Pulmonary Hypertension
Conditions
Keywords
Pulmonary Arterial Hypertension
Brief summary
This study is an international, multi-center, randomized, double-blind, placebo-controlled study in subjects with PAH who are currently receiving approved therapy for their PAH (i.e., endothelin receptor antagonist and/or phosphodiesterase-5 inhibitor)or as a monotherapy treatment. Study visits will occur at 4 week intervals for 12 weeks with the key measure of efficacy being the 6-minute walk test. Study procedures include routine blood tests, medical history, physical exams, disease evaluation, and exercise tests. At the end of the first 12-weeks, the patient will be un-blinded. Patients will continue with another 12-Week open label portion with visits occuring at 4-week intervals. Patients who complete all assessments for 24-weeks will also be eligible to enter a 36 month open-label, extension phase study (FREEDOM - EXT).
Detailed description
This study is an international, multi-center, randomized, double-blind, placebo-controlled study in subjects with PAH who are currently receiving approved therapy for their PAH (i.e., endothelin receptor antagonist and/or phosphodiesterase-5 inhibitor)or as a monotherapy treatment. Study visits will occur at 4 week intervals for 12 weeks with the key measure of efficacy being the 6-minute walk test. Study procedures include routine blood tests, medical history, physical exams, disease evaluation, and exercise tests. At the end of the first 12-weeks, the patient will be un-blinded. Patients will continue with another 12-Week open label portion with visits occuring at 4-week intervals. Patients who complete all assessments for 24-weeks will also be eligible to enter a 36 month open-label, extension phase study (FREEDOM - EXT).
Interventions
DRUGUT-15C 1 mg
UT-15C 1 mg
DRUGUT-15C 0.25 mg
UT-15C 0.25 mg
DRUGUT-15C 5 mg
UT-15C 5 mg
DRUGPlacebo
Placebo
Sponsors
United Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Between 18 and 70 years of age, inclusive * Body weight at least 50 kilograms * PAH that is either idiopathic/familial; associated with repaired congenital systemic-to-pulmonary shunts (repaired ≥ 5 years); associated with collagen vascular disease; associated with HIV. * 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 or not currently receiving approved PAH therapy. * 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in six-minute walk distance from Baseline to Week 12 | 12 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Clinical Worsening Assessment | 12 weeks |
| Dyspnea Fatigue Index | 12 weeks |
| Symptoms of PAH | 12 weeks |
| World Health Organization (WHO) Functional Class | 12 weeks |
| Trough 6-Minute Walk Distance | 12 weeks |
| Borg Dyspnea Score | 12 weeks |
| Pro-B-type natriuretic peptide (Pro-BNP) | 12 weeks |
| Optional hemodynamic parameters | 12 weeks |
| Adverse events | 12 weeks |
| Clinical Laboratory parameters | 12 weeks |
| Electrocardiogram findings | 12 weeks |
| Trough Borg Dyspnea Score | 12 weeks |
Countries
Australia, Mexico, United States
Outcome results
None listed