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Restore CFTR Protein Completed with Results
Phase 3 study of Ataluren in people with CF who have a nonsense mutation and are not taking aminoglycosides (PTC124-GD-021-CF)
This study evaluated the safety and effectiveness of the drug Ataluren (PTC124®) compared with placebo. This study was for people with CF who have a nonsense mutation and were not receiving chronic inhaled aminoglycosides.
Participants were randomized to receive either oral Ataluren three times per day (10mg in the morning, 10mg in the afternoon, and 20mg in the evening) or matched placebo for 48 weeks.
Eligibility
See other primary eligibility criteria for more information.
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Age:
6 Years and Older -
Mutation(s):
One Copy F508del or No Copies F508del -
FEV1% Predicted:
60 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
nonsense mutation in at least 1 allele of the CFTR gene
Study Results
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What We Learned:
This study found that there was no significant difference in lung function or rate of pulmonary exacerbation between participants taking Ataluren or placebo. This study did not meet its primary endpoint. Overall, Ataluren was well-tolerated. However, more participants taking Ataluren experienced gastrointestinal disorders than participants taking placebo.
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Primary Findings:
Effectiveness:
This study was conducted between August 2014 and November 2016. The study enrolled 279 patients (N= 138 Ataluren; N=136 placebo).
The primary study objective was to evaluate change in lung function (measured by absolute change in average ppFEV1) over 48 weeks. This study did not meet its primary endpoint. The 0.6% lung function improvement in favor of Ataluren versus placebo was not statistically significant.
Pulmonary exacerbation rate over 48 weeks was not significantly different between Ataluren (0.95) versus placebo (1.13).
Safety:
Overall, Ataluren was well-tolerated and the safety profile was similar between the two groups. However, more participants taking Ataluren experienced gastrointestinal disorders than participants taking placebo (22.1% versus 15.8%).
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Citation:
J Cyst Fibros 2020;DOI 10.1016/j.jcf.2020.01.007 [Epub ahead of print]
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
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Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
56 weeks -
Number of Study Visits:
10
Additional Information
-
Phase: ?more info
Phase Three -
Study Sponsor: ?more info
PTC -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
6 Years and Older -
Mutation(s):
One Copy F508del or No Copies F508del -
FEV1% Predicted:
60 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
nonsense mutation in at least 1 allele of the CFTR gene

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