Inhaled Treprostinil in Participants With Pulmonary Hypertension Due to Chronic Obstructive Pulmonary Disease (PH-COPD) (PERFECT OLE)

An Open-Label Extension Study of Inhaled Treprostinil in Patients With Pulmonary Hypertension Due to Chronic Obstructive Pulmonary Disease (PH-COPD)

This open-label study will evaluate the safety of continued therapy with inhaled treprostinil in participants who have completed Study RIN-PH-304 (NCT03496623). This study hypothesizes that long-term safety findings will be similar to those observed in the randomized, placebo-controlled, double-blind, adaptive study 'A Phase 3, Randomized, Placebo-controlled, Double-blind, Adaptive Study to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Patients with Pulmonary Hypertension due to Chronic Obstructive Pulmonary Disease (PH-COPD)(RIN-PH-304).

Study Overview

• Status: Terminated
• Conditions: Chronic Obstructive Pulmonary Disease; Pulmonary Hypertension
• Intervention / Treatment: Drug: Inhaled treprostinil solution

Detailed Description

This is a multi-center, open-label study for eligible participants who completed all scheduled study visits during the Treatment Period of Study RIN-PH-304.

Participants who provide informed consent for this open-label extension study on or prior to the final study visit of RIN-PH-304 may participate in the study, provided all other eligibility criteria are met. The RIN-PH-304 final study visit and the RIN-PH-305 Enrollment Visit will occur on the same day.

All participants will reinitiate inhaled treprostinil at 3 breathes (18 micrograms [mcg]) 4 times daily (QID) during waking hours. Study drug doses should be maximized to tolerability throughout the study, and dose titrations should occur as rapidly as possible (as directed by the Investigator) with a target dosing regimen of 15 breaths QID or the maximum tolerated dose.

• Study Type: Interventional
• Enrollment (Actual): 41
• Phase: Phase 3

Contacts and Locations

Study Locations

• Israel
  • Haifa, Israel, 34362
    • Lady Davis Carmel Medical Centre
  • Jerusalem, Israel, 9112001
    • Hadassah-Hebrew University Hospital
  • Petah Tiva, Israel, 49100
    • Rabin Medical Center
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • The University of Alabama at Birmingham
  • California
    • Sacramento, California, United States, 95817
      • University of California Davis Medical Center
  • Florida
    • Clearwater, Florida, United States, 33765
      • St. Francis Sleep Allergy & Lung Institute
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic - Jacksonville
    • Jacksonville, Florida, United States, 32204
      • St. Vincent's Lung, Sleep, and Criticial Care Specialists
    • Miami, Florida, United States, 33136
      • University of Miami Hospital
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Pulmonary & Critical Care of Atlanta
    • Austell, Georgia, United States, 30106
      • Georgia Clinical Research
  • Illinois
    • Chicago, Illinois, United States, 60612
      • University of Illinois Medical Center
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • St. Vincent Medical Group, Inc.
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Kentuckiana Pulmonary Associates
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Brigham and Women's Hospital
  • Michigan
    • Grand Rapids, Michigan, United States, 49546
      • Spectrum Health
  • New York
    • Albany, New York, United States, 12208
      • Albany Medical Center
    • New York, New York, United States, 10029
      • Mount Sinai Medical Center
    • Rochester, New York, United States, 14623
      • University of Rochester Medical Center
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • University of North Carolina at Chapel Hill
  • Ohio
    • Cincinnati, Ohio, United States, 45219
      • The Carl and Edyth Lindner Research Center at The Christ Hospital
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15212
      • Allegheny General Hospital
    • Pittsburgh, Pennsylvania, United States, 15213
      • University of Pittsburgh Medical Center
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Virginia
    • Falls Church, Virginia, United States, 22042
      • Inova Fairfax Hospital
    • Richmond, Virginia, United States, 23230
      • Pulmonary Associates of Richmond, Inc.
    • Roanoke, Virginia, United States, 24014
      • Carilion Clinic
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • University of Wisconsin School of Medicine and Public Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Participant voluntarily gives informed consent to participate in the study.
2. Participant completed Study RIN-PH-304.
3. Women of childbearing potential (defined as less than 1 year post-menopausal and not surgically sterile) must agree to practice abstinence or use 2 highly effective methods of contraception (defined as a method of birth control that results in a low failure rate, [less than 1% per year], such as approved hormonal contraceptives, barrier methods [such as condom or diaphragm] used with a spermicide, or an intrauterine device) for the duration of study treatment and for 48 hours after discontinuing study drug.
4. Males with a partner of childbearing potential must agree to use a barrier method (condom) with a spermicide for the duration of treatment and for at least 48 hours after discontinuing study drug.

Exclusion Criteria:

1. The participant is pregnant or lactating.
2. The participant was prematurely discontinued from Study RIN-PH-304.
3. The participant is intolerant to inhaled prostanoid therapy.
4. The participant is unwilling or unable to use Sponsor-provided devices (actigraph, spirometer, or smart device).
5. The participant is scheduled to receive another investigational drug, device, or therapy during the course of this study.
6. Any other clinically significant illness or abnormal laboratory value(s) that, in the opinion of the Investigator, might adversely affect the interpretation of the study data.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Inhaled Treprostinil Solution; Inhaled treprostinil solution (0.6 milligrams per milliliter [mg/mL], 6 mcg/breath) QID during waking hours.	Drug: Inhaled treprostinil solution; Inhaled treprostinil solution per dose and schedule specified in the arm

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	An adverse event (AE) can be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. Treatment-emergent was defined as any AE occurring/worsening at any time after a participant was exposed to study drug up until 7 days after the last dose of study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.	Up to 4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline to Week 6 in 6-Minute Walk Distance (6MWD)	6MWD was calculated at peak exposure (10 to 60 minutes after dosing). 6-minute walk test (6MWT) was performed by standardized procedures for all participants. Participants were asked to walk a set course for 6 minutes (timed) and the distance walked (in meters) was recorded.	Baseline, Week 6
Change From Baseline to Week 6 in Borg Dyspnea Score	The Borg Dyspnea Score was a 11-point scale rating the maximum level of dyspnea experienced during the 6MWT. Scores range from 0 (no dyspnea at all) to 10 (very, very severe dyspnea), with lower scores indicating less exertion (a better outcome). The Borg Dyspnea Score was to be evaluated immediately after the 6MWT.	Baseline, Week 6
Change From Baseline to Week 6 in N-terminal Pro-brain Natriuretic Peptide (NT-proBNP)	The NT-proBNP concentration is a biomarker associated with changes in right heart morphology and function. Improvement is defined as a decrease in the NT-proBNP plasma concentration.	Baseline, Week 6

Collaborators and Investigators

• Sponsor: United Therapeutics
• Collaborators: Lung Biotechnology PBC

Study record dates

Study Major Dates

• Study Start (Actual): December 21, 2018
• Primary Completion (Actual): November 29, 2022
• Study Completion (Actual): November 29, 2022

Study Registration Dates

• First Submitted: January 2, 2019
• First Submitted That Met QC Criteria: January 3, 2019
• First Posted (Actual): January 7, 2019

Study Record Updates

• Last Update Posted (Estimated): December 18, 2023
• Last Update Submitted That Met QC Criteria: November 27, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: Vascular Diseases; Hypertension; Lung Diseases; Treprostinil
• Additional Relevant MeSH Terms: Pathologic Processes; Cardiovascular Diseases; Vascular Diseases; Respiratory Tract Diseases; Disease Attributes; Chronic Disease; Hypertension; Lung Diseases; Lung Diseases, Obstructive; Pulmonary Disease, Chronic Obstructive; Hypertension, Pulmonary; Antihypertensive Agents; Treprostinil
• Other Study ID Numbers: RIN-PH-305

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No