| Methods |
Phase I, randomised, double‐blind, placebo‐controlled trial |
| Participants |
64 participants with SMA types I, II or III aged 2 to 55 years or below 7 months |
| Interventions |
RO6885247 orally once daily for 12 weeks versus 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 compound muscle action potential, 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. The trial in participants with SMA was therefore terminated in December 2016. Last update received December 22, 2016 (www.clinicaltrials.gov), but results have not yet been published. |
