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Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects With Progressive Pulmonary Fibrosis (TETON-PPF)

Study Purpose

Study RIN-PF-305 is designed to evaluate the safety and efficacy of inhaled treprostinil in subjects with progressive pulmonary fibrosis (PPF) over a 52-week period.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Subject gives voluntary informed consent to participate in the study. 2. Subject is ≥18 years of age, inclusive, at the time of signing informed consent. 3. Subject has radiological evidence of pulmonary fibrosis of >10% extent on an HRCT scan in the previous 12 months (confirmed by central review). 4. Subject has a diagnosis of PPF (other than IPF) that fulfills at least 1 of the following criteria for progression within 24 months of screening despite standard treatment of ILD, as assessed by the Investigator: 1. Clinically significant decline in % predicted FVC based on ≥10% relative decline. 2. Marginal decline in % predicted FVC based on ≥5% to <10% relative decline combined with worsening of respiratory symptoms. 3. Marginal decline in % predicted FVC based on ≥5% to <10% relative decline combined with increasing extent of fibrotic changes on chest imaging. 4. Worsening of respiratory symptoms as well as increasing extent of fibrotic changes on chest imaging. 5. FVC ≥45% predicted at Screening (confirmed by central review). 6. Subjects must be on 1 of the following: 1. On nintedanib or pirfenidone for ≥90 days prior to Baseline and in the Investigator's opinion, are planning to continue treatment through the study. 2. Not on treatment with nintedanib or pirfenidone for ≥90 days prior to Baseline and in the Investigator's opinion, not planning to initiate either treatment during the study. Concomitant use of both nintedanib and pirfenidone is not permitted. 7. Subjects treated with immunosuppressive agents (eg, mycophenolate, methotrexate, azathioprine, oral corticosteroids, rituximab) need to be on treatment for at least 120 days prior to Baseline and, in the Investigator's clinical opinion, must be refractory to treatment. 8. Women of childbearing potential must be non-pregnant (as confirmed by a urine pregnancy test at Screening and Baseline) and non-lactating, and will agree to do 1 of the following: 1. Abstain from intercourse (when it is in line with their preferred and usual lifestyle) 2. Use 2 medically acceptable, highly effective forms of contraception for the duration of the study, and at least 30 days after discontinuing study drug. i. Medically acceptable, highly effective forms of contraception can include approved hormonal contraceptives (oral, injectable, and implantable) and barrier methods (such as a condom or diaphragm) when used with a spermicide. Women who are successfully sterilized (including hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) or postmenopausal (defined as amenorrhea for at least 12 consecutive months) are not considered to be of reproductive potential. 9. Males with a partner of childbearing potential must agree to use a condom for the duration of treatment and for at least 48 hours after discontinuing study drug. 10. 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 (forced expiratory volume in 1 second/FVC <0.70 at Screening) or greater extent of emphysema than fibrosis on HRCT (confirmed by central review). 3. Subject has a diagnosis of IPF. 4. 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. 5. 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 prior to any study-related efficacy assessments. 6. Subject is receiving >10 L/min of oxygen supplementation by any mode of delivery at rest at Baseline. 7. Exacerbation of ILD 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 ILD or a pulmonary or upper respiratory infection, subjects must have been discharged more than 90 days prior to Baseline to be eligible. 8. Subject has uncontrolled cardiac disease, defined as myocardial infarction within 6 months prior to Baseline or unstable angina within 30 days prior to Baseline. 9. 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. 10. Acute pulmonary embolism within 90 days prior to Baseline. 11. 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. 12. In the opinion of the Investigator, life expectancy <12 months due to ILD or a concomitant illness.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT05943535
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 3
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

United Therapeutics
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries Canada, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Progressive Pulmonary Fibrosis, Interstitial Lung Disease
Additional Details

Study RIN-PF-305 is a Phase 3, multinational, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of inhaled treprostinil in subjects with PPF 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 (forced vital capacity [FVC]), time to clinical worsening, time to first acute exacerbation of interstitial lung disease (ILD), 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.

Arms & Interventions

Arms

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.

Interventions

Drug: - Placebo

Placebo administered QID

Drug: - Inhaled Treprostinil

Inhaled treprostinil (6 mcg/breath) administered QID

Device: - Treprostinil Ultrasonic Nebulizer

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

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

UAB Lung Health Center, Birmingham, Alabama

Status

Recruiting

Address

UAB Lung Health Center

Birmingham, Alabama, 35233

Site Contact

Melessia Wells

mrasheed@uabmc.edu

205-975-9332

NewportNativeMD, Inc., Newport Beach, California

Status

Recruiting

Address

NewportNativeMD, Inc.

Newport Beach, California, 92663

Site Contact

Grace Harvey

grace@newportnativemd.com

949-791-8599 #107

Paradigm Clinical Research, San Diego, California

Status

Recruiting

Address

Paradigm Clinical Research

San Diego, California, 92108

Site Contact

Chery Bolovits

cbolovits@paradigm-research.com

858-274-4226

Stanford University Medical Center, Stanford, California

Status

Recruiting

Address

Stanford University Medical Center

Stanford, California, 94305

Site Contact

Jannet Estrada

janestr@stanford.edu

919-485-8350

Jacksonville, Florida

Status

Recruiting

Address

Ascension Medical Group St. Vincent's Lung Institute

Jacksonville, Florida, 32204

Site Contact

Jennifer McWilliams

jennifer.mcwilliams@ascension.org

904-308-5764

Chicago, Illinois

Status

Recruiting

Address

Rush University Medical Center Outpatient Pulmonary Clinic

Chicago, Illinois, 60612

Site Contact

Kimberly Saulsberry

Kimberly_P_Saulsberry@rush.edu

312-996-6275

UI Health Hospital, Chicago, Illinois

Status

Recruiting

Address

UI Health Hospital

Chicago, Illinois, 60612

Site Contact

Hannah Carlson

hrc@uic.edu

919-485-8350

University of Kansas Medical Center, Kansas City, Kansas

Status

Recruiting

Address

University of Kansas Medical Center

Kansas City, Kansas, 66160

Site Contact

Kimberly Lovell

klovell@kumc.edu

913-588-6067

Tulane Medical Center, New Orleans, Louisiana

Status

Recruiting

Address

Tulane Medical Center

New Orleans, Louisiana, 45227

Site Contact

Sandy Ditta

sditta@tulane.edu

504-988-4040

Silver Spring, Maryland

Status

Recruiting

Address

Adventist Healthcare White Oak Medical Center

Silver Spring, Maryland, 20904

Site Contact

Jennifer Hernandez

jhernandez1@adventisthealthcare.com

240-637-5024

Tufts Medical Center, Boston, Massachusetts

Status

Recruiting

Address

Tufts Medical Center

Boston, Massachusetts, 02111

Infinity Medical Center, North Dartmouth, Massachusetts

Status

Recruiting

Address

Infinity Medical Center

North Dartmouth, Massachusetts, 02747

Beaumont Hospital, Royal Oak, Royal Oak, Michigan

Status

Recruiting

Address

Beaumont Hospital, Royal Oak

Royal Oak, Michigan, 48073

Site Contact

Beverly Mauer

beverly.maurer@corewellhealth.org

248-551-2581

Minneapolis, Minnesota

Status

Recruiting

Address

University of Minnesota Health Clinical Research Unit (CRU)

Minneapolis, Minnesota, 55455

Site Contact

Amanda DeGrote

carl1032@umn.edu

919-485-8350

Mayo Clinic, Rochester, Minnesota

Status

Recruiting

Address

Mayo Clinic

Rochester, Minnesota, 55905

Site Contact

Teng Moua

moua.teng@mayo.edu

507-284-5398

The Lung Research Center, LLC, Chesterfield, Missouri

Status

Recruiting

Address

The Lung Research Center, LLC

Chesterfield, Missouri, 63017

Site Contact

Anna Shipp

Anna.Shipp@stlukes-stl.com

314-682-3653

Montefiore Medical Center, Bronx, New York

Status

Recruiting

Address

Montefiore Medical Center

Bronx, New York, 10467

Site Contact

Jane Wilson

jwilson4@montefiore.org

718-920-7916

Northwell Health, New Hyde Park, New York

Status

Recruiting

Address

Northwell Health

New Hyde Park, New York, 11042

Site Contact

Sara Khan

skhan109@northwell.edu

516-465-5437

PulmonIx LLC, Greensboro, North Carolina

Status

Recruiting

Address

PulmonIx LLC

Greensboro, North Carolina, 27403

Site Contact

Lauren Hinshaw

lauren.hinshaw@pulmonix.com

336-522-8870

East Carolina University, Greenville, North Carolina

Status

Recruiting

Address

East Carolina University

Greenville, North Carolina, 27834

Site Contact

Shravya Dharambhat

dharambhats22@ecu.edu

252-744-1116

Columbus, Ohio

Status

Recruiting

Address

The Ohio State University Wexner Medical CEnter

Columbus, Ohio, 43201

Site Contact

Benjamin Hood

benjamin.hood2@osumc.edu

614-293-3351

Philadelphia, Pennsylvania

Status

Recruiting

Address

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104

Charleston, South Carolina

Status

Recruiting

Address

Medical University of South Carolina-Nexus

Charleston, South Carolina, 29425

Site Contact

Angela Francisco

millare@musc.edu

843-792-3710

Prisma Health Pulmonology-Richland, Columbia, South Carolina

Status

Recruiting

Address

Prisma Health Pulmonology-Richland

Columbia, South Carolina, 29203

Site Contact

Keera McKenna

keera.mckenna2@prismahealth.org

803-434-8262

Franklin, Tennessee

Status

Recruiting

Address

Clinical Trials Center of Middle Tennessee, LLC

Franklin, Tennessee, 37067

Site Contact

Lindsey Robbs

lrobbs@ctcmidtn.com

615-205-8350

StatCare Pulmonary Consultants, PLLC, Knoxville, Tennessee

Status

Recruiting

Address

StatCare Pulmonary Consultants, PLLC

Knoxville, Tennessee, 37919

Site Contact

Tabitha McCauley

tabitha.mccauley@biomed-research.com

865-934-2672

A & A Research Consultants, LLC, McAllen, Texas

Status

Recruiting

Address

A & A Research Consultants, LLC

McAllen, Texas, 78503

Site Contact

Jaedan Chapa

jaedanchapa@hotmail.com

972-802-1512

Metroplex Pulmonary and Sleep Center, McKinney, Texas

Status

Recruiting

Address

Metroplex Pulmonary and Sleep Center

McKinney, Texas, 75069

Site Contact

Ambreen Ahmed

aahmed@mpsleepcenter.com

972-838-1892

University of Utah Health, Salt Lake City, Utah

Status

Recruiting

Address

University of Utah Health

Salt Lake City, Utah, 84108

Site Contact

Heather Hammerschmidt

heather.hammerschmidt@hsc.utah.edu

801-585-5194

Christopher King, MD, Falls Church, Virginia

Status

Recruiting

Address

Christopher King, MD

Falls Church, Virginia, 22042

Site Contact

Priscila Dauphin

priscila.dauphin@inova.org

703-776-7128

Pulmonary Associates of Richmond, Inc., Richmond, Virginia

Status

Recruiting

Address

Pulmonary Associates of Richmond, Inc.

Richmond, Virginia, 23230

Site Contact

Betsy Daniel

bdaniel@paraccess.com

804-288-5947

International Sites

Dynamic Drug Advancement Limited, Ajax, Ontario, Canada

Status

Recruiting

Address

Dynamic Drug Advancement Limited

Ajax, Ontario, L1S 2J5

CIC Mauricie inc., Québec, Quebec, Canada

Status

Recruiting

Address

CIC Mauricie inc.

Québec, Quebec, G8T 7A1

Site Contact

Mireille Devost

mdevost@cic-mauricie.ca

(819) 373-1128