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Restore CFTR Protein Completed with Results
Study of ELX-02 in adults with cystic fibrosis who have at least one nonsense mutation. (Eloxx EL-012 (Kalydeco or Trikafta))
This study evaluated the safety and tolerability of ELX-02, a drug intended to improve CFTR function. Multiple doses of ELX-02, both alone and with ivacaftor, were tested in adults with cystic fibrosis who have at least one G542X mutation.
This was an open-label study, and all participants received ELX-02 for either one or two weeks alone or with ivacaftor. Multiple cohorts of participants were included in the study with each successive cohort receiving an increasing dose of ELX-02.
Eligibility
See other primary eligibility criteria for more information.
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Age:
18 Years and Older -
Mutation(s):
No Copies F508del -
FEV1% Predicted:
40% or greater
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Participants must have two G542X or phenotypically similar nonsense mutation, OR they must have one G542X or similar nonsense mutations (Allele 1 List) AND their second mutation must be one of the mutations included (Allele 2 List).
Study Results
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What We Learned:
This study was conducted between December 2019 and September 2022. While ELX-02 was well tolerated, no improvement in lung function or decrease in sweat chloride was observed.
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Primary Findings:
Effectiveness:
This small study of multiple different doses of ELX-002 did not show improvement in lung function or decrease in sweat chloride.
Safety:
Results have not been peer-reviewed and come from https://www.thepharmaletter.com/pharmaceutical/eloxx-tanks-as-elx-02-with-ivacaftor-fails-in-phase-ii-study-in-class-1-cf
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Citation:
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
No -
Placebo Controlled: ?more info
No -
Length of Participation:
9 weeks -
Number of Study Visits:
8
Additional Information
-
Phase: ?more info
Phase Two -
Study Sponsor: ?more info
Eloxx Pharmaceuticals Inc. -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
18 Years and Older -
Mutation(s):
No Copies F508del -
FEV1% Predicted:
40% or greater
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Participants must have two G542X or phenotypically similar nonsense mutation, OR they must have one G542X or similar nonsense mutations (Allele 1 List) AND their second mutation must be one of the mutations included (Allele 2 List).

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