A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia

Phase 2

Withdrawn

• Conditions: Aniridia
• Interventions: Drug: Ataluren

• Registration Number: NCT04117880
• Lead Sponsor: PTC Therapeutics

Brief Summary

This is a Phase 2, multicenter, open-label study evaluating the overall systemic and ocular safety profile of ataluren in nonsense mutation aniridia as determined by the incidences of treatment-emergent adverse events (TEAEs) as well as abnormal findings on laboratory assessments, vital signs, physical examinations, ophthalmoscopy, and slit-lamp examination.

Participants who complete PTC124-GD-028 ANI (NCT02647359) meet all inclusion and none of the exclusion criteria will be enrolled into this study.

Detailed Description

This study was a planned extension study of PTC124-GD-028 ANI (NCT02647359) and was never initiated.

Study Timeline

• Study Start: 2018-12-31
• Status Verified: 2019-09
• Study First Submitted: 2019-09-19
• Study First Submitted QC: 2019-10-03
• Last Update Submitted: 2019-10-03
• Study First Posted: 2019-10-07
• Last Update Posted: 2019-10-07
• Primary Completion: 2021-01-31
• Study Completion: 2021-01-31

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ataluren	Ataluren	Ataluren Oral suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mid-day, and 20 mg/kg in the evening).

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	104 weeks

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Severity of Corneal Keratopathy at Week 104	104 weeks
Change From Baseline in Visual Acuity at Week 104	104 weeks