution computed tomography (HRCT) (performed within the previous 12 months), and if available, surgical lung biopsy. 4. FVC ≥45% predicted at Screening. 5. Subjects on pirfenidone or nintedanib must be on a stable and optimized dose for ≥30 days prior to Baseline. Concomitant use of both pirfenidone and nintedanib is not permitted. 6. Women of childbearing potential must be non-pregnant (as confirmed by a urine pregnancy test at Screening and Baseline) and non-lactating, and will abstain from intercourse (when it is in line with their preferred and usual lifestyle) or use 2 medically acceptable, highly effective forms of contraception for the duration of the study, and at least 30 days after discontinuing study drug. 7. Males with a partner of childbearing potential must use a condom for the duration of treatment and for at least 48 hours after discontinuing study drug. 8. In the opinion of the Investigator, the subject is able to communicate effectively with study personnel, and is considered reliable, willing, and likely to be cooperative with protocol requirements, including attending all study visits.
Exclusion Criteria
- Subject is pregnant or lactating. 2. Subject has primary obstructive airway physiology: FEV1/FVC <0.70 at Screening. 3. The subject has shown intolerance or significant lack of efficacy to a prostacyclin or prostacyclin analogue that resulted in discontinuation or inability to effectively titrate that therapy. 4. The subject has received any PAH-approved therapy, including prostacyclin therapy (epoprostenol, treprostinil, iloprost, or beraprost; except for acute vasoreactivity testing), IP receptor agonists (selexipag), endothelin receptor antagonists, phosphodiesterase type 5 inhibitors (PDE5-Is), or soluble guanylate cyclase stimulators within 60 days prior to Baseline. As needed use of a PDE5-I for erectile dysfunction is permitted, provided no doses are taken within 48 hours of any study-related efficacy assessments. 5. Use of any of the following medications: azathioprine (AZA), cyclosporine, mycophenolate mofetil, tacrolimus, oral corticosteroids (OCS) >20 mg/day or the combination of OCS+AZA+N-acetylcysteine within 30 days prior to Baseline; cyclophosphamide within 60 days prior to Baseline; or rituximab within 6 months prior to Baseline. 6. The subject is receiving >10 L/min of oxygen supplementation by any mode of delivery at rest at Baseline. 7. Exacerbation of IPF or active pulmonary or upper respiratory infection within 30 days prior to Baseline. Subjects must have completed any antibiotic or steroid regimens for treatment of the infection or acute exacerbation more than 30 days prior to Baseline to be eligible. If hospitalized for an acute exacerbation of IPF or a pulmonary or upper respiratory infection, subjects must have been discharged more than 90 days prior to Baseline to be eligible. 8. Uncontrolled cardiac disease, defined as myocardial infarction within 6 months prior to Baseline or unstable angina within 30 days prior to Baseline. 9. In the opinion of the Investigator, the subject has any condition that would interfere with the interpretation of study assessments or would impair study participation or cooperation. 10. Use of any other investigational drug/device or participation in any investigational study in which the subject received a medical intervention (ie, procedure, device, medication/supplement) within 30 days prior to Screening. Subjects participating in non-interventional, observational, or registry studies are eligible. 11. Life expectancy <6 months due to IPF or a concomitant illness. 12. Acute pulmonary embolism within 90 days prior to Baseline.
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Placebo Comparator Placebo |
Matching placebo inhaled using an ultrasonic nebulizer QID |
|
|
Experimental Inhaled Treprostinil |
Treprostinil for inhalation solution (0.6 mg/mL) delivered via an ultrasonic nebulizer which emits a dose of approximately 6 mcg per breath. Inhaled QID and titrated up to a target of 12 breaths QID or until the subject reaches their maximum clinically tolerated dose. |
|
Recruiting Locations
The University of Alabama at Birmingham
Birmingham, Alabama 35233
Birmingham, Alabama 35233
Northwell Health
New Hyde Park, New York 11040
New Hyde Park, New York 11040
University of Cincinnati
Cincinnati, Ohio 45267
Cincinnati, Ohio 45267
Southeastern Research Center
Winston-Salem, North Carolina 27103
Winston-Salem, North Carolina 27103
East Carolina University and Leo Jenkins Cancer Center
Greenville, North Carolina 27834
Greenville, North Carolina 27834
PulmonIx, LLC
Greensboro, North Carolina 27403
Greensboro, North Carolina 27403
Stony Brook Advanced Specialty Care
Stony Brook, New York 11725
Stony Brook, New York 11725
University of Rochester Medical center
Rochester, New York 14642
Rochester, New York 14642
St Joseph's Physician's Pulmonary Health
Liverpool, New York 13088
Liverpool, New York 13088
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma 73104
Oklahoma City, Oklahoma 73104
Montefiore Medical Center
Bronx, New York 10467
Bronx, New York 10467
University of New Mexico Health Sciences Center
Albuquerque, New Mexico 87106
Albuquerque, New Mexico 87106
Creighton University Clinical Research Office
Omaha, Nebraska 68124
Omaha, Nebraska 68124
Washington University School of Medicine
Saint Louis, Missouri 63110
Saint Louis, Missouri 63110
Saint Luke's Hospital of Kansas City
Kansas City, Missouri 64111
Kansas City, Missouri 64111
The Lung Research Center
Chesterfield, Missouri 63017
Chesterfield, Missouri 63017
University of Mississippi Medical Center
Jackson, Mississippi 39216
Jackson, Mississippi 39216
The Ohio State University Wexner Medical Center - Martha Morehouse Medical Pavilion
Columbus, Ohio 43221
Columbus, Ohio 43221
Corewell Health William Beaumont Hospital
Royal Oak, Michigan 48073
Royal Oak, Michigan 48073
Premier Pulmonary Critical Care and Sleep Medicine
Denison, Texas 75020
Denison, Texas 75020
Pulmonary Associates of Richmond, Inc.
Richmond, Virginia 23230
Richmond, Virginia 23230
Inova Fairfax Hospital
Falls Church, Virginia 22042
Falls Church, Virginia 22042
University of Virginia Health System
Charlottesville, Virginia 22903
Charlottesville, Virginia 22903
University of Utah Health
Salt Lake City, Utah 84108
Salt Lake City, Utah 84108
Intermountain Medical Center
Murray, Utah 84107
Murray, Utah 84107
Metroplex Pulmonary and Sleep Center PA
McKinney, Texas 75069
McKinney, Texas 75069
The University of Texas Health Science Center at Houston
Houston, Texas 77030
Houston, Texas 77030
Baylor Clinic-Baylor College of Medicine
Houston, Texas 77030
Houston, Texas 77030
Baylor University Medical Center
Dallas, Texas 75246
Dallas, Texas 75246
Penn State Milton S. Hershey Medical Center/Penn State College of Medicine
Hershey, Pennsylvania 17033
Hershey, Pennsylvania 17033
Vanderbilt University Medical Center
Nashville, Tennessee 37204
Nashville, Tennessee 37204
Statecare Pulmonary Consultants
Knoxville, Tennessee 37919
Knoxville, Tennessee 37919
Clinical Trials Center of Middle Tennessee
Franklin, Tennessee 37067
Franklin, Tennessee 37067
Clinical Research of Rock Hill
Rock Hill, South Carolina 29732
Rock Hill, South Carolina 29732
Medical University of South Carolina
Charleston, South Carolina 29425
Charleston, South Carolina 29425
Temple Lung Center
Philadelphia, Pennsylvania 19140
Philadelphia, Pennsylvania 19140
Thoams Jefferson,Hospital University
Philadelphia, Pennsylvania 19107
Philadelphia, Pennsylvania 19107
University of Pennsylvania
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
University of Minnesota
Minneapolis, Minnesota 55455
Minneapolis, Minnesota 55455
St. Joseph's Hospital and Medical Center - Norton Thoracic Institute
Phoenix, Arizona 85013
Phoenix, Arizona 85013
St. Francis Sleep Allergy & Lung Institute
Clearwater, Florida 33765
Clearwater, Florida 33765
Advanced Pulmonary Research Institute
Palm Beach, Florida 33470
Palm Beach, Florida 33470
Central Florida Pulmonary Group, PA
Orlando, Florida 32803
Orlando, Florida 32803
Lakeland Regional Health
Lakeland, Florida 33805
Lakeland, Florida 33805
Mayo Clinic Florida
Jacksonville, Florida 32224
Jacksonville, Florida 32224
University of Florida
Jacksonville, Florida 32209
Jacksonville, Florida 32209
Ascension St. Vincent's
Jacksonville, Florida 32204
Jacksonville, Florida 32204
University of Florida Health at Shands
Gainesville, Florida 32610
Gainesville, Florida 32610
Georgetown University Hospital
Washington, District of Columbia 20007
Washington, District of Columbia 20007
University of South Florida Health
Tampa, Florida 33606
Tampa, Florida 33606
National Jewish Health
Denver, Colorado 80206
Denver, Colorado 80206
Stanford University Medical Center
Stanford, California 94305
Stanford, California 94305
University of California - San Francisco
San Francisco, California 94143
San Francisco, California 94143
Palmtree Clinical Research, Inc.
Palm Springs, California 92262
Palm Springs, California 92262
University of California, Irvine
Orange, California 92868
Orange, California 92868
NewportNativeMD, Inc
Newport Beach, California 92663
Newport Beach, California 92663
David Geffen School of Medicine at UCLA
Los Angeles, California 90095
Los Angeles, California 90095
University of Arizona
Tucson, Arizona 85724
Tucson, Arizona 85724
Coastal Pulmonary & Critical Care PLC
Saint Petersburg, Florida 33704
Saint Petersburg, Florida 33704
University of Michigan Int Med Pulmonary and critical care
Ann Arbor, Michigan 48109
Ann Arbor, Michigan 48109
University of Louisville
Louisville, Kentucky 40202
Louisville, Kentucky 40202
Brigham and Women's Hospital
Boston, Massachusetts 02115
Boston, Massachusetts 02115
Massachusetts General Hospital
Boston, Massachusetts 02114
Boston, Massachusetts 02114
Tufts Medical Center
Boston, Massachusetts 02111
Boston, Massachusetts 02111
Adventist Healthcare White Oak Medical CEnter
Silver Spring, Maryland 20904
Silver Spring, Maryland 20904
Johns Hopkins Asthma & Allergy Center
Baltimore, Maryland 21224
Baltimore, Maryland 21224
LSU Health Science Center Shreveport
Shreveport, Louisiana 71103
Shreveport, Louisiana 71103
Tulane University Medical Center
New Orleans, Louisiana 70112
New Orleans, Louisiana 70112
LSU Health Sciences Center New Orleans
New Orleans, Louisiana 70112
New Orleans, Louisiana 70112
Norton Pulmonary Specialists
Louisville, Kentucky 40202
Louisville, Kentucky 40202
Piedmont Healthcare Atlanta
Atlanta, Georgia 30309
Atlanta, Georgia 30309
The University of Kansas Medical Center
Kansas City, Kansas 66160
Kansas City, Kansas 66160
Community Health Network
Indianapolis, Indiana 46250
Indianapolis, Indiana 46250
Loyola University Medical Center
Maywood, Illinois 60153
Maywood, Illinois 60153
University of Illinois at Chicago
Chicago, Illinois 60612
Chicago, Illinois 60612
Rush University Medical Center Outpatient Pulmonary Clinic
Chicago, Illinois 60612
Chicago, Illinois 60612
Northwestern Memorial Hospital
Chicago, Illinois 60611
Chicago, Illinois 60611
The Queen's Medical Center
Honolulu, Hawaii 96813
Honolulu, Hawaii 96813
University of Wisconsin School of Medicine and Public health
Madison, Wisconsin 53792
Madison, Wisconsin 53792
More Details
- NCT ID
- NCT04708782
- Status
- Recruiting
- Sponsor
- United Therapeutics
Study Contact
United Therapeutics Global Medical Information919-485-8350
clinicaltrials@unither.com
Detailed Description
Study RIN-PF-301 is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the superiority of inhaled treprostinil against placebo for the change in absolute FVC in subjects with IPF over a 52-week period. Subjects will be randomly allocated 1:1 to receive inhaled treprostinil or placebo. All subjects will initiate inhaled treprostinil or placebo at a dose of 3 breaths administered 4 times daily (QID) and will titrate to a target dosing regimen of 12 breaths QID. Study drug doses may be titrated up as tolerated, until the target dose or maximum clinically tolerated dose is achieved. Once eligible, 6 Treatment Period visits to the clinic will be required at Weeks 4, 8, 16, 28, 40, and 52. Efficacy assessments include spirometry (FVC), time to clinical worsening, time to first acute exacerbation of IPF, overall survival, King's Brief Interstitial Lung Disease (K-BILD) questionnaire, plasma N-terminal pro-brain natriuretic peptide (NT-proBNP) concentration, supplemental oxygen use, and lung diffusion capacity (DLCO). Safety assessments include the development of adverse events (AEs)/serious adverse events (SAEs), vital signs, clinical laboratory parameters, and electrocardiogram (ECG) parameters. Subjects who complete the Week 52 Visit may be offered the opportunity to enter an open-label extension (OLE) study after completing the final study visit.