Safety and Efficacy of APD811 in Pulmonary Arterial Hypertension

Phase 2 Completed 61 enrolled United Therapeutics

RandomizedParallel-groupTriple-blindTreatment

Summary

The study was conducted as a placebo-controlled, randomized, 22-week double-blind study which included a dose titration period. An additional transition period occurred for those patients who elected to enroll into the open-label extension study, APD811-007. A total of 61 patients with PAH were enrolled.

Timeline

Start: 2014-12
Primary completion: 2017-06
Completion: 2017-06

Outcome

Mixed primary results

registry analysis (superiority test); APD811 vs Placebo; p = 0.0220; Multiple imputation 0.742 (95% CI 0.575 to 0.958); ANCOVA NCT02279160 ↗

registry analysis (superiority test); APD811 vs Placebo; p = 0.9002; Hodges-Lehmann estimate 0.0 (95% CI -28.0 to 30.0); Stratified Wilcoxon NCT02279160 ↗

Publications

Torres F, Farber H, Ristic A, McLaughlin V, Adams J, Zhang J, Klassen P, Shanahan W, Grundy J, Hoffmann I, Cabell C, Escribano Subias P, Sood N, Keogh A, D'Souza G, Rubin L. Efficacy and safety of ralinepag, a novel oral IP agonist, in PAH patients on mono or dual background therapy: results from a phase 2 randomised, parallel group, placebo-controlled trial. Eur Respir J. 2019 Oct 10;54(4):1901030. doi: 10.1183/13993003.01030-2019. Print 2019 Oct.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ralinepag	Small molecule	0.3 mg	Oral