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Restore CFTR Protein Completed with Results
Study of VX-445 triple combination in children 6-11 years old with cystic fibrosis (Vertex VX18-445-106)
This study evaluated the safety and effectiveness of the triple combination modulator therapy, elexacaftor/tezacaftor/ivacaftor (Trikafta®) in children ages 6-11 years with CF who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.
This 24-week study was open-label meaning all participants received elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA). Children weighing <30 kg received 50% of the ELX/TEZ/IVA adult daily dose (ELX 100 mg once daily, TEZ 50 mg once daily, and IVA 75 mg every 12 hours) while children weighing ≥30 kg received the full adult daily dose (ELX 200 mg once daily, TEZ 100 mg once daily, and IVA 150 mg every 12 hours).
Eligibility
See other primary eligibility criteria for more information.
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Age:
6 Years to 11 Years -
Mutation(s):
Two Copies F508del or One Copy 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
Study participants must have two copies of the F508del CFTR mutation, or one copy of F508del and one copy of a minimal function CFTR mutation. Click here to see a list of eligible minimal function mutations.
Study Results
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What We Learned:
This study found that ELX/TEZ/IVA in children ages 6-11 years with CF, who have either two copies of the F508del mutation or one copy of the F508del mutation, and one minimal function mutation was generally well tolerated and the safety data was consistent with that observed in previous Phase 3 studies. The most common reported adverse events included cough, headache, and fever.
In addition, clinically meaningful improvements were seen, including a 10.2% improvement in lung function (ppFEV1) (95% confidence interval [CI], 7.9 to 12.6), a significant decrease in sweat chloride concentration (mean absolute change from baseline of −60.9 mmol/L through Week 24;
95% CI, −63.7 to −58.2), and improvements in the Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score (mean absolute change from baseline of 7.0 points; 95% CI, 4.7 to 9.2).
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Primary Findings:
Effectiveness:
Safety:
This study was conducted from August 5, 2019 to August 7, 2020. The study enrolled 66 children and 64 children completed the treatment period (97%).
The primary endpoint of the study was safety and tolerability, and the results showed that ELX/TEZ/IVA was generally well-tolerated, and the safety data was consistent with that observed in previous Phase 3 studies. The most common reported adverse events included cough, headache, and fever.
In addition, clinically meaningful improvements were seen, including a 10.2% improvement in lung function (ppFEV1) (95% confidence interval [CI], 7.9 to 12.6), a significant decrease in sweat chloride concentration (mean absolute change from baseline of −60.9 mmol/L through Week 24;
95% CI, −63.7 to −58.2), and improvements in the Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score (mean absolute change from baseline of 7.0 points; 95% CI, 4.7 to 9.2).
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Citation:
Am J Respir Crit Care Med ;DOI DOI: 10.1164/rccm.202102-0509OC
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:
32 weeks -
Number of Study Visits:
9
Additional Information
-
Phase: ?more info
Phase Three -
Study Sponsor: ?more info
Vertex -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
6 Years to 11 Years -
Mutation(s):
Two Copies F508del or One Copy 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
Study participants must have two copies of the F508del CFTR mutation, or one copy of F508del and one copy of a minimal function CFTR mutation. Click here to see a list of eligible minimal function mutations.

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