A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)

Summary

The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of the following two clinical studies: TRO19622 CL E Q 1115-1 (open-label Phase Ib, multicenter, single- and multiple- dose study) or TRO19622 CL E Q 1275-1 (NCT01302600, Phase II/III, adaptive, parallel-group, double blind, randomized, placebo-controlled, multicenter, multinational study).

Timeline

Start

2016-01-20

Primary completion

2018-12-18

Completion

2018-12-18

Publications

• Vincent-Genod D, Rippert P, Coton J, Le Goff L, Barriere A, Berruyer A, Bernard M, Garde C, Gutierrez-Garcia M, Gilabert S, Gomes-Lisboade-Souza A, Daron A, Servais L, Thomann G, Vuillerot C. Scoring People With Spinal Muscular Atrophy on the Motor Function Measure Using the Microsoft Kinect. Pediatr Phys Ther. 2023 Jan 1;35(1):36-41. doi: 10.1097/PEP.0000000000000968. Epub 2022 Oct 25.

Drugs

Indications