A Phase 2b/3, Randomized, Double-Blind, Placebo- Controlled, 24-Week Dose Ranging and Confirmatory Study to Evaluate the Safety and Efficacy of AV-101 in Patients with Pulmonary Arterial Hypertension (PAH)
Overall Design: Study AV-101-002 consists of three parts with continuous recruitment:
- Phase 2b Part - A Phase 2b placebo-controlled dose response part to evaluate 3 doses of AV-101 (10 mg, 35 mg, and 70 mg BID) and placebo in patients with PAH over 24 weeks from which one optimal dose will be selected based primarily upon the effect on change in PVR as well as safety and tolerability.
- Intermediate Part – A placebo-controlled part to evaluate 3 doses of AV-101 (10 mg, 35 mg, and 70 mg BID) and placebo in patients with PAH over 24 weeks, with 6MWD as the primary endpoint. Recruitment will be continuous between completing enrollment in the Phase 2b Part and selecting the optimal dose.
- Phase 3 Part - A confirmatory Phase 3 placebo-controlled part to evaluate 1 dose of AV-101 (optimal dose) and placebo in patients with PAH over 24 weeks, with 6MWD as the primary endpoint. The Phase 3 primary analysis on change in 6MWD will also include subjects on the optimal dose who were enrolled during the Intermediate Part
All subjects in the study will be able to enroll in a LTE (Study AV-101-003) on completion of the
24-week placebo-controlled parts of the study.
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Primary Investigator: Peter Engel