Study to Assess the Efficacy and Safety of Raxone in LHON Patients

Phase 4 Completed 199 enrolled Santhera Pharmaceuticals

NaSingle-groupOpen-labelTreatment

Summary

LEROS is an open-label interventional Phase IV study, designed to further assess the efficacy and safety of Raxone® in the long-term treatment of LHON patients.

Timeline

Start: 2016-05
Primary completion: 2021-03-29
Completion: 2021-03-29

Outcome

Met primary endpoint

registry analysis (superiority test); Raxone; p = 0.0021; Odds Ratio (OR) 2.286 (95% CI 1.352 to 3.884); Wald Chi-square NCT02774005 ↗

paper LEROS meets its primary endpoint and confirms the long-term efficacy of idebenone PMID 38428428 ↗

release “Santhera Announces Phase 4 LEROS Trial with Raxone® Met Primary Endpoint in Patients with Leber’s Hereditary Optic Neuropathy” santhera.com ↗

Publications

Yu-Wai-Man P, Carelli V, Newman NJ, Silva MJ, Linden A, Van Stavern G, Szaflik JP, Banik R, Lubinski W, Pemp B, Liao YJ, Subramanian PS, Misiuk-Hojlo M, Newman S, Castillo L, Kociecki J, Levin MH, Munoz-Negrete FJ, Yagan A, Cherninkova S, Katz D, Meunier A, Votruba M, Korwin M, Dziedziak J, Jurkute N, Harvey JP, La Morgia C, Priglinger C, Lloria X, Tomasso L, Klopstock T; LEROS Study Group. Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. Cell Rep Med. 2024 Mar 19;5(3):101437. doi: 10.1016/j.xcrm.2024.101437. Epub 2024 Feb 29.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Idebenone	Small molecule	900 mg	—