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

United Therapeutics

Status and phase: Completed; Phase 3

Conditions: Pulmonary Hypertension

Treatments: Other: Placebo; Drug: Oral treprostinil (UT-15C) Sustained Release Tablets

Study type: Interventional

Funder types: Industry

Identifiers

NCT00325403

TDE-PH-302

Details and patient eligibility

About

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).

Enrollment: 349 patients

Sex: All

Ages: 12 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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349 participants in 2 patient groups, including a placebo group

UT-15C (oral treprositnil)

Active Comparator group

Description:

Subjects receive UT-15C (oral treprostinil) twice daily.

Treatment:

Drug: Oral treprostinil (UT-15C) Sustained Release Tablets

Placebo

Placebo Comparator group

Description:

Subjects receive placebo (sugar pill) twice daily.

Treatment:

Other: Placebo