Phase 3
Completed N=349
FREEDOM - M: Oral Treprostinil as Monotherapy for the Treatment of Pulmonary Arterial Hypertension (PAH)
Source: ClinicalTrials.gov NCT00325403 ↗Enrolled (actual)
349
Serious AEs
18.4%
Results posted
Mar 2013
Primary outcomePrimary: Six Minute Walk Distance (6MWD) — 339; 350; 343; 370 meters — p=0.0125
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Six Minute Walk Distance (6MWD) |
339; 350; 350; 360; 0; 7 | 0.0518 |
| SECONDARY Six Minute Walk Distance (6MWD) |
339; 350; 350; 360; 0; 7 | 0.0518 |
| SECONDARY Clinical Worsening Assessment |
8; 15 | 1.000 |
| SECONDARY World Health Organization Functional Classification for PAH |
1; 2; 29; 59; 38; 75 | 0.7380 |
| SECONDARY Borg Dyspnea Score |
3; 3; 3; 3; 0; 0 | 0.4887 |
| SECONDARY Dyspnea-Fatigue Index |
5.8; 6.2; 5.5; 5.9; -0.3; -0.3 | 0.6116 |
| SECONDARY Symptoms of PAH |
0.1; 0.0; -0.1; -0.2; 0.3; 0.2 | — |
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- Nursing or pregnant.
- Currently receiving an endothelin receptor antagonist, a phosphodiesterase-5 inhibitor, or prostacyclin within 30 days of Baseline.
- 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 (NCT00325403). 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. Informational only — not medical advice.