Queen Square Centre for Neuromuscular Diseases



Phase II, multicentre, 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

Funder Association Française contre les Myopathies
CI Professor Volker Straub, Professor Francesco Muntoni
sites Newcastle, London GOSH

Newcastle: becky.davis@newcastle.ac.uk

London: ich.neuromusculartrials@ucl.ac.uk

More information


Olesoxime is a neuroprotective drug that acts to prevent the death of neurons. It has an excellent safety profile and has been well tolerated in phase I clinical trials in healthy volunteers.

Trial information

This was a multi-centre, double-blind, randomized, placebo-controlled study in patients with SMA type 2 or non-ambulant type 3.

The study was conducted in multiple centres across Europe. The sponsor was Trophos (French biopharmaceutical company) and the funder AFM (Association francaise contre les myopathies).

Primary objective

To assess efficacy, futility, safety and tolerability of the drug.


  • documented homozygous absence of SMN1 exon 7 and/or deletion and mutation on the other allele SMA II and III
  • not taking any medication for the treatment of SMA within 30 days before the study

Eligible patients can take oral salbutamol if started at least six months before enrolment and with a stable dose during the study.

This study involved 4 weeks of screening period followed by 24 months of treatment for each participant. Patients were randomised to receive either olesoxime or placebo in a 2:1 ratio. The drug or placebo was taken daily at a dose of 10mg/kg.


  • total 150 subjects 3-26 years old, with age of onset of symptoms at or before 3 years.
  • target of 30 patients in the UK

After 45 patients across Europe have taken it for 3 months, an independent Data Monitoring Committee (DMC) reviewed the findings.

Preliminary results

Results presented at the 66th American Academy of Neurology annual meeting in Philadelphia, 2014.

Trophos has been granted Orphan Drug designation for olesoxime for the treatment of SMA by the European Commission and the US Food and Drug Administration.