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| Sponsor / Funder | F. Hoffmann-La Roche Ltd |
| CI | Professor Hanns Lochmüller |
| sites | Newcastle, London GOSH |
| Contact |
Newcastle: becky.davis@newcastle.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.
Recruitment
- 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