PH-201 (TRANSCEND)

PH-201 (TRANSCEND)

A Randomized, Double-blind, Placebo-conTRolled, SAfety aNd Efficacy Study of VI-0106 (Extended-release TaCrolimus) in SubjEcts with PAH aND Functional Limitations Despite Optimized Treatment with Available PAH Medications

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Pulmonary hypertension
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All genders
person-wave 18-75
world
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Overview
Principal Investigator: Nicholas Hill, MD

This study is a randomized, parallel group, double-blind, placebo-controlled, multicenter, safety and efficacy study in subjects with a confirmed diagnosis of PAH, and who are WHO functional class II - IV despite optimized therapy with one or more PAH medications. Approximately 300 subjects will be treated at approximately 80 sites worldwide. The study will consist of a doubleblind and an open-label treatment period. The double-blind treatment period will have an initial duration of 24 weeks, with analyses of primary and key secondary study endpoints at the completion of this period. This will be followed by a 4-week temporary VI-0106 withdrawal to evaluate the persistence of effects. On completion of the temporary VI-0106 withdrawal period, subjects will continue with their randomized treatment until 1 year after initial randomization, at which point they will become eligible for open-label treatment with VI-0106 for an additional year. 

Study details
Inclusion Criteria
  • Be an adult age 18 to 75 years inclusive
  • Have a diagnosis of WHO Group 1 PH: Pulmonary Arterial Hypertension
  • Screening 6MWD >75 meters to ≤450 meters. Measurements should be repeated with at least 4 hours (but less than 1 week) between attempts, and distances must be within 15% of each other using the highest value as a reference.
Exclusion Criteria
  • Total Lung Capacity (TLC) <60% predicted. If TLC is greater than or equal to ≥60% but <70% predicted, interstitial lung disease of greater than mild severity should be excluded by CT scan
  • FEV1/FVC <70% predicted or FEV1 <60% predicted within 6 months prior to study entry
  • BMI greater than or equal to ≥35 kg/m2
Study Requirements

The total duration of participation could be up to two years. Part 1 will consist of approximately 9 visits and Part 2 will consist of approximately 6 visits. The total amount of blood that will be drawn during the study is about 210 mL, or about 7/8 of a cup (1 US cup = 237 mL). CT imaging will be performed at screening, Week 24 and Week 28.