Clinical trial NCT00887978: Efficacy and Safety of Oral UT-15C Tablets to Treat Pulmonary Arterial Hypertension
Official title: A 16-Week, International, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study of the Efficacy and Safety of Oral UT-15C Sustained Release Tablets in Subjects With Pulmonary Arterial Hypertension
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Pulmonary Hypertension
Lead sponsor: United Therapeutics
Sponsor class: INDUSTRY
First posted: 2009-04-24
Enrollment: 310
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is an international, multi-center, randomized, double-blind, placebo-controlled study in subjects with PAH who are currently receiving approved therapy for their PAH (i.e., endothelin receptor antagonist and/or phosphodiesterase-5 inhibitor). Study visits will occur at 4 week intervals for 16 weeks with the key measure of efficacy being the 6-minute walk test. Study procedures include routine blood tests, medical history, physical exams, disease evaluation, and exercise tests.

Patients who complete all assessments for 16-weeks will also be eligible to enter an open-label, extension phase study (FREEDOM - EXT).


Eligibility criteria: Inclusion Criteria:

* A subject is eligible for inclusion in this study if all of the following criteria apply:
* Between 18 and 75 years of age, inclusive.
* Body weight at least 40 kg (approximately 90 lbs.)
* PAH that is either idiopathic/heritable; associated with appetite suppressant or toxin use; associated with collagen vascular disease; associated with repaired congenital shunts; associated with HIV.
* Currently receiving an approved endothelin receptor antagonist and/or an approved phosphodiesterase-5 inhibitor for at least 90 days and on a stable dose for at least the last 30 days.
* Baseline six-minute walk distance (6MWD) between 150-425 meters
* Previous testing (e.g., right heart catheterization, echocardiography) consistent with the diagnosis of PAH.
* Reliable and cooperative with protocol requirements.

Primary outcomes:
measure: 6-minute Walk Distance (6MWD)
timeFrame: Baseline and 16 weeks
Secondary outcomes:
measure: Clinical Worsening Assessment
timeFrame: Baseline and 16 Weeks
measure: Borg Dyspnea Score
timeFrame: Baseline and 16 Weeks
measure: World Health Organization (WHO) Functional Class
timeFrame: Baseline and 16 Weeks
measure: Symptoms of PAH
timeFrame: Baseline and 16 Weeks
measure: Dyspnea Fatigue Index
timeFrame: Baseline and 16 Weeks
measure: N-terminal proBNP (NT-proBNP)
timeFrame: Baseline and 16 Weeks
measure: Quality of Life (QoL) Assessment: Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR)
timeFrame: Baseline and 16 Weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama-Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Arizona Pulmonary Specialist, LTD
state: Arizona
city: Fresno
country: United States
facility: University of California, San Francisco-Fresno
state: California
city: La Jolla
country: United States
facility: UCSD Medical Center
state: California
city: Los Angeles
country: United States
facility: West Los Angeles VA Healthcare Center
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: Torrance
country: United States
facility: Harbor-UCLA Medical Center
state: California
city: Aurora
country: United States
facility: University of Colorado Health Science Center
state: Colorado
city: Jacksonville
country: United States
facility: University of Florida-Jacksonville
state: Florida
city: Weston
country: United States
facility: Cleveland Clinic Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Chicago
country: United States
facility: University of Chicago Hospitals
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Kansas City
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Louisville
country: United States
facility: Kentuckiana Pulmonary Associates
state: Kentucky
city: Portland
country: United States
facility: Maine Medical Center
state: Maine
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Pulmonary Critical Care Medicine, Brigham and Women's Hospital
state: Massachusetts
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: St. Louis
country: United States
facility: Washington University Hospital
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Newark
country: United States
facility: Newark Beth Israel Medical Center
state: New Jersey
city: Mineola
country: United States
facility: Winthrop University Hospital
state: New York
city: New York
country: United States
facility: Columbia University Presbyterian Medical Center
state: New York
city: Rochester
country: United States
facility: Mary M Parkes Center for Asthma, Allergy and Pulmonary Care
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Lindner Center
state: Ohio
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals Case Medical Center
state: Ohio
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Toledo
country: United States
facility: The University of Toledo
state: Ohio
city: Portland
country: United States
facility: Legacy Pulmonary Northwest
state: Oregon
city: Portland
country: United States
facility: OHSU
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Allegheny General Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Dallas
country: United States
facility: UT Southwestern
state: Texas
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
city: Murray
country: United States
facility: Intermountain Medical Center
state: Utah
city: Falls Church
country: United States
facility: Inova Transplant Center
state: Virginia
city: Leuven
country: Belgium
facility: University Hospital Gasthuisberg
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta Hospitals
state: Alberta
city: Vancouver
country: Canada
facility: Vancouver Coastal Health Respiratory Clinic
state: British Columbia
city: London
country: Canada
facility: London Health Sciences Center
state: Ontario
city: Toronto
country: Canada
facility: Toronto General Hospital
state: Ontario
city: Lille
country: France
facility: Hospital Claude Huriez
state: Cedex
city: Pessac
country: France
facility: Hospital Haut Leveque
state: Cedex
city: Brest
country: France
facility: Hospital Cavale Blanche
city: Dresden
country: Germany
facility: Universitaetsklinikum Dresden
city: Greifswald
country: Germany
facility: University Hospital Greifswald
city: Heidelberg
country: Germany
facility: Universitaetsklinikum Heidelberg
city: Haifa
country: Israel
facility: Pulmonology Department Rambam Medical Center
city: Haifa
country: Israel
facility: Lady Davis Carmel Medical Centre
city: Ramat Gan
country: Israel
facility: Pulmonary institute
city: Naples
country: Italy
facility: Azienda Ospedaliera Universitaria
city: Amsterdam
country: Netherlands
facility: VUMC
city: Lisboa
country: Portugal
facility: Hospital de Santa Marta
city: Barcelona
country: Spain
facility: Hospital Clinic I Provincial de Barcelona
city: Madrid
country: Spain
facility: Hospital 12 de Octubre
city: Lund
country: Sweden
facility: Lund University Hospital
city: London
country: United Kingdom
facility: Royal Free Hospital NHS Trust
Study officials:
affiliation: University of California, San Diego
name: Lewis Rubin, MD
role: STUDY_CHAIR
References:
citation: Richter MJ, Schermuly R, Seeger W, Rao Y, Ghofrani HA, Gall H. Relevance of angiopoietin-2 and soluble P-selectin levels in patients with pulmonary arterial hypertension receiving combination therapy with oral treprostinil: a FREEDOM-C2 biomarker substudy. Pulm Circ. 2016 Dec;6(4):516-523. doi: 10.1086/688671.
citation: Tapson VF, Jing ZC, Xu KF, Pan L, Feldman J, Kiely DG, Kotlyar E, McSwain CS, Laliberte K, Arneson C, Rubin LJ; FREEDOM-C2 Study Team. Oral treprostinil for the treatment of pulmonary arterial hypertension in patients receiving background endothelin receptor antagonist and phosphodiesterase type 5 inhibitor therapy (the FREEDOM-C2 study): a randomized controlled trial. Chest. 2013 Sep;144(3):952-958. doi: 10.1378/chest.12-2875.