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