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Restore CFTR Protein Completed with Results
Ataluren (PTC 124) in Cystic Fibrosis (PTC124-GD-009-CF)
This trial looked at the safety and effectiveness of ataluren in people with CF and at least one CF nonsense mutation. Participants were randomized to receive either ataluren or placebo three times a day 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:
40 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
To be included in this study patients are required to have documentation of the simultaneous presence of a nonsense mutation in at least 1 allele of the CFTR gene and a CF-causing mutation in the other CFTR allele, as determined by gene sequencing from a laboratory certified by the College of American Pathologists (CAP), or under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an equivalent organization.
Study Results
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What We Learned:
Study results show that there was no significant change in lung function in the treatment group compared to the placebo group. Additionally, there were no serious safety concerns associated with ataluren treatment.
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Primary Findings:
Effectiveness:
This multi-center, multi-national phase 3 study was conducted between August 2009 and November 2011. A total of 238 participants were enrolled (120 in the ataluren group and 118 in the placebo group). The primary endpoint of the study, the relative change from baseline in % predicted FEV1 at 48 weeks, did not differ significantly between ataluren and placebo.
The secondary endpoint, the rate of pulmonary exacerbations did not reach statistical significance.
Safety:
Ataluren was generally well tolerated with incidence of adverse events similar in both groups through Week 48 except for cases of increased creatinine concentrations in the ataluren group.
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Citation:
Lancet 2014;DOI 10.1016/S2213-2600(14)70100-6;2(7):539-547
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
-
Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
48 weeks -
Number of Study Visits:
15
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:
40 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
To be included in this study patients are required to have documentation of the simultaneous presence of a nonsense mutation in at least 1 allele of the CFTR gene and a CF-causing mutation in the other CFTR allele, as determined by gene sequencing from a laboratory certified by the College of American Pathologists (CAP), or under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an equivalent organization.

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