Safety and Efficacy of Olesoxime (TRO19622) in 3-25 yrs SMA patients
Research type
Research Study
Full 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
IRAS ID
57754
Contact name
Francesco Muntoni
Sponsor organisation
Trophos
Eudract number
2010-020386-24
ISRCTN Number
Not Submitted
Clinicaltrials.gov Identifier
N/A
Research summary
Spinal Muscular Atrophy is an autosomal recessive genetic disease that affects the motor neurons of the voluntary muscles that are used for activities such as crawling, walking, head and neck control, and swallowing. Approximately 1 in 6000 babies born are affected and about 1 in 40 people are genetic carriers. SMA patients are divided into three subtypes depending on disease onset and severity but all suffer from degeneration of motor neurons controlling voluntary muscles with proximal limb and trunk muscle weakness leading to respiratory distress and in the most severe cases, ultimately death. 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. The patients must not be walkers to be eligible for the study. There are 3 stages to the study. Stage 1 Safety assessment: An independent Data monitoring Committee (DMC) will assess the safety of olesoxime every 3 months. Stage 2 Efficacy/futility analysis 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 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 duration will be 33 months with a 9 months recruitment period and a 2 years (104 weeks) treatment period. The total number of subjects will be 150 with 100 in the olesoxime group and 50 in placebo group.
REC name
London - Hampstead Research Ethics Committee
REC reference
10/H0720/92
Date of REC Opinion
31 Jan 2011
REC opinion
Further Information Favourable Opinion