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Trials / Terminated

TerminatedNCT02456103

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis

Status
Terminated
Phase
Phase 3
Study type
Interventional
Enrollment
246 (actual)
Sponsor
PTC Therapeutics · Industry
Sex
All
Age
6 Years
Healthy volunteers
Not accepted

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).

Conditions

Interventions

TypeNameDescription
DRUGAtalurenAtaluren will be provided as a vanilla-flavored powder to be mixed with water or milk.

Timeline

Start date
2015-08-31
Primary completion
2017-06-02
Completion
2017-06-02
First posted
2015-05-28
Last updated
2020-04-27
Results posted
2020-04-15

Locations

70 sites across 16 countries: United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Spain, United Kingdom

Source: ClinicalTrials.gov record NCT02456103. Inclusion in this directory is not an endorsement.

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis (NCT02456103) · Clinical Trials Directory