FREEDOM - M: Oral Treprostinil as Monotherapy for the Treatment of Pulmonary Arterial Hypertension (PAH)

• Status: Completed
• Trial end date: 2011-04-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: United Therapeutics
• Treatments: Treprostinil

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.