An Open Label Extension Study to Evaluate Inhaled Treprostinil in Adult PH With ILD Including CPFE

Phase 3TerminatedNCT02633293

United Therapeutics243 enrolled

Overview

This is a multicenter, open-label trial to evaluate the safety and efficacy of inhaled treprostinil in subjects with pre-capillary pulmonary hypertension (PH) associated with interstitial lung disease (ILD) including combined pulmonary fibrosis and emphysema (CPFE). The study will include about 266 patients who completed all required assessments in the RIN-PH-201 study at approximately 100 clinical trial centers. The study will continue Your participation in this study is voluntary and will last until you discontinue from the study or the study ends. The study will continue until each subject reaches the Week 108 visit or until inhaled treprostinil become commercially available for patients with PH associated with ILD including CPFE (whichever is sooner).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

243

Conditions

Pulmonary Hypertension Interstitial Lung Disease Combined Pulmonary Fibrosis and Emphysema

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Subject voluntarily gives informed consent to participate in the study. 2. The subject participated in study RIN-PH-201, remained on study drug, was compliant with RIN-PH-201 study procedures or was enrolled in study RIN-PH-201 at the time that the study was discontinued by the sponsor. 3. Females of reproductive potential must be non-pregnant (as confirmed by a urine pregnancy test at Baseline) and non-lactating, and will: * Either abstain from intercourse (when it is in line with their preferred and usual lifestyle), or * Use two medically acceptable, highly-effective forms of contraception for the duration of study, and at least 30 days after discontinuing study drug. 4. Males must use a condom for the duration of treatment and for at least 48 hours after discontinuing study drug. Exclusion Criteria: 1. The subject is pregnant or lactating. 2. The subject was prematurely discontinued from study RIN-PH-201. 3. The subject developed a concurrent illness or condition during the conduct of RIN-PH-201 which, in the opinion of the Investigator, would represent a risk to overall health if they enrolled in this study.

Locations (92)

University of Alabama at Birmingham

Birmingham, Alabama, United States

IMC-Diagnostic & Medical Clinic

Mobile, Alabama, United States

Arizona Pulmonary Specialists, Ltd.

Phoenix, Arizona, United States

University of Arizona

Tucson, Arizona, United States

Cedars-Sinai Medical Center

Beverly Hills, California, United States

Outcomes

Primary Outcomes

Change in Peak 6-Minute Walk Distance (6MWD) From Baseline

The intent of the 6MWD test is to evaluate exercise capacity associated with carrying out activities of daily living. Change in 6MWD from Baseline correlates with the current clinical standard for assessing patient functional status in the treatment of PH and is considered an objective measure of patient functional status. Subjects will be instructed to walk down a corridor at a comfortable speed as far as they can manage for 6 minutes. Distance \<500 meters suggests considerable exercise limitation; Distance 500-800 meters suggests moderate limitation; Distance \>800 meters (with no rests) suggests mild or no limitation. Peak exposure 6MWD will occur by conducting 6MWT within 10 to 60 minutes after the most recent dose of study drug dose.

Time frame: Baseline through Week 124 in RIN-PH-202 (Combined data from parent study RIN-PH-201 [NCT02630316] and this open-label extension study RIN-PH-202)

Secondary Outcomes

Change in Plasma Concentration of N-terminal Pro-Brain Natriuretic Peptide (NT-proBNP) From Baseline

The N-terminal pro-BNP (NT-proBNP) serum concentration is a useful biomarker associated with changes in right heart morphology and function. NT-proBNP serum concentration will be assessed to compare the severity of heart failure at Baseline and each visit through Week 124. Blood for NT-proBNP assessment must be drawn prior to conducting the 6-minute walk test (6MWT).

Time frame: Baseline through Week 124 in RIN-PH-202 (Combined data from parent study RIN-PH-201 [NCT02630316] and this open-label extension study RIN-PH-202)

Change in Percent Predicted Forced Vital Capacity (FVC) From Baseline

Change in pulmonary function following inhaled treprostinil therapy will be measured by Forced Vital Capacity (FVC), calculated from a Pulmonary Function Test (PFT) performed at Baseline and Weeks 12, 48, and 108.

Time frame: Baseline and Weeks 12, 48, and 108