MedTrace Logo

Study of Ataluren in Participants With Nonsense Mutation Aniridia

NCT02647359 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 39

Last updated 2022-05-27

Study results available
· View outcomes & findings →

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

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.

Sponsors & Collaborators

Principal Investigators

  • Quintus Ngumah, OD, PhD · PTC Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-31
Primary Completion
2021-01-22
Completion
2021-01-22

Countries

  • United States
  • Canada

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02647359 on ClinicalTrials.gov