Clinical trial NCT03043651: Open-label Extension of Oral Treprostinil in Subjects With PH Associated With HFpEF
Official title: An Open-label Extension Study of Oral Treprostinil in Subjects With Pulmonary Hypertension (PH) Associated With Heart Failure With Preserved Ejection Fraction (HFpEF) - A Long-term Follow-up to Study TDE-HF-301
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Pulmonary Hypertension Associated With HFpEF
Keywords: Pulmonary Hypertension
HFpEF
6-Minute Walk Test
Oral Treprostinil
Lead sponsor: United Therapeutics
Sponsor class: INDUSTRY
First posted: 2017-02-06
Enrollment: 48
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This was an open-label study to evaluate the safety of continued therapy with oral treprostinil in subjects who completed Study TDE-HF-301. This study provided long-term, open-label data regarding the effect of continued long-term oral treprostinil therapy for the treatment of pulmonary hypertension (PH) associated with heart failure with preserved ejection fraction (HFpEF). Subject visits occurred at Baseline, Weeks 6, 12, 18, 24, and every 12 weeks thereafter until either oral treprostinil was commercially available to treat PH associated with HFpEF or the study was discontinued by the Sponsor.

The Sponsor terminated Studies TDE-HF-301 and TDE-HF-302 on 14 October 2019 due to slow enrollment. Safety data from the final subject in Study TDE-HF-302 were recorded on 02 March 2020. Due to the lower than expected number of subjects enrolled, the planned secondary efficacy-related endpoints were not analyzed.

Detailed description: Study TDE-HF-302 was a multicenter, open-label study in subjects participating and completing all required visits for Study TDE-HF-301. This study assessed the long-term safety of oral treprostinil in subjects with PH associated with HFpEF.

Subjects received oral treprostinil as 0.125-, 0.25-, 1-, and 2.5-mg sustained-release tablets. For subjects who were randomly allocated to receive oral treprostinil in Study TDE-HF-301, the initial dose of oral treprostinil was the same as the final dose in Study TDE-HF-301. Subjects randomly allocated to receive placebo in Study TDE-HF-301 were administered the initial dose of oral treprostinil at 0.125 mg 3 times daily (TID). Dose increases could occur in 0.125-mg increments every 72 hours at the discretion of the Investigator up to 6 mg TID, the maximum allowable dose determined by the Data Monitoring Committee during Study TDE-HF-301. Doses of study drug were to be increased in the absence of dose-limiting drug-related adverse events (AEs) to ensure that each subject received the optimal dose throughout the study. Subjects returned for visits at Weeks 6, 12, 18, and 24, and every 12 weeks thereafter. Subjects who terminated the study early were asked to return to the study center for a final evaluation.

Safety assessments consisted of AEs, clinical laboratory parameters, and clinical assessment of heart failure signs and symptoms.

Due to the lower than expected number of subjects enrolled, the planned secondary efficacy-related endpoints were not analyzed.

Eligibility criteria: Inclusion Criteria:

1. The subject participated in Study TDE-HF-301, remained on study drug, was compliant with study procedures and assessments during Study TDE-HF-301, and completed through Week 24 of that study.

Exclusion Criteria:

1. The subject was pregnant or lactating.
2. The subject was prematurely discontinued from Study TDE-HF-301 for any reason.
3. The subject developed a concurrent illness or condition during Study TDE-HF-301, which, in the opinion of the Investigator, represented a risk to the subject's overall health if they enrolled in this study.

Primary outcomes:
measure: Long-term Safety of Oral Treprostinil in Subjects With PH Associated With HFpEF for Subjects Who Completed Study TDE-HF-301
timeFrame: Baseline through study completion, up to approximately 25 months
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Banner University Medical Center Phoenix
state: Arizona
city: Tucson
country: United States
facility: University of Arizona
state: Arizona
city: Los Angeles
country: United States
facility: VA Healthcare System of Greater Los Angeles
state: California
city: Los Angeles
country: United States
facility: University of California Los Angeles Pulmonary Division
state: California
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Sacramento
country: United States
facility: University of California - Davis Medical Center
state: California
city: Santa Barbara
country: United States
facility: Santa Barbara Cottage Hospital
state: California
city: Aurora
country: United States
facility: Aurora Denver Cardiology Associates
state: Colorado
city: Aurora
country: United States
facility: University of Colorado Denver
state: Colorado
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Littleton
country: United States
facility: South Denver Cardiology
state: Colorado
city: Washington
country: United States
facility: MedStar Georgetown University Hospital
state: District of Columbia
city: Washington
country: United States
facility: Medical Faculty Associates, George Washington University
state: District of Columbia
city: Brandon
country: United States
facility: Bay Area Cardiology Associates
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic - Jacksonville
state: Florida
city: Jacksonville
country: United States
facility: St. Vincent's Lung, Sleep, and Critical Care Specialists
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary Group, P.A.
state: Florida
city: Orlando
country: United States
facility: Florida Hospital
state: Florida
city: Tampa
country: United States
facility: University of South Florida ; Tampa General Hospital
state: Florida
city: Weston
country: United States
facility: Cleveland Clinic of Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University Hospital
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Austell
country: United States
facility: Piedmont Physicians Georgia Lung
state: Georgia
city: Marietta
country: United States
facility: WellStar Medical Group
state: Georgia
city: Chicago
country: United States
facility: University of Illinois at Chicago Hospital
state: Illinois
city: Maywood
country: United States
facility: Loyola University Medical Center
state: Illinois
city: Oak Lawn
country: United States
facility: Advocate Christ Medical Center
state: Illinois
city: Peoria
country: United States
facility: OSF HealthCare
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University Health Methodist Research Institute, INC
state: Indiana
city: Indianapolis
country: United States
facility: Community Heart and Vascular Hospital
state: Indiana
city: Indianapolis
country: United States
facility: Saint Vincent Hospital and Health Services
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: West Des Moines
country: United States
facility: Iowa Heart Center
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky Medical Center
state: Kentucky
city: Louisville
country: United States
facility: Kentuckiana Pulmonary Associates
state: Kentucky
city: Louisville
country: United States
facility: University of Louisville Physicians Outpatient Center
state: Kentucky
city: South Portland
country: United States
facility: Chest Medicine Associates
state: Maine
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Brighton
country: United States
facility: St. Elizabeth's Medical Center
state: Massachusetts
city: Detroit
country: United States
facility: Henry Ford Health System
state: Michigan
city: Grand Rapids
country: United States
facility: Spectrum Health Medical Group
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Chesterfield
country: United States
facility: St. Luke's Hospital
state: Missouri
city: Kansas City
country: United States
facility: Saint Luke's Hospital of Kansas City
state: Missouri
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: Newark
country: United States
facility: Barnabas Health Lung Center
state: New Jersey
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: New York
country: United States
facility: Mount Sinai Medical Center
state: New York
city: Syracuse
country: United States
facility: Pulmonary Health Physicians, PC
state: New York
city: Asheville
country: United States
facility: Asheville Cardiology Associates
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Pinehurst
country: United States
facility: Pinehurst Medical Clinic
state: North Carolina
city: Cincinnati
country: United States
facility: The Lindner Research Center The Christ Hospital Health Network
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Toledo
country: United States
facility: University of Toldedo Medical Center
state: Ohio
city: Portland
country: United States
facility: The Oregon Clinic
state: Oregon
city: Lancaster
country: United States
facility: Lancaster General Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Allegheny General Hospital
state: Pennsylvania
city: Anderson
country: United States
facility: AnMed Health Pulmonary and Sleep Medicine
state: South Carolina
city: Anderson
country: United States
facility: VitaLink Research - Anderson
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Germantown
country: United States
facility: Stern Cardiovascular Foundation
state: Tennessee
city: Knoxville
country: United States
facility: Summit Medical Group
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: Baylor University Medical Center
state: Texas
city: Houston
country: United States
facility: Houston Methodist Research Institute
state: Texas
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
city: Lubbock
country: United States
facility: Texas Tech University Health Sciences Center
state: Texas
city: Murray
country: United States
facility: Intermountain Medical Center
state: Utah
city: Falls Church
country: United States
facility: Inova Heart and Vascular Institute
state: Virginia
city: Norfolk
country: United States
facility: Sentara Cardiovascular Research Institute
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Roanoke
country: United States
facility: Carilion Clinic
state: Virginia
city: Spokane
country: United States
facility: Providence Medical Research Center
state: Washington
city: Morgantown
country: United States
facility: West Virginia University Hospital
state: West Virginia
city: Milwaukee
country: United States
facility: Aurora St. Luke's Medical Center
state: Wisconsin
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: George Washington University
name: Mardi Gomberg-Maitland, MD
role: PRINCIPAL_INVESTIGATOR
References: