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SAFETY AND EFFICACY RESULTS IN PEOPLE 12 YEARS AND OLDER

SAFETY STUDY IN PEOPLE 6-11 YEARS OLD

Safety Study in People 2-5 Years
Old

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.

Symptoms measured: mucus production, waking up from coughing, cough, difficulty coughing, wheezing, and congestion

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. These results are not included in the full Prescribing Information for TRIKAFTA.

†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.

Keep Learning

About TRIKAFTA

About TRIKAFTA

About TRIKAFTA

Hear about others' TRIKAFTA experiences Get fat-containing food ideas

Clayton, age 5
F508del/G542X

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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 patients 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 doctor 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

Before taking TRIKAFTA, tell your doctor about all of your medical conditions, including if you:

  • are allergic to TRIKAFTA or any ingredients in TRIKAFTA. See the Patient Information for a list of ingredients
  • have kidney problems
  • have or have had liver problems
  • are pregnant or plan to become pregnant. It is not known if TRIKAFTA will harm your unborn baby. You and your doctor should decide if you will take TRIKAFTA while you are pregnant
  • are breastfeeding or planning to breastfeed. It is not known if TRIKAFTA passes into your breast milk. You and your doctor should decide if you will take TRIKAFTA while you are breastfeeding

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

  • antibiotics such as rifampin (RIFAMATE®, RIFATER®) or rifabutin (MYCOBUTIN®)
  • seizure medicines such as phenobarbital, carbamazepine (TEGRETOL®, CARBATROL®, EQUETRO®), or phenytoin (DILANTIN®, PHENYTEK®)
  • St. John’s wort
  • antifungal medicines including ketoconazole, itraconazole (such as SPORANOX®), posaconazole (such as NOXAFIL®), voriconazole (such as VFEND®), or fluconazole (such as DIFLUCAN®)
  • antibiotics including telithromycin, clarithromycin (such as BIAXIN®), or erythromycin (such as ERY-TAB®)

What should I avoid while taking TRIKAFTA?

  • Avoid food or drink that contains grapefruit while you are taking TRIKAFTA

What are the possible side effects of TRIKAFTA?

TRIKAFTA can cause serious side effects, including:

  • Liver damage and worsening of liver function in people with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in people without liver disease
  • High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. These can be serious and may be a sign of liver injury. Your doctor will do blood tests to check your liver:
  • before you start TRIKAFTA
  • every 3 months during your first year of taking TRIKAFTA
  • every year while you are taking TRIKAFTA

Your doctor may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past.

Call your doctor right away if you have any of the following symptoms of liver problems:

  • pain or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • loss of appetite
  • nausea or vomiting
  • dark, amber-colored urine
  • Serious allergic reactions have happened to people who are treated with TRIKAFTA. Call your healthcare provider or go to the emergency room right away if you have any symptoms of an allergic reaction. Symptoms of an allergic reaction may include:
  • rash or hives
  • tightness of the chest or throat or difficulty breathing
  • swelling of the face, lips, and/or tongue, or difficulty swallowing
  • light-headedness or dizziness
  • Abnormality of the eye lens (cataract), which has happened in some children and adolescents treated with TRIKAFTA. If you are a child or adolescent, your doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts

The most common side effects of TRIKAFTA include:

  • headache
  • upper respiratory tract infection (common cold) including stuffy and runny nose
  • stomach (abdominal) pain
  • diarrhea
  • rash
  • increase in liver enzymes
  • increase in a certain blood enzyme called creatine phosphokinase
  • flu (influenza)
  • inflamed sinuses
  • increase in blood bilirubin

Tell your doctor if you have any side effect that bothers you or that does not go away.

These are not all the possible side effects of TRIKAFTA. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

For further information, please see full Prescribing Information, including Patient Information.

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Important Safety Information and Indication

Información Importante de Seguridad e Indicación

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AMPLIAR

COLLAPSE

REDUCIR

What is TRIKAFTA® (elexacaftor/tezacaftor/ ivacaftor and ivacaftor)?

TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients 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 doctor 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

Before taking TRIKAFTA, tell your doctor about all of your medical conditions, including if you:

  • are allergic to TRIKAFTA or any ingredients in TRIKAFTA. See the Patient Information for a list of ingredients
  • have kidney problems
  • have or have had liver problems
  • are pregnant or plan to become pregnant. It is not known if TRIKAFTA will harm your unborn baby. You and your doctor should decide if you will take TRIKAFTA while you are pregnant
  • are breastfeeding or planning to breastfeed. It is not known if TRIKAFTA passes into your breast milk. You and your doctor should decide if you will take TRIKAFTA while you are breastfeeding

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

  • antibiotics such as rifampin (RIFAMATE®, RIFATER®) or rifabutin (MYCOBUTIN®)
  • seizure medicines such as phenobarbital, carbamazepine (TEGRETOL®, CARBATROL®, EQUETRO®), or phenytoin (DILANTIN®, PHENYTEK®)
  • St. John’s wort
  • antifungal medicines including ketoconazole, itraconazole (such as SPORANOX®), posaconazole (such as NOXAFIL®), voriconazole (such as VFEND®), or fluconazole (such as DIFLUCAN®)
  • antibiotics including telithromycin, clarithromycin (such as BIAXIN®), or erythromycin (such as ERY-TAB®)

What should I avoid while taking TRIKAFTA?

  • Avoid food or drink that contains grapefruit while you are taking TRIKAFTA

What are the possible side effects of TRIKAFTA?

TRIKAFTA can cause serious side effects, including:

  • Liver damage and worsening of liver function in people with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in people without liver disease
  • High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. These can be serious and may be a sign of liver injury. Your doctor will do blood tests to check your liver:
  • before you start TRIKAFTA
  • every 3 months during your first year of taking TRIKAFTA
  • every year while you are taking TRIKAFTA

Your doctor may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past.

Call your doctor right away if you have any of the following symptoms of liver problems:

  • pain or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • loss of appetite
  • nausea or vomiting
  • dark, amber-colored urine
  • Serious allergic reactions have happened to people who are treated with TRIKAFTA. Call your healthcare provider or go to the emergency room right away if you have any symptoms of an allergic reaction. Symptoms of an allergic reaction may include:
  • rash or hives
  • tightness of the chest or throat or difficulty breathing
  • swelling of the face, lips, and/or tongue, or difficulty swallowing
  • light-headedness or dizziness
  • Abnormality of the eye lens (cataract), which has happened in some children and adolescents treated with TRIKAFTA. If you are a child or adolescent, your doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts

The most common side effects of TRIKAFTA include:

  • headache
  • upper respiratory tract infection (common cold) including stuffy and runny nose
  • stomach (abdominal) pain
  • diarrhea
  • rash
  • increase in liver enzymes
  • increase in a certain blood enzyme called creatine phosphokinase
  • flu (influenza)
  • inflamed sinuses
  • increase in blood bilirubin

Tell your doctor if you have any side effect that bothers you or that does not go away.

These are not all the possible side effects of TRIKAFTA. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

For further information, please see full Prescribing Information, including Patient Information.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

  • antibiotics such as rifampin (RIFAMATE®, RIFATER®) or rifabutin (MYCOBUTIN®)
  • seizure medicines such as phenobarbital, carbamazepine (TEGRETOL®, CARBATROL®, EQUETRO®), or phenytoin (DILANTIN®, PHENYTEK®)
  • St. John’s wort
  • antifungal medicines including ketoconazole, itraconazole (such as SPORANOX®), posaconazole (such as NOXAFIL®), voriconazole (such as VFEND®), or fluconazole (such as DIFLUCAN®)
  • antibiotics including telithromycin, clarithromycin (such as BIAXIN®), or erythromycin (such as ERY-TAB®)

What should I avoid while taking TRIKAFTA?

  • Avoid food or drink that contains grapefruit while you are taking TRIKAFTA

What are the possible side effects of TRIKAFTA?

TRIKAFTA can cause serious side effects, including:

  • Liver damage and worsening of liver function in people with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in people without liver disease
  • High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. These can be serious and may be a sign of liver injury. Your doctor will do blood tests to check your liver:
  • before you start TRIKAFTA
  • every 3 months during your first year of taking TRIKAFTA
  • every year while you are taking TRIKAFTA

Your doctor may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past.

Call your doctor right away if you have any of the following symptoms of liver problems:

  • pain or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • loss of appetite
  • nausea or vomiting
  • dark, amber-colored urine
  • Serious allergic reactions have happened to people who are treated with TRIKAFTA. Call your healthcare provider or go to the emergency room right away if you have any symptoms of an allergic reaction. Symptoms of an allergic reaction may include:
  • rash or hives
  • tightness of the chest or throat or difficulty breathing
  • swelling of the face, lips, and/or tongue, or difficulty swallowing
  • light-headedness or dizziness
  • Abnormality of the eye lens (cataract), which has happened in some children and adolescents treated with TRIKAFTA. If you are a child or adolescent, your doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts

The most common side effects of TRIKAFTA include:

  • headache
  • upper respiratory tract infection (common cold) including stuffy and runny nose
  • stomach (abdominal) pain
  • diarrhea
  • rash
  • increase in liver enzymes
  • increase in a certain blood enzyme called creatine phosphokinase
  • flu (influenza)
  • inflamed sinuses
  • increase in blood bilirubin

Tell your doctor if you have any side effect that bothers you or that does not go away.

These are not all the possible side effects of TRIKAFTA. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

For further information, please see full Prescribing Information, including Patient Information.