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

NCT ID: NCT04708782

Last Updated: 2026-01-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

576 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-06-01

Study Completion Date

2026-01-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Idiopathic Pulmonary Fibrosis Interstitial Lung Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Placebo

Matching placebo inhaled using an ultrasonic nebulizer QID

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo administered QID

Treprostinil Ultrasonic Nebulizer

Intervention Type DEVICE

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

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.

Group Type EXPERIMENTAL

Inhaled Treprostinil

Intervention Type DRUG

Inhaled treprostinil (6 mcg/breath) administered QID

Treprostinil Ultrasonic Nebulizer

Intervention Type DEVICE

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

Interventions

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Placebo

Placebo administered QID

Intervention Type DRUG

Inhaled Treprostinil

Inhaled treprostinil (6 mcg/breath) administered QID

Intervention Type DRUG

Treprostinil Ultrasonic Nebulizer

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

Intervention Type DEVICE

Other Intervention Names

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Tyvaso

Eligibility Criteria

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Inclusion Criteria

1. 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

1. 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.
Minimum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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United Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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The University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Banner University Medical Center-Phoenix

Phoenix, Arizona, United States

Site Status

Arizona Pulmonary Specialists, Ltd.

Phoenix, Arizona, United States

Site Status

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

Phoenix, Arizona, United States

Site Status

University of Arizona

Tucson, Arizona, United States

Site Status

UC San Diego Health

La Jolla, California, United States

Site Status

David Geffen School of Medicine at UCLA

Los Angeles, California, United States

Site Status

NewportNativeMD, Inc

Newport Beach, California, United States

Site Status

University of California, Irvine

Orange, California, United States

Site Status

Palmtree Clinical Research, Inc.

Palm Springs, California, United States

Site Status

UC Davis Health Medical Center

Sacramento, California, United States

Site Status

University of California - San Francisco

San Francisco, California, United States

Site Status

Stanford University Medical Center

Stanford, California, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

St. Francis Sleep Allergy & Lung Institute

Clearwater, Florida, United States

Site Status

University of Florida Health at Shands

Gainesville, Florida, United States

Site Status

Ascension St. Vincent's

Jacksonville, Florida, United States

Site Status

University of Florida

Jacksonville, Florida, United States

Site Status

Mayo Clinic Florida

Jacksonville, Florida, United States

Site Status

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

Kissimmee, Florida, United States

Site Status

Central Florida Pulmonary Group, PA

Orlando, Florida, United States

Site Status

Coastal Pulmonary & Critical Care PLC

St. Petersburg, Florida, United States

Site Status

University of South Florida Health

Tampa, Florida, United States

Site Status

Cleveland Clinic Florida

Weston, Florida, United States

Site Status

Piedmont Healthcare Atlanta

Atlanta, Georgia, United States

Site Status

Emory University Hospital

Atlanta, Georgia, United States

Site Status

The Queen's Medical Center

Honolulu, Hawaii, United States

Site Status

Northwestern Memorial Hospital

Chicago, Illinois, United States

Site Status

Rush University Medical Center Outpatient Pulmonary Clinic

Chicago, Illinois, United States

Site Status

Loyola University Medical Center

Maywood, Illinois, United States

Site Status

Community Health Network

Indianapolis, Indiana, United States

Site Status

The University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Norton Pulmonary Specialists

Louisville, Kentucky, United States

Site Status

University of Louisville

Louisville, Kentucky, United States

Site Status

LSU Health Sciences Center New Orleans

New Orleans, Louisiana, United States

Site Status

Tulane University Medical Center

New Orleans, Louisiana, United States

Site Status

LSU Health Science Center Shreveport

Shreveport, Louisiana, United States

Site Status

Johns Hopkins Asthma & Allergy Center

Baltimore, Maryland, United States

Site Status

Adventist Healthcare White Oak Medical CEnter

Silver Spring, Maryland, United States

Site Status

Tufts Medical Center

Boston, Massachusetts, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan Int Med Pulmonary and critical care

Ann Arbor, Michigan, United States

Site Status

Henry Ford Hospital

Detroit, Michigan, United States

Site Status

Corewell Health William Beaumont Hospital

Royal Oak, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

University of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status

The Lung Research Center

Chesterfield, Missouri, United States

Site Status

Saint Luke's Hospital of Kansas City

Kansas City, Missouri, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Creighton University Clinical Research Office

Omaha, Nebraska, United States

Site Status

University of New Mexico Health Sciences Center

Albuquerque, New Mexico, United States

Site Status

St Joseph's Physician's Pulmonary Health

Liverpool, New York, United States

Site Status

Northwell Health

New Hyde Park, New York, United States

Site Status

University of Rochester Medical center

Rochester, New York, United States

Site Status

Stony Brook Advanced Specialty Care

Stony Brook, New York, United States

Site Status

Montefiore Medical Center

The Bronx, New York, United States

Site Status

PulmonIx, LLC

Greensboro, North Carolina, United States

Site Status

East Carolina University and Leo Jenkins Cancer Center

Greenville, North Carolina, United States

Site Status

Southeastern Research Center

Winston-Salem, North Carolina, United States

Site Status

University of Cincinnati

Cincinnati, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

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

Columbus, Ohio, United States

Site Status

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Site Status

The Oregon Clinic, PC

Portland, Oregon, United States

Site Status

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

Hershey, Pennsylvania, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Thoams Jefferson,Hospital University

Philadelphia, Pennsylvania, United States

Site Status

Temple Lung Center

Philadelphia, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Prisma Health Midlands

Columbia, South Carolina, United States

Site Status

Clinical Research of Rock Hill

Rock Hill, South Carolina, United States

Site Status

Clinical Trials Center of Middle Tennessee

Franklin, Tennessee, United States

Site Status

Statecare Pulmonary Consultants

Knoxville, Tennessee, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Baylor University Medical Center

Dallas, Texas, United States

Site Status

Univrsity of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Premier Pulmonary Critical Care and Sleep Medicine

Denison, Texas, United States

Site Status

Baylor Clinic-Baylor College of Medicine

Houston, Texas, United States

Site Status

The University of Texas Health Science Center at Houston

Houston, Texas, United States

Site Status

A & A Research Consultants, LLC

McAllen, Texas, United States

Site Status

Metroplex Pulmonary and Sleep Center PA

McKinney, Texas, United States

Site Status

Renovatio Clinical Consultants, LLC

The Woodlands, Texas, United States

Site Status

Intermountain Medical Center

Murray, Utah, United States

Site Status

University of Utah Health

Salt Lake City, Utah, United States

Site Status

University of Virginia Health System

Charlottesville, Virginia, United States

Site Status

Inova Fairfax Hospital

Falls Church, Virginia, United States

Site Status

Pulmonary Associates of Richmond, Inc.

Richmond, Virginia, United States

Site Status

University of Wisconsin School of Medicine and Public health

Madison, Wisconsin, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

St.Paul's Hospital

Vancouver, British Columbia, Canada

Site Status

Dynamic Drug Advancement Limited

Ajax, Ontario, Canada

Site Status

St. Joseph's Healthcare Hamilton

Hamilton, Ontario, Canada

Site Status

Toronto General Hospital

Toronto, Ontario, Canada

Site Status

Centre d'investigation Clinique Mauricie

Trois-Rivières, Quebec, Canada

Site Status

Institut Universitaire de Cardiologie et de Pneumologie de Quebec-Universite

Québec, , Canada

Site Status

Centro Respiratorio Integral LTDA. (CENRESIN)

Quillota, Región de Valparaíso, Chile

Site Status

Fundacion Medica San Cristobal

Santiago, Santiago Metropolitan, Chile

Site Status

Countries

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United States Canada Chile

Other Identifiers

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RIN-PF-301

Identifier Type: -

Identifier Source: org_study_id

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