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