Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
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|ClinicalTrials.gov Identifier: NCT01302600|
Recruitment Status : Completed
First Posted : February 24, 2011
Last Update Posted : November 22, 2016
|Condition or disease||Intervention/treatment||Phase|
|Spinal Muscular Atrophy Type II Spinal Muscular Atrophy Type III Non Ambulant||Drug: Olesoxime Drug: Placebo||Phase 2|
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 (Clinical Trial)|
|Actual Enrollment :||165 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||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.|
|Study Start Date :||November 2010|
|Actual Primary Completion Date :||October 2013|
|Actual Study Completion Date :||October 2013|
100 patients in this arm. liquid suspension
Liquid suspension formulation, 100 mg/ml at a dose of 10 mg/kg will be administered once a day with food at dinner
Placebo Comparator: Placebo
50 patients enrolled in this arm. liquid suspension
0.1ml/kg once a day with food at dinner.
- Motor Function Measure [ Time Frame: every 6 months ]Motor function Measure (MFM) D1+D2 score
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01302600
|Principal Investigator:||Enrico Bertini, MD||Bambino Gesu Hospital|