| Methods |
Phase I, randomised, double‐blind, placebo‐controlled trial |
| Participants |
64 participants with SMA types I, II or III, aged 2–55 years or < 7 months |
| Interventions |
RO6885247 orally once daily for 12 weeks or placebo orally once daily for 12 weeks |
| Outcomes |
Safety (incidence of adverse events), pharmacokinetics (plasma concentrations of RO6885247 and RO6885247 exposure), pharmacodynamics (SMN protein levels in blood and in vivo splicing of SMN2 mRNA in blood), effect on CMAP, effect on electrical impedance myography. |
| Notes |
Recruitment of participants suspended since April 2015 for safety reasons. In parallel to the MOONFISH trial, Hoffmann‐La Roche have been investigating the effects of the long‐term use of RG7800 in animals. These animal studies are a standard requirement in the development of new medicines. In this study, they observed an unexpected safety finding in the eye of animals and subsequently immediately suspended dosing in the MOONFISH trial as a precautionary measure. Trial terminated in July 2015. No results available at time of writing. |
