Queen Square Centre for Neuromuscular Diseases


A trial of RO6885247 in Adult and Paediatric Patients with Spinal Muscular Atrophy 

Sponsor / Funder F. Hoffmann-La Roche Ltd
CI Professor Hanns Lochmüller
sites Newcastle, London GOSH

Newcastle: becky.davis@newcastle.ac.uk

London: ich.neuromusculartrials@ucl.ac.uk

More information on the trial

This is a multi-centre, randomised, double-blind, 12-week, placebo-controlled multiple dose Phase Ib study in adult and child patients with SMA.

Primary objective

To investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug RO6885247.


  • about 48 male and female SMA patients between the ages of 2−55 years (inclusive)
  • randomized in a 2:1 ratio to receive either the drug or placebo