FREEDOM - M: Oral Treprostinil as Monotherapy for the Treatment of Pulmonary Arterial Hypertension (PAH)
A 12-Week, International, Multicenter, Double-Blind, Randomized, Placebo-Controlled Comparison of the Efficacy and Safety of Oral UT-15C Sustained Release Tablets in Subjects With Pulmonary Arterial Hypertension
1 other identifier
interventional
349
13 countries
77
Brief Summary
This study was an international, multicenter, randomized (2:1 active:placebo), double-blind, placebo-controlled study in subjects with PAH who were NOT currently receiving approved therapy for their PAH. Study visits occurred at 4 week intervals for 12 weeks (with an additional visit at Week 11) with the key measure of efficacy being the 6-minute walk test. Study procedures included routine blood tests, medical history, physical exams, disease evaluation, and exercise tests. Two optional substudies were also a part of FREEDOM-M at select centers - a hemodynamic substudy with a right heart catheterization at Baseline and Week 12 and a genetics and biomarkers substudy with blood samples collected at Baseline and Week 12. Patients who completed all assessments for 12 weeks were also eligible to enter an open-label, extension phase study (FREEDOM - EXT).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Oct 2006
Longer than P75 for phase_3
77 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 11, 2006
CompletedFirst Posted
Study publicly available on registry
May 12, 2006
CompletedStudy Start
First participant enrolled
October 1, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2011
CompletedResults Posted
Study results publicly available
March 18, 2013
CompletedJanuary 3, 2024
February 1, 2013
4.5 years
May 11, 2006
January 3, 2013
December 12, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Six Minute Walk Distance (6MWD)
Placebo corrected change in six minute walk distance (6MWD) from Baseline to Week 12, correlates with the historical clinical standard for assessing patient functional status in the treatment of PAH and is considered an objective measure of patient functional status by the American Thoracic Society (ATS). The six minute walk test was to be conducted 3 to 6 hours after the previous dose of study drug. The Hodges-Lehmann median difference between treatment groups was used to estimate the treatment effect on 6MWD from Baseline to Week 12. A rank-based methodology was used instead of parametric-based methodology to avoid statistical bias caused by extreme outliers resulting from the handling of data that are missing due to death or clinical worsening of PAH. It is a more robust estimator than the between-treatment difference in medians.
Baseline and Week 12
Secondary Outcomes (8)
Six Minute Walk Distance (6MWD)
Baseline and Week 11
Six Minute Walk Distance (6MWD)
Baseline and Week 8
Six Minute Walk Distance (6MWD)
Baseline and Week 4
Clinical Worsening Assessment
Baseline and Week 12
World Health Organization Functional Classification for PAH
Baseline and Week 12
- +3 more secondary outcomes
Study Arms (2)
UT-15C (oral treprositnil)
ACTIVE COMPARATORSubjects receive UT-15C (oral treprostinil) twice daily.
Placebo
PLACEBO COMPARATORSubjects receive placebo (sugar pill) twice daily.
Interventions
Sustained release oral tablet, twice daily
Eligibility Criteria
You may qualify if:
- Between 12 and 75 years of age, inclusive.
- Body weight at least 40 kg with a Body Mass Index \< 45
- PAH that is either idiopathic/heritable; associated with repaired congenital systemic-to-pulmonary shunts (repaired ≥ 5 years); associated with collagen vascular disease; associated with HIV.
- Previous testing (e.g., right heart catheterization, echocardiography) consistent with the diagnosis of PAH.
- Baseline 6-minute walk distance between 200 and 425 meters, inclusive.
- Reliable and cooperative with protocol requirements.
You may not qualify if:
- Nursing or pregnant.
- Currently receiving an endothelin receptor antagonist, a phosphodiesterase-5 inhibitor, or prostacyclin within 30 days of Baseline.
- History of uncontrolled sleep apnea, renal insufficiency, anemia, left sided heart disease, uncontrolled systemic hypertension, or parenchymal lung disease.
- Use of an investigational drug within 30 days of Baseline.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (77)
University of Alabama-Birmingham
Birmingham, Alabama, United States
Arizona Pulmonary Specialist
Phoenix, Arizona, United States
Mayo Clinic Arizona
Phoenix, Arizona, United States
University of California, San Francisco-Fresno
Fresno, California, United States
West Los Angeles VA Healthcare Center
Los Angeles, California, United States
UC Davis Medical Center
Sacramento, California, United States
Harbor-UCLA Medical Center
Torrance, California, United States
University of Colorado Health Science Center
Aurora, Colorado, United States
University of Florida - Shands Hospital
Jacksonville, Florida, United States
Cleveland Clinic Florida
Weston, Florida, United States
Emory University Hospital
Atlanta, Georgia, United States
University of Chicago Hospitals
Chicago, Illinois, United States
University of Iowa Hospital and Clinics
Iowa City, Iowa, United States
Kentuckiana Pulmonary Associates
Louisville, Kentucky, United States
Alexandria Cardiology Clinic
Alexandria, Louisiana, United States
Maine Medical Center
Portland, Maine, United States
University of Maryland School of Medicine
Baltimore, Maryland, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Tufts Medical Center
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University Hospital
St Louis, Missouri, United States
Nebraska Medical Center
Omaha, Nebraska, United States
Newark Beth Israel Medical Center
Newark, New Jersey, United States
Winthrop University Hospital
Mineola, New York, United States
Columbia Presbyterian Medical Center
New York, New York, United States
Mary M Parkes Center for Asthma, Allergy and Pulmonary Care - University of Rochester
Rochester, New York, United States
The Lindner Center
Cincinnati, Ohio, United States
University of Cincinnati
Cincinnati, Ohio, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, United States
University Hospitals of Cleveland
Cleveland, Ohio, United States
Ohio State University
Columbus, Ohio, United States
Legacy Clinic Northwest
Portland, Oregon, United States
Oregon Health and Science University
Portland, Oregon, United States
Allegheny General Hospital
Pittsburgh, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Texas Southwestern
Dallas, Texas, United States
Baylor College of Medicine, Pulmonary & Critical Care
Houston, Texas, United States
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
Inova Fairfax Hospital
Falls Church, Virginia, United States
University of Washington Medical Center
Seattle, Washington, United States
Heart Care Associates
Milwaukee, Wisconsin, United States
Universitaet Wien
Vienna, Austria
Hospital Erasme
Brussels, Belgium
University Hospital Gasthuisberg
Leuven, Belgium
Peter Lougheed Centre
Calgary, Alberta, Canada
London Health Science Centre
London, Ontario, Canada
SMBD Jewish General Hospital
Montreal, Quebec, Canada
Beijing Shijitan Hospital
Beijing, China
Peking Union Medical College Hospital
Beijing, China
Shanghai Pulmonary Hospital
Shanghai, China
Hospital Antoine Beclere
Clamart, France
PRIME Hospitals
Hyderbad, Andhra Pradesh, India
Care Institute of Medical Science
Ahmedabad, Gujarat, India
Lifecare Institute of Medical Sciences & Research
Ahmedabad, Gujarat, India
Narayana Hrudayalaya Institute of Medical Sciences
Bangalore, Karnataka, India
Asian Heart Institute and Research Centre
Parel Mumbai, Maharashtra, India
Poona Hospital and Research Centre
Pune, Maharashtra, India
Sri Ramachandra Medical College
Chennai, Tamil Nadu, India
G. Kuppuswamy Naidu Memorial Hospital
Coimbatore, Tamil Nadu, India
K. S. Hospital
Chennai, Tamil Nadu, India
Sir Ganga Ram Hospital
New Delhi, India
Ruby Hall Clinic
Pune, India
Care Hospital
Visakhapatnam, Andra Pradesh, India
Hadassah Ein-Kerem Medical Center
Jerusalem, Israel
Rabin Medical Center
Petah Tikva, Israel
Tel Hashomer Medical Center
Ramat Gan, Israel
Universita degli Studi Bologna
Bologna, Italy
Instituto Nacional de Cardiologia
Mexico City, Mexico City, Mexico
Hospital Universitario UANL
Monterrey, Nuevo León, Mexico
Unidad de Investigacion Clinica en Medicina (UDICEM)
Monterrey, Mexico
VU Medisch Centrum (VUCM)
Amsterdam, Netherlands
Krakowski Szpital Specjalistyczny im. (Kraków Specialist Hospital)
Krakow, Poland
National Tuberculosis and Lung Disease Research Institute
Warsaw, Poland
Wojewódzki Szpital Specjalistyczny we Wroclawiu (Provincial Specialist Hospital in Wrocław)
Wroclaw, Poland
Auxilio Mutuo Hospital
Guaynabo, Puerto Rico
Related Publications (1)
Jing ZC, Parikh K, Pulido T, Jerjes-Sanchez C, White RJ, Allen R, Torbicki A, Xu KF, Yehle D, Laliberte K, Arneson C, Rubin LJ. Efficacy and safety of oral treprostinil monotherapy for the treatment of pulmonary arterial hypertension: a randomized, controlled trial. Circulation. 2013 Feb 5;127(5):624-33. doi: 10.1161/CIRCULATIONAHA.112.124388. Epub 2013 Jan 10.
PMID: 23307827DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Kevin Laliberte; Senior Director, Product Development
- Organization
- United Therapeutics Corporation
Study Officials
- STUDY DIRECTOR
Kevin Laliberte, PharmD
United Therapeutics
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 11, 2006
First Posted
May 12, 2006
Study Start
October 1, 2006
Primary Completion
April 1, 2011
Study Completion
April 1, 2011
Last Updated
January 3, 2024
Results First Posted
March 18, 2013
Record last verified: 2013-02