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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
598
Placebo administered QID
Inhaled treprostinil (6 mcg/breath) administered QID
Treprostinil ultrasonic nebulizer which emits a dose of approximately 6 mcg per breath.
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.
Time frame: Baseline to Week 52
Time to Clinical Worsening
Clinical worsening was monitored from randomization until 1 of the following criteria were met: death (all causes), hospitalization due to a respiratory indication, or 10% relative decline in % predicted FVC.
Time frame: Baseline to Week 52
Time to First Acute Exacerbation of IPF
An exacerbation of IPF is defined as an acute, clinically significant, respiratory deterioration characterized by evidence of new widespread alveolar abnormality.
Time frame: Baseline to Week 52
Overall Survival at Week 52
Vital status will be assessed for all subjects at Week 52, including those who discontinue the study prematurely or who withdraw consent.
Time frame: Week 52
Change in % Predicted 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. Percent predicted FVC is calculated based on factors such as ethnicity, sex, age, height, and weight.
Time frame: Baseline to Week 52
Change in K-BILD Questionnaire Score from Baseline to Week 52
The K-BILD is a self-administered, 15-item questionnaire validated for patients with interstitial lung disease (ILD) consisting of 3 domains (breathlessness and activities, psychological, and chest symptoms).
Time frame: Baseline to Week 52
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
The University of Alabama at Birmingham
Birmingham, Alabama, United States
Banner University Medical Center-Phoenix
Phoenix, Arizona, United States
Arizona Pulmonary Specialists, Ltd.
Phoenix, Arizona, United States
St. Joseph's Hospital and Medical Center - Norton Thoracic Institute
Phoenix, Arizona, United States
University of Arizona
Tucson, Arizona, United States
UC San Diego Health
La Jolla, California, United States
David Geffen School of Medicine at UCLA
Los Angeles, California, United States
NewportNativeMD, Inc
Newport Beach, California, United States
University of California, Irvine
Orange, California, United States
Palmtree Clinical Research, Inc.
Palm Springs, California, United States
...and 88 more locations
Change in DLCO from Baseline to Week 52
The DLCO measurement measures how well oxygen moves from the lungs to the blood.
Time frame: Baseline to Week 52