Trials / Withdrawn
WithdrawnNCT04117880
A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study
- Status
- Withdrawn
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- PTC Therapeutics · Industry
- Sex
- All
- Age
- 2 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Ataluren | Oral |
Timeline
- Start date
- 2018-12-31
- Primary completion
- 2021-01-31
- Completion
- 2021-01-31
- First posted
- 2019-10-07
- Last updated
- 2019-10-07
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04117880. Inclusion in this directory is not an endorsement.