Assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 non ambulant patients aged 3-25 years
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
165
University Hospitals
Ghent, Belgium
University Hospitals
Leuven, Belgium
Motor Function Measure
Motor function Measure (MFM) D1+D2 score
Time frame: every 6 months
responder analyses on MFM and HFMS, time to 4 point decrease on HFMS, CMAP/MUNE, PedsQL, FVC, CGI and safety
Time frame: every 3 months
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Hôpital Femme-Mère-Enfant
Bron, France
Hôpital Raymond Poincaré
Garches, France
CHRU Hôpital R. Salengro
Lille, France
Hôpital La Timone
Marseille, France
CHU de Montpellier, Hôpital Gui de Chauliac
Montpellier, France
Groupe hospitalier Armand-Trousseau
Paris, France
University of Essen
Essen, Germany
Universitat Klinikum Freiburg
Freiburg im Breisgau, Germany
...and 12 more locations