Phase 3
N=350
FREEDOM-C: Oral Treprostinil in Combination With an Endothelin Receptor Antagonist (ERA) and/or a Phosphodiesterase-5 (PDE-5) Inhibitor for the Treatment of Pulmonary Arterial Hypertension (PAH)
Pulmonary Hypertension
Bottom Line
View on ClinicalTrials.gov: NCT00325442 ↗Enrolled (actual)
350
Serious AEs
18.6%
Results posted
Jun 2013
Primary outcome: Primary: Six Minute Walk Distance (6MWD) — 362.5; 362.5; 367.0; 381.0 meters — p=0.072
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Oral treprostinil (UT-15C) sustained release tablets (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- United Therapeutics
- Primary completion
- Sep 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Six Minute Walk Distance (6MWD) |
362.5; 362.5; 363.0; 370.5; 2.1; 5.5 | 0.238 |
| SECONDARY Borg Dyspnea Score |
4.26; 4.22; 4.64; 4.18; 0.38; -0.03 | 0.062 |
| SECONDARY Clinical Worsening Assessment |
12; 8 | 0.491 |
| SECONDARY Dyspnea-Fatigue Index |
5.5; 5.7; 5.1; 5.7; -0.4; 0.0 | 0.011 sig |
| SECONDARY World Health Organization Functional Classification for PAH |
1; 2; 48; 58; 114; 103 | — |
| SECONDARY Six Minute Walk Distance (6MWD) |
362.5; 362.5; 363.0; 370.5; 2.1; 5.5 | 0.238 |
| SECONDARY Six Minute Walk Distance (6MWD) |
362.5; 362.5; 363.0; 370.5; 2.1; 5.5 | 0.238 |
| SECONDARY Six Minute Walk Distance (6MWD) |
362.5; 362.5; 363.0; 370.5; 2.1; 5.5 | 0.238 |
| SECONDARY Change in Symptoms of PAH From Baseline to Week 16 |
0.0; 0.01; 0.00; -0.01; 0.00; -0.06 | — |
Summary
This study was an international, multi-center, randomized, double-blind, placebo-controlled study in subjects with PAH who were currently receiving approved therapy for their PAH (i.e., endothelin receptor antagonist and/or phosphodiesterase-5 inhibitor). Study visits occurred at 4 week intervals for 16 weeks; the key measure of efficacy was the 6-minute walk test. Study procedures included routine blood tests, medical history, physical exams, disease evaluation, and exercise tests. One optional substudy was also a part of FREEDOM-C at select centers - a hemodynamic substudy with a right heart catheterization at Baseline and Week 16.
Patients who completed all assessments for 16-weeks were also eligible to enter an open-label, extension phase study (FREEDOM - EXT).
Eligibility Criteria
Inclusion Criteria
- Between 12 and 70 years of age, inclusive.
- Body weight at least 45 kg (approximately 100 pounds).
- PAH that is either idiopathic/heritable (including PAH associated with appetite suppressant/toxin use); PAH associated with repaired congenital systemic-to-pulmonary shunts (repaired ≥ 5 years); PAH associated with collagen vascular disease; or PAH associated with HIV.
- Baseline 6-minute walk distance between 150 and 450 meters, inclusive.
- 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.
- Previous testing (e.g., right heart catheterization, echocardiography) consistent with the diagnosis of PAH.
- Reliable and cooperative with protocol requirements.
Exclusion Criteria
- Nursing or pregnant.
- Received a prostacyclin within the past 30 days.
- PAH due to conditions other than noted in the above inclusion criteria.
- 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.
Data sourced from ClinicalTrials.gov (NCT00325442). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.