A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases

Summary

The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with sickle cell disease (SCD).

Timeline

Start

2017-04-11

Primary completion

2022-05-04

Completion

2022-05-04

Outcome

Results reported · no primary-endpoint met/missed verdict (typical of a dose-finding / safety study)

paper 22·7% (n=15) in the riociguat group and 31·3% (n=20) in the placebo group (difference -8·5% [90% CI -21·4 to 4·5]; p=0·19). PMID 38554715 ↗

Publications

• Wood KC, Nouraie SM, Gladwin MT, Novelli EM, Gordeuk VR, Morris CR, Minniti CP, Ataga KI, Desai PC, Abebe KZ, Straub AC. The CYB5R3 T117S Missense Variant is Associated with Attenuated Riociguat Efficacy in Sickle Cell Disease. Blood. 2026 Jul 8:blood.2026034193. doi: 10.1182/blood.2026034193. Online ahead of print.
• Gladwin MT, Gordeuk VR, Desai PC, Minniti C, Novelli EM, Morris CR, Ataga KI, De Castro L, Curtis SA, El Rassi F, Ford HJ, Harrington T, Klings ES, Lanzkron S, Liles D, Little J, Nero A, Smith W, Taylor JG 6th, Baptiste A, Hagar W, Kanter J, Kinzie A, Martin T, Rafique A, Telen MJ, Lalama CM, Kato GJ, Abebe KZ. Riociguat in patients with sickle cell disease and hypertension or proteinuria (STERIO-SCD): a randomised, double-blind, placebo controlled, phase 1-2 trial. Lancet Haematol. 2024 May;11(5):e345-e357. doi: 10.1016/S2352-3026(24)00045-0. Epub 2024 Mar 27.
• Azbell RCG, Desai PC. Treatment dilemmas: strategies for priapism, chronic leg ulcer disease, and pulmonary hypertension in sickle cell disease. Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):411-417. doi: 10.1182/hematology.2021000275.

Drugs

Indications