Early Signs of Efficacy Study With Riociguat in Adult Homozygous Delta F508 Cystic Fibrosis Patients

Phase 2 Terminated 21 enrolled Bayer Merck Sharp & Dohme LLC · collab

RandomizedParallel-groupQuadruple-blindTreatment

Summary

Assessment of the safety, tolerability and early signs of efficacy of three times a day orally administered BAY63-2521 in adult delta F508 homozygous Cystic Fibrosis patients not on treatment with Orkambi

Timeline

Start: 2014-09-30
Primary completion: 2017-01-17
Completion: 2017-09-22

Outcome

Missed primary endpoint

Stopped: “Study was terminated after completion of Part 1, because Part 2 was no longer appropriate.”

paper Riociguat did not alter CFTR activity (change in sweat chloride) or lung function (change in ppFEV1) at doses up to 1.0 mg tid after 28 days. PMID 34419414 ↗

registry analysis (other test); Riociguat (Adempas, BAY63-2521) vs Placebo; Bayesian analysis -5.0 (90% CI -12.4 to 2.4) NCT02170025 ↗

Publications

Results Derichs N, Taylor-Cousar JL, Davies JC, Fajac I, Tullis E, Nazareth D, Downey DG, Rosenbluth D, Malfroot A, Saunders C, Jensen R, Solomon GM, Vermeulen F, Kaiser A, Willmann S, Saleh S, Droebner K, Sandner P, Bear CE, Hoffmann A, Ratjen F, Rowe SM; Rio-CF Study Group. Riociguat for the treatment of Phe508del homozygous adults with cystic fibrosis. J Cyst Fibros. 2021 Nov;20(6):1018-1025. doi: 10.1016/j.jcf.2021.07.015. Epub 2021 Aug 19.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Riociguat	Other / unclassified	1 mg	Oral