drugset

drugset / Trial / NCT01302600

Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.

NCT01302600 ↗

Phase 2 Completed 165 enrolled Hoffmann-La Roche Association Française contre les Myopathies (AFM), Paris · collab

RandomizedParallel-groupQuadruple-blindTreatment

Summary

Assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 non ambulant patients aged 3-25 years

Timeline

Start: 2010-11
Primary completion: 2013-10
Completion: 2013-10

Outcome

Missed primary endpoint

paper Although the primary endpoint was not met PMID 28460889 ↗

paper treatment difference 2·00 points, 96% CI -0·25 to 4·25, p=0·0676 PMID 28460889 ↗

Publications

Bertini E, Dessaud E, Mercuri E, Muntoni F, Kirschner J, Reid C, Lusakowska A, Comi GP, Cuisset JM, Abitbol JL, Scherrer B, Ducray PS, Buchbjerg J, Vianna E, van der Pol WL, Vuillerot C, Blaettler T, Fontoura P; Olesoxime SMA Phase 2 Study Investigators. Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial. Lancet Neurol. 2017 Jul;16(7):513-522. doi: 10.1016/S1474-4422(17)30085-6. Epub 2017 Apr 28.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Olesoxime	Small molecule	10 mg/kg	Oral

Indications