Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations

Status Completed

Clinical Trial Summary

The purpose of this study is to explore the combination of Ataluren and ivacaftor as a treatment for patients with a specific cystic fibrosis mutation

Clinical Trial Description

In about 10% of patients with CF, the defect in the gene is known as a stop mutation. This mutation truncates the cystic fibrosis transductive regulator (CFTR) protein production by introducing a premature stop in the messenger RNA (mRNA), this type of mutation is known as a stop mutation. Ataluren is a novel, oral drug that promotes this gene to work effectively and readthrough that premature "stop sign". It is hypothesized that ivacaftor may increase the efficacy of Ataluren by activating a specific protein that may not be functioning properly. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03256799
Study type	Interventional
Source	University of Alabama at Birmingham
Contact
Status	Completed
Phase	Phase 4
Start date	March 17, 2017
Completion date	February 16, 2018

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