NCT01302600

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

Phase II, Multicenter, Randomized, Adaptive, Double-blind, Placebo Controlled Study to Assess Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Year Old Spinal Muscular Atrophy (SMA) Patients.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 165 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 3 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This study is a multicenter, double-blind, randomized, adaptive, parallel groups, placebo controlled 3-stage study in patients with SMA type 2 or non ambulant type 3. Stage 1 DMC 3-month safety assessment: An independent Data Monitoring Committee (DMC)will assess the safety of olesoxime every 3 months. Stage 2 Efficacy/futility analyses at one year: A first interim efficacy analysis will be performed after all patients have been treated for one year (52 weeks) in order to assess the need to continue the study to reach the planned objective. In the event of positive and significant results in favor of olesoxime, the study will be considered as successful and all patients will be switched to olesoxime to allow the assessment of the sustainability of the treatment effect and safety. If the results are significantly in favor of placebo, the study will be discontinued for failure (futility). Stage 3 Efficacy and safety analysis at two years: The expected study duration is of 2 years (104 weeks) to show efficacy. If the study is not discontinued for futility or medication regimen is changed due to success, the study will therefore continue until planned completion i.e. 104 weeks.

Conditions

• Spinal Muscular Atrophy Type II
• Spinal Muscular Atrophy Type III Non Ambulant

Interventions

Type	Name	Description
DRUG	Olesoxime	Liquid suspension formulation, 100 mg/ml at a dose of 10 mg/kg will be administered once a day with food at dinner
DRUG	Placebo	0.1ml/kg once a day with food at dinner.

Timeline

• Start date: 2010-11-01
• Primary completion: 2013-10-01
• Completion: 2013-10-01
• First posted: 2011-02-24
• Last updated: 2016-11-22

Locations

22 sites across 7 countries: Belgium, France, Germany, Italy, Netherlands, Poland, United Kingdom