A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
Status:
Terminated
Trial end date:
2016-10-01
Target enrollment:
Participant gender:
Summary
This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects
with DMD open to eligible US and Canadian subjects who previously participated in the
following studies of drisapersen: DMD114876, DMD114044 and DMD114349. Subjects will receive
6mg/kg subcutaneous drisapersen on a weekly basis. For subjects who have previously
experienced significant safety or tolerability issues or who experience these during the
study, there is the potential of an alternate intermittent dosing arm that will be given as a
regimen of 6 mg/kg weekly for 8 weeks followed by 4 weeks off treatment. For subjects who
experience or have previously experienced significant safety/tolerability issues, side
effects or reactions or intermittent dosing, intravenous dosing will be made available.