Study of Ataluren in Participants With Nonsense Mutation Aniridia
Status:
Completed
Trial end date:
2021-01-22
Target enrollment:
Participant gender:
Summary
This study is designed to evaluate the effect of ataluren on Maximum Reading Speed as
measured using the Minnesota Low Vision Reading Test (MNREAD) Acuity Charts in participants
with nonsense mutation aniridia. This study involves a 4-week screening period, a 144-week
treatment period (Stage 1: Weeks 1 to 48 [double-masked treatment] and Stage 2: Weeks 49 to
144 [open label treatment]), an optional 96-week open label extension sub-study, and a 4-week
post-treatment follow-up period (either study completion or early termination). Participants
that choose not to participate in the sub-study will be required to complete the
post-treatment follow-up visit at the end of the Stage 2 open-label extension.