Ignite Creation Date:
2025-12-24 @ 7:42 PM
Ignite Modification Date:
2026-02-13 @ 3:16 PM
Study NCT ID:
NCT02456103
Status:
TERMINATED
Last Update Posted:
2020-04-27
First Post:
2015-05-26
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
Sponsor:
PTC Therapeutics
Organization:
Study Overview
Official Title:
Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis
Status:
TERMINATED
Status Verified Date:
2020-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Cystic Fibrosis (CF) data from the double-blind CF Study PTC124-GD-021-CF did not meet endpoints.
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.
Detailed Description:
The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).
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?: