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A Phase III, International, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Clinical Worsening Study of UT-15C in Subjects with Pulmonary Arterial Hypertension Receiving Background Oral Monotherapy

Phase 3
Conditions
Health Condition 1: null- Pulmonary Arterial Hypertension
Registration Number
CTRI/2014/10/005122
Lead Sponsor
nited Therapeutics Corporation
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Closed to Recruitment of Participants
Sex
Not specified
Target Recruitment
0
Inclusion Criteria

•Between 18 and 75 years of age (inclusive)

•PAH that is idiopathic / heritable, PAH associated with connective tissue disease, HIV infection, repaired congenital systemic-to-pulmonary shunts (repaired > 1 year), or appetite suppressant / toxin use

•Subject, if known positive for HIV infection, has a CD4 lymphocyte count of at least 200 cells/mm3 and is receiving current standard of care anti-retroviral or other effective medication for treatment of HIV infection.

•Subject must have a Baseline 6MWD greater than or equal to 150 meters, in the absence of a concurrent injury, illness.

•The subject must be optimally treated with conventional pulmonary hypertension therapy (e.g. oral vasodilators, oxygen, digoxin, diuretics, anticoagulants as deemed appropriate by the Investigator) with no additions, discontinuations, or dose changes for a minimum of 10 days prior to randomization.

•The subject must have been receiving a PAH-approved oral monotherapy at a minimum dose that complies with the approved prescribing information for the product for at least 30 days prior to randomization.

Previous testing:

•Right heart catheterization within three years prior to the start of screening

•Echocardiography with evidence of clinically normal left systolic and diastolic ventricular function and absence of any clinically significant left sided heart disease

•Previous ventilation perfusion lung scan, and/or high resolution CT scan of the chest, and/or pulmonary angiography that are consistent with the diagnosis of PAH.

•Pulmonary function tests conducted within 6 months to confirm Total lung capacity (TLC) is at least 60% (predicted value) and Forced expiratory volume at one second (FEV1) of at least 50% (predicted value)

Exclusion Criteria

•Pregnant or lactating.

•Subject has previously received UT-15C.

•Subject has received a prostacyclin, (except if used during acute vasoreactivity

testing) within 30 days prior to randomization or had previous intolerance or significant

lack of efficacy to any prostacyclin, or prostacyclin analogue, that resulted in discontinuation or inability to titrate that therapy effectively.

•The subject has had any background conventional therapies for PAH added, removed or

dose adjusted (including but not limited to oxygen, vasodilators, diuretics, digoxin, anticoagulants) within 10 days prior to randomization. The exceptions are removal or

dose adjustments of anticoagulants and / or dose adjustments of diuretics.

•Any disease associated with PAH other than CTD, HIV infection, repaired (for atleast one year) congenital systemic-to-pulmonary shunt, PAH associated with appetite suppressant / toxin use.

•Uncontrolled sleep apnea

•Ischemic heart disease, including a previous myocardial infarction or symptomatic coronary artery disease within 6 months prior to Screening or a history of left sided myocardial disease

•Uncontrolled systemic hypertension

•ALT or AST levels at least greater than 3 times the upper limit of normal, clinically significant liver disease

•Musculoskeletal disorder (e.g., arthritis affecting the lower limbs, recent hip or knee joint replacement, artificial leg),

•Chronic renal insufficiency as defined by either a Screening creatinine value greater than 2.5 mg/dL

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
1. Change in 6 minute walk distance <br/ ><br>2. Time to first clinical worsening eventTimepoint: 1. From Baseline to Week 24 <br/ ><br>2. From randomization to approximately 4 years
Secondary Outcome Measures
NameTimeMethod
1. Borg Dyspnea Score <br/ ><br>2. Combined 6MWD and Borg Dyspnea Score <br/ ><br>3. WHO Functional Class for Pulmonary Hypertension <br/ ><br>4. N-terminal proBNP <br/ ><br>5. Hemodynamics (optional) <br/ ><br>6. Exercise capacity <br/ ><br>7. Safety (vital signs, adverse events, clinical laboratory parameters, electrocardiograms)Timepoint: 1. from Baseline to each <br/ ><br>follow-up assessment <br/ ><br>2. walk distances and Borg dyspnea scores from <br/ ><br>each follow-up 6MWT <br/ ><br>3. from Baseline to each follow up assessment <br/ ><br>4. Baseline to each follow up assessment <br/ ><br>5. Changes from Baseline to Week 24 <br/ ><br>6.Assessed by 6MWD at each visit other than Week 24
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