Study 4

Study 4

Study 4

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 2 through 5 years (Study 4). 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 2 through 5 years (Study 4). 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 2 through 5 years (Study 4). 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 also evaluated:

Sweat chloride, which is a measure of the amount of salt in a person’s sweat. Sweat chloride level measurement is used to help diagnose CF. High sweat chloride levels are a hallmark of CF and are connected to the way CF works in the body.

Body mass index, which is a measure of someone’s weight in relation to their height.

All participants knew they were taking TRIKAFTA, and no children in the study took placebo.

Each participant's dose of TRIKAFTA granules 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 Results?

For children with CF age 2-5 years, the safety of TRIKAFTA observed in this study (Study 4) was similar to what was observed in people with CF age 12 years and older (Study 1).

What Else Was Found in the Study?

Sweat chloride decreased by 57.9 mmol/L on average through 24 weeks. On average, children started the study with a sweat chloride level of 100.7 mmol/L.

Body mass index (BMI) increased by 0.03 kg/m2 on average at 24 weeks. For example, a child who is 3’2’’ and weighs 38 pounds would gain about an ounce at 24 weeks.

The BMI result is not included in the full Prescribing Information for TRIKAFTA.

The BMI result is not included in the full Prescribing Information for TRIKAFTA.

The BMI result is not included in the full Prescribing Information for TRIKAFTA.

Clayton, age 5
F508del/G542X

Clayton, age 5
F508del/G542X

Clayton, age 5
F508del/G542X

Keep Learning

About TRIKAFTA

About TRIKAFTA

About TRIKAFTA

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

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

 

Your healthcare provider may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past or are experiencing signs or symptoms of liver injury.

Stop taking TRIKAFTA and call your healthcare provider right away if you have any of the following symptoms of liver problems:

  • pain, swelling, or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • mental changes
  • nausea or vomiting
  • dark, amber-colored urine
  • loss of appetite
  • fluid in your stomach area (ascites)
  • pain, swelling, or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • mental changes
  • nausea or vomiting
  • dark, amber-colored urine
  • loss of appetite
  • fluid in your stomach area (ascites)

What should I tell my healthcare provider before taking TRIKAFTA?

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

  • have or have had liver problems
  • are allergic to TRIKAFTA or any ingredients in TRIKAFTA. See the end of the Medication Guide for a complete list of ingredients in TRIKAFTA
  • have kidney problems
  • are pregnant or plan to become pregnant. It is not known if TRIKAFTA will harm your unborn baby. You and your healthcare provider 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 healthcare provider should decide if you will take TRIKAFTA while you are breastfeeding

Tell your healthcare provider 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 healthcare provider or pharmacist for a list of these medicines if you are not sure.

Especially tell your healthcare provider 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®)

Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

What should I avoid while taking TRIKAFTA?

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

What are the possible or reasonably likely side effects of TRIKAFTA?

TRIKAFTA can cause serious side effects, including:

  • See “What is the most important information I should know about TRIKAFTA?”
  • Serious Allergic Reactions can happen 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) has happened in some children and adolescents treated with TRIKAFTA. If you are a child or adolescent, your healthcare provider 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
  • constipation 

Tell your healthcare provider 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 healthcare provider 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 Medication Guide with Important Warning.

Important Safety Information, including Important Warning, and Indication

Información Importante de Seguridad e Indicación

EXPAND

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 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

 

Your healthcare provider may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past or are experiencing signs or symptoms of liver injury.

Stop taking TRIKAFTA and call your healthcare provider right away if you have any of the following symptoms of liver problems:

  • pain, swelling, or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • mental changes
  • nausea or vomiting
  • dark, amber-colored urine
  • loss of appetite
  • fluid in your stomach area (ascites)
  • pain, swelling, or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • mental changes
  • nausea or vomiting
  • dark, amber-colored urine
  • loss of appetite
  • fluid in your stomach area (ascites)

What should I tell my healthcare provider before taking TRIKAFTA?

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

  • have or have had liver problems
  • are allergic to TRIKAFTA or any ingredients in TRIKAFTA. See the end of the Medication Guide for a complete list of ingredients in TRIKAFTA
  • have kidney problems
  • are pregnant or plan to become pregnant. It is not known if TRIKAFTA will harm your unborn baby. You and your healthcare provider 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 healthcare provider should decide if you will take TRIKAFTA while you are breastfeeding

Tell your healthcare provider 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 healthcare provider or pharmacist for a list of these medicines if you are not sure.

Especially tell your healthcare provider 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®)

Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

What should I avoid while taking TRIKAFTA?

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

What are the possible or reasonably likely side effects of TRIKAFTA?

TRIKAFTA can cause serious side effects, including:

  • See “What is the most important information I should know about TRIKAFTA?”
  • Serious Allergic Reactions can happen 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) has happened in some children and adolescents treated with TRIKAFTA. If you are a child or adolescent, your healthcare provider 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
  • constipation 

Tell your healthcare provider 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 healthcare provider 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 Medication Guide with Important Warning.

 

Your healthcare provider may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past or are experiencing signs or symptoms of liver injury.

Stop taking TRIKAFTA and call your healthcare provider right away if you have any of the following symptoms of liver problems:

  • pain, swelling, or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • mental changes
  • nausea or vomiting
  • dark, amber-colored urine
  • loss of appetite
  • fluid in your stomach area (ascites)
  • pain, swelling, or discomfort in the upper right stomach (abdominal) area
  • yellowing of your skin or the white part of your eyes
  • mental changes
  • nausea or vomiting
  • dark, amber-colored urine
  • loss of appetite
  • fluid in your stomach area (ascites)

What should I tell my healthcare provider before taking TRIKAFTA?

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

  • have or have had liver problems
  • are allergic to TRIKAFTA or any ingredients in TRIKAFTA. See the end of the Medication Guide for a complete list of ingredients in TRIKAFTA
  • have kidney problems
  • are pregnant or plan to become pregnant. It is not known if TRIKAFTA will harm your unborn baby. You and your healthcare provider 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 healthcare provider should decide if you will take TRIKAFTA while you are breastfeeding

Tell your healthcare provider 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 healthcare provider or pharmacist for a list of these medicines if you are not sure.

Especially tell your healthcare provider 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®)

Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

What should I avoid while taking TRIKAFTA?

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

What are the possible or reasonably likely side effects of TRIKAFTA?

TRIKAFTA can cause serious side effects, including:

  • See “What is the most important information I should know about TRIKAFTA?”
  • Serious Allergic Reactions can happen 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) has happened in some children and adolescents treated with TRIKAFTA. If you are a child or adolescent, your healthcare provider 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
  • constipation 

Tell your healthcare provider 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 healthcare provider 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 Medication Guide with Important Warning.