NCT02647359

Study of Ataluren in Participants With Nonsense Mutation Aniridia

A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 39 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Aniridia

Interventions

Type	Name	Description
DRUG	Ataluren	Ataluren oral suspension will be administered as per the dose and schedule specified in the respective arms.
DRUG	Placebo	Placebo will be administered as per the schedule specified in the respective arm.

Timeline

• Start date: 2016-01-31
• Primary completion: 2021-01-22
• Completion: 2021-01-22
• First posted: 2016-01-06
• Last updated: 2022-05-27
• Results posted: 2022-05-27

Locations

3 sites across 2 countries: United States, Canada