Study 3
Study 3
Study 3
How TRIKAFTA was studied
How TRIKAFTA was studied
How TRIKAFTA was studied
This study evaluated the safety and tolerability of TRIKAFTA in children with cystic fibrosis (CF) age 6 through 11 years (Study 3). Because the focus was safety and side effects, the study did not use a placebo. The efficacy and safety of TRIKAFTA were evaluated in 2 studies of people with CF age 12 years and older, Study 1 and Study 2.
This study evaluated the safety and tolerability of TRIKAFTA in children with cystic fibrosis (CF) age 6 through 11 years (Study 3). Because the focus was safety and side effects, the study did not use a placebo. The efficacy and safety of TRIKAFTA were evaluated in 2 studies of people with CF age 12 years and older, Study 1 and Study 2.
This study evaluated the safety and tolerability of TRIKAFTA in children with cystic fibrosis (CF) age 6 through 11 years (Study 3). Because the focus was safety and side effects, the study did not use a placebo. The efficacy and safety of TRIKAFTA were evaluated in 2 studies of people with CF age 12 years and older, Study 1 and Study 2.
66 children with CF age 6 through 11 years with either one copy of the F508del mutation and a mutation defined in the study* or two copies of the F508del mutation participated in the 24-week safety study.
Each child took TRIKAFTA tablets every 12 hours with fat-containing food for 24 weeks (~6 months).
All participants knew they were taking TRIKAFTA, and no children in the study took placebo.
Each participant's dose of TRIKAFTA was based on their weight.
Learn about the recommended dose.
All participants continued to take their other prescribed CF therapies.
*Mutations that either do not make a CFTR protein or make a protein that is not responsive to ivacaftor and tezacaftor/ivacaftor.
*Mutations that either do not make a CFTR protein or make a protein that is not responsive to ivacaftor and tezacaftor/ivacaftor.
*Mutations that either do not make a CFTR protein or make a protein that is not responsive to ivacaftor and tezacaftor/ivacaftor.
What Should I Keep in Mind About the Study?
Because no one took placebo in the safety study, it is not known if changes seen in the study were due to TRIKAFTA.
Keep in mind that all results shown are an average of all people studied and differed among individuals and mutations. Your loved one may have a different experience.
This study took place during the COVID-19 pandemic. As a result, Vertex put in place certain processes and guidelines during the study, which may have affected the study results. Talk to your healthcare provider if you have any questions.
Results
Results
Results
What Were the Safety Study Results?
For children with CF age 6-11 years, the safety of TRIKAFTA observed in this study (Study 3) was similar to what was observed in people with CF age 12 years and older (Study 1).
Please see Important Safety Information.
What Else Was Found in the Study?
Lung function (FEV1*) increased by 10.2 percentage points on average through 24 weeks.
Lung function (FEV1*) increased by 10.2 percentage points on average through 24 weeks.
Lung function (FEV1*) increased by 10.2 percentage points on average through 24 weeks.
On average, children in the study started with an FEV1 of 88.8%.
*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.
*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.
*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.
Sweat chloride decreased by 60.9 mmol/L on average through 24 weeks.
Sweat chloride decreased by 60.9 mmol/L on average through 24 weeks.
Sweat chloride decreased by 60.9 mmol/L on average through 24 weeks.
On average, children started the study with a sweat chloride level of 102.2 mmol/L.
Sweat chloride is a measure of the amount of salt in a person's sweat.
Respiratory symptom score increased by 7 points on average through 24 weeks.
Respiratory symptom score increased by 7 points on average through 24 weeks.
Respiratory symptom score increased by 7 points on average through 24 weeks.
On average, children began the study with a score of 80.3 points.
Respiratory symptoms were measured using a tool called the Cystic Fibrosis Questionnaire-Revised Respiratory Domain (CFQ-R) score.
The average increase in CFQ-R score means that, overall, the symptoms studied have improved. It does not mean there was an improvement in each symptom measured.
Body mass index (BMI†) increased by 1 kg/m2 on average at 24 weeks.
Body mass index (BMI†) increased by 1 kg/m2 on average at 24 weeks.
Body mass index (BMI†) increased by 1 kg/m2 on average at 24 weeks.
For example, a child whose BMI put them in the 60th percentile of weight for children of the same gender and age rose to the 70th percentile.
†BMI=a measure of someone’s weight in relation to their height.
†BMI=a measure of someone’s weight in relation to their height.
†BMI=a measure of someone’s weight in relation to their height.
Because this was a safety trial, lung function, respiratory symptom scores, and BMI results were not included in the full Prescribing Information.
Clayton, age 5
F508del/G542X
Clayton, age 5
F508del/G542X
Clayton, age 5
F508del/G542X
Keep Learning
About TRIKAFTA
About TRIKAFTA
About TRIKAFTA
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What is TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)?
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in people aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA.
Talk to your healthcare provider to learn if you have an indicated CF gene mutation.
It is not known if TRIKAFTA is safe and effective in children under 2 years of age.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about TRIKAFTA?
TRIKAFTA can cause serious liver damage and liver failure. Liver failure leading to transplantation and death has been seen in some people with or without a history of liver problems taking TRIKAFTA.
Your healthcare provider will do blood tests to check your liver:
- before you start TRIKAFTA
- then every month during your first 6 months of taking TRIKAFTA
- then every 3 months during the next 12 months of taking TRIKAFTA
- then at least every year while you are taking TRIKAFTA
What is TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)?
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in people aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA.
Talk to your healthcare provider to learn if you have an indicated CF gene mutation.
It is not known if TRIKAFTA is safe and effective in children under 2 years of age.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about TRIKAFTA?
TRIKAFTA can cause serious liver damage and liver failure. Liver failure leading to transplantation and death has been seen in some people with or without a history of liver problems taking TRIKAFTA.
Your healthcare provider will do blood tests to check your liver:
- before you start TRIKAFTA
- then every month during your first 6 months of taking TRIKAFTA
- then every 3 months during the next 12 months of taking TRIKAFTA
- then at least every year while you are taking TRIKAFTA