Ignite Creation Date:
2025-12-24 @ 3:06 PM
Ignite Modification Date:
2025-12-24 @ 3:06 PM
Study NCT ID:
NCT02090959
Status:
TERMINATED
Last Update Posted:
2020-08-11
First Post:
2014-03-17
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
Sponsor:
PTC Therapeutics
Organization:
Study Overview
Official Title:
A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
Status:
TERMINATED
Status Verified Date:
2020-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Sponsor decision due to commercial availability of ataluren.
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren \[PTC124-GD-020-DMD {NCT01826487}\]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study.
This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.
This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: