Study of Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis

NCT04708782 | Completed Phase 3 DMC FDA Drug FDA Device

• 1 other identifier: RIN-PF-301
• Study Type: interventional
• Target: 598
• Locations: 3 countries
• Sites: 98

Brief Summary

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

Keywords

Treprostinil; IPF; ILD

Outcome Measures

Primary Outcomes (1)

• Change in Absolute FVC from Baseline to Week 52

  The FVC measurement indicates the amount of air a person can forcefully and quickly exhale after taking a deep breath.

  Baseline to Week 52

Secondary Outcomes (6)

• Time to Clinical Worsening

  Baseline to Week 52

• Time to First Acute Exacerbation of IPF

  Baseline to Week 52

• Overall Survival at Week 52

  Week 52

• Change in % Predicted FVC from Baseline to Week 52

  Baseline to Week 52

• Change in K-BILD Questionnaire Score from Baseline to Week 52

  Baseline to Week 52

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Matching placebo inhaled using an ultrasonic nebulizer QID

Drug: Placebo; Device: Treprostinil Ultrasonic Nebulizer

Inhaled Treprostinil

EXPERIMENTAL

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.

Drug: Inhaled Treprostinil; Device: Treprostinil Ultrasonic Nebulizer

Interventions

Placebo DRUG

Placebo administered QID

Placebo

Inhaled Treprostinil DRUG

Inhaled treprostinil (6 mcg/breath) administered QID

Also known as: Tyvaso

Inhaled Treprostinil

Treprostinil Ultrasonic Nebulizer DEVICE

Treprostinil ultrasonic nebulizer which emits a dose of approximately 6 mcg per breath.

Inhaled Treprostinil; Placebo

Eligibility Criteria

• Age: 40 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject gives voluntary informed consent to participate in the study.
• Subject is ≥40 years of age, inclusive, at the time of signing informed consent.
• 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.
• FVC ≥45% predicted at Screening.
• 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.
• 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.
• 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.
• 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.

You may not qualify if:

• Subject is pregnant or lactating.
• Subject has primary obstructive airway physiology: FEV1/FVC \<0.70 at Screening.
• 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.
• 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.
• 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.
• The subject is receiving \>10 L/min of oxygen supplementation by any mode of delivery at rest at Baseline.
• 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.
• Uncontrolled cardiac disease, defined as myocardial infarction within 6 months prior to Baseline or unstable angina within 30 days prior to Baseline.
• 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.
• 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.
• Life expectancy \<6 months due to IPF or a concomitant illness.
• Acute pulmonary embolism within 90 days prior to Baseline.

Sponsors & Collaborators

• United Therapeutics: lead

Study Sites (98)

The University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

Banner University Medical Center-Phoenix

Phoenix, Arizona, 85006, United States

Location

Arizona Pulmonary Specialists, Ltd.

Phoenix, Arizona, 85012, United States

Location

St. Joseph's Hospital and Medical Center - Norton Thoracic Institute

Phoenix, Arizona, 85013, United States

Location

University of Arizona

Tucson, Arizona, 85724, United States

Location

UC San Diego Health

La Jolla, California, 92037, United States

Location

David Geffen School of Medicine at UCLA

Los Angeles, California, 90095, United States

Location

NewportNativeMD, Inc

Newport Beach, California, 92663, United States

Location

University of California, Irvine

Orange, California, 92868, United States

Location

Palmtree Clinical Research, Inc.

Palm Springs, California, 92262, United States

Location

UC Davis Health Medical Center

Sacramento, California, 95817, United States

Location

University of California - San Francisco

San Francisco, California, 94143, United States

Location

Stanford University Medical Center

Stanford, California, 94305, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

St. Francis Sleep Allergy & Lung Institute

Clearwater, Florida, 33765, United States

Location

University of Florida Health at Shands

Gainesville, Florida, 32610, United States

Location

Ascension St. Vincent's

Jacksonville, Florida, 32204, United States

Location

University of Florida

Jacksonville, Florida, 32209, United States

Location

Mayo Clinic Florida

Jacksonville, Florida, 32224, United States

Location

Pulmonary Disease Specialists, PA d/b/a PDS Research

Kissimmee, Florida, 34741, United States

Location

Central Florida Pulmonary Group, PA

Orlando, Florida, 32803, United States

Location

Coastal Pulmonary & Critical Care PLC

St. Petersburg, Florida, 33704, United States

Location

University of South Florida Health

Tampa, Florida, 33606, United States

Location

Cleveland Clinic Florida

Weston, Florida, 33331, United States

Location

Piedmont Healthcare Atlanta

Atlanta, Georgia, 30309, United States

Location

Emory University Hospital

Atlanta, Georgia, 30322, United States

Location

The Queen's Medical Center

Honolulu, Hawaii, 96813, United States

Location

Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

Location

Rush University Medical Center Outpatient Pulmonary Clinic

Chicago, Illinois, 60612, United States

Location

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

Community Health Network

Indianapolis, Indiana, 46250, United States

Location

The University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Norton Pulmonary Specialists

Louisville, Kentucky, 40202, United States

Location

University of Louisville

Louisville, Kentucky, 40202, United States

Location

LSU Health Sciences Center New Orleans

New Orleans, Louisiana, 70112, United States

Location

Tulane University Medical Center

New Orleans, Louisiana, 70112, United States

Location

LSU Health Science Center Shreveport

Shreveport, Louisiana, 71103, United States

Location

Johns Hopkins Asthma & Allergy Center

Baltimore, Maryland, 21224, United States

Location

Adventist Healthcare White Oak Medical CEnter

Silver Spring, Maryland, 20904, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Michigan Int Med Pulmonary and critical care

Ann Arbor, Michigan, 48109, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Corewell Health William Beaumont Hospital

Royal Oak, Michigan, 48073, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Mayo Clinic

Rochester, Minnesota, 55902, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

The Lung Research Center

Chesterfield, Missouri, 63017, United States

Location

Saint Luke's Hospital of Kansas City

Kansas City, Missouri, 64111, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Creighton University Clinical Research Office

Omaha, Nebraska, 68124, United States

Location

University of New Mexico Health Sciences Center

Albuquerque, New Mexico, 87106, United States

Location

St Joseph's Physician's Pulmonary Health

Liverpool, New York, 13088, United States

Location

Northwell Health

New Hyde Park, New York, 11040, United States

Location

University of Rochester Medical center

Rochester, New York, 14642, United States

Location

Stony Brook Advanced Specialty Care

Stony Brook, New York, 11725, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

PulmonIx, LLC

Greensboro, North Carolina, 27403, United States

Location

East Carolina University and Leo Jenkins Cancer Center

Greenville, North Carolina, 27834, United States

Location

Southeastern Research Center

Winston-Salem, North Carolina, 27103, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45267, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

The Ohio State University Wexner Medical Center - Martha Morehouse Medical Pavilion

Columbus, Ohio, 43221, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

The Oregon Clinic, PC

Portland, Oregon, 97220, United States

Location

Penn State Milton S. Hershey Medical Center/Penn State College of Medicine

Hershey, Pennsylvania, 17033, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Thoams Jefferson,Hospital University

Philadelphia, Pennsylvania, 19107, United States

Location

Temple Lung Center

Philadelphia, Pennsylvania, 19140, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Prisma Health Midlands

Columbia, South Carolina, 29203, United States

Location

Clinical Research of Rock Hill

Rock Hill, South Carolina, 29732, United States

Location

Statecare Pulmonary Consultants

Knoxville, Tennessee, 37919, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37204, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

Univrsity of Texas Southwestern Medical Center

Dallas, Texas, 75390-8550, United States

Location

Premier Pulmonary Critical Care and Sleep Medicine

Denison, Texas, 75020, United States

Location

Baylor Clinic-Baylor College of Medicine

Houston, Texas, 77030, United States

Location

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

A & A Research Consultants, LLC

McAllen, Texas, 78503, United States

Location

Metroplex Pulmonary and Sleep Center PA

McKinney, Texas, 75069, United States

Location

Renovatio Clinical Consultants, LLC

The Woodlands, Texas, 77380, United States

Location

Intermountain Medical Center

Murray, Utah, 84107, United States

Location

University of Utah Health

Salt Lake City, Utah, 84108, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22903, United States

Location

Inova Fairfax Hospital

Falls Church, Virginia, 22042, United States

Location

Pulmonary Associates of Richmond, Inc.

Richmond, Virginia, 23230, United States

Location

University of Wisconsin School of Medicine and Public health

Madison, Wisconsin, 53792, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

St.Paul's Hospital

Vancouver, British Columbia, V6Z1Y6, Canada

Location

Dynamic Drug Advancement Limited

Ajax, Ontario, L1S 2J5, Canada

Location

St. Joseph's Healthcare Hamilton

Hamilton, Ontario, L8N 4A6, Canada

Location

Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

Centre d'investigation Clinique Mauricie

Trois-Rivières, Quebec, G8T 7A1, Canada

Location

Institut Universitaire de Cardiologie et de Pneumologie de Quebec-Universite

Québec, G1V 4G5, Canada

Location

Fundacion Medica San Cristobal

Santiago, Santiago Metropolitan, 7630226, Chile

Location

Centro Respiratorio Integral LTDA. (CENRESIN)

Quillota, Valparaiso, 2260000, Chile

Location

MeSH Terms

Conditions

Idiopathic Pulmonary Fibrosis; Lung Diseases, Interstitial

Interventions

treprostinil

Condition Hierarchy (Ancestors)

Pulmonary Fibrosis; Lung Diseases; Respiratory Tract Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 12, 2021
• First Posted: January 14, 2021
• Study Start: June 1, 2021
• Primary Completion: February 2, 2026
• Study Completion: February 2, 2026
• Last Updated: May 5, 2026

Record last verified: 2026-05

Locations

CL (2); US (90); CA (6)