Study 6

Study 6 (In children with CF age 2 to less than 6 years)

In a 24-week, open-label, clinical study, KALYDECO® oral granules were given to 34 children with CF who were 2 to less than 6 years of age. Patients with the following mutations were eligible for this study: G551D, G1244E, G1349D, G178R, G551S, G970R*, S1251N, S1255P, S549N, or S549R. Of 34 patients enrolled, 32 had the G551D mutation and 2 had the S549N mutation.

*KALYDECO is not indicated for people with CF who have the G970R mutation.

Study 6 was a 24-week study in which all children took KALYDECO. Safety and tolerability was the main endpoint, or measure, of the pediatric clinical study.

Children who weighed 31 lbs (14 kg) or less received KALYDECO oral granules (50 mg) mixed in 1 teaspoon (5 mL) of soft food or liquid every 12 hours with fat-containing food.

Children who weighed more than 31 lbs (14 kg) received KALYDECO oral granules (75 mg) mixed in 1 teaspoon (5 mL) of soft food or liquid every 12 hours with fat-containing food.

Safety Results

The type and frequency of side effects for children age 2 to less than 6 years who participated in the 24-week study (Study 6) were similar to those seen in other clinical studies with patients 6 years and older.

High liver enzymes were more common in children who had abnormal liver enzymes before starting KALYDECO.

Learn about safety information for KALYDECO in people 6 years and older.

Select Safety Information

  • KALYDECO can cause serious side effects
  • High liver enzymes in the blood have been reported in patients receiving KALYDECO
    • The doctor will do blood tests to check the liver before starting KALYDECO, every 3 months during the first year of taking KALYDECO, and every year while taking KALYDECO
    • For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often
    • Call the doctor right away if the patient has any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of his/her eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine
  • Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving KALYDECO. The doctor should perform eye examinations prior to and during treatment with KALYDECO to look for cataracts
  • The most common side effects include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness

Tell the doctor if the patient has any side effect that bothers him/her or that does not go away. These are not all the possible side effects of KALYDECO. For more information, ask the doctor or pharmacist.

If you have any questions about KALYDECO, talk to your child's healthcare provider.

Other results

Sweat chloride results

A decrease in sweat chloride levels does not always mean there will be an improvement in lung function (FEV1).

What is KALYDECO® (ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 12 months and older who have at least one mutation in their CF gene that is responsive to KALYDECO.

Talk to your doctor to learn if you have an indicated CF gene mutation.

It is not known if KALYDECO is safe and effective in children under 12 months of age.

Important Safety Information

Who should not take KALYDECO?

Do not take KALYDECO if you take certain medicines or herbal supplements, such as:

  • the antibiotics rifampin (Rifamate®, Rifater®) or rifabutin (Mycobutin®)
  • seizure medicines such as: phenobarbital, carbamazepine (Tegretol®, Carbatrol®, and Equetro®), or phenytoin (Dilantin®, Phenytek®)
  • St. John’s wort (Hypericum perforatum)

Talk to your doctor before taking KALYDECO if you take any of the medicines or supplements listed above.

Important Safety Information

Who should not take KALYDECO?

Do not take KALYDECO if you take certain medicines or herbal supplements, such as:

  • the antibiotics rifampin (Rifamate®, Rifater®) or rifabutin (Mycobutin®)
  • seizure medicines such as phenobarbital, carbamazepine (Tegretol®, Carbatrol®, and Equetro®), or phenytoin (Dilantin®, Phenytek®)
  • St. John’s wort (Hypericum perforatum)

Talk to your doctor before taking KALYDECO if you take any of the medicines or supplements listed above.

What should I tell my doctor before taking KALYDECO?

Before you take KALYDECO, tell your doctor if you:

  • have liver or kidney problems
  • drink grapefruit juice, or eat grapefruit or Seville oranges
  • are pregnant or plan to become pregnant. It is not known if KALYDECO will harm your unborn baby. You and your doctor should decide if you will take KALYDECO while you are pregnant
  • are breastfeeding or planning to breastfeed. It is not known if KALYDECO passes into your breast milk. You and your doctor should decide if you will take KALYDECO while you are breastfeeding

KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works.

Tell your doctor about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements, as the dose of KALYDECO may need to be adjusted when taken with certain medications.

Especially tell your doctor if you take:

  • antifungal medications such as ketoconazole (e.g., Nizoral®), itraconazole (e.g., Sporanox®), posaconazole (e.g., Noxafil®), voriconazole (e.g., Vfend®), or fluconazole (e.g., Diflucan®)
  • antibiotics such as telithromycin (e.g., Ketek®), clarithromycin (e.g., Biaxin®), or erythromycin (e.g., Ery-Tab®)

What should I avoid while taking KALYDECO?

  • KALYDECO can cause dizziness in some people who take it. Do not drive a car, use machinery, or do anything that needs you to be alert until you know how KALYDECO affects you
  • You should avoid food containing grapefruit or Seville oranges while you are taking KALYDECO

What are the possible side effects of KALYDECO?

KALYDECO can cause serious side effects.

High liver enzymes in the blood have been reported in patients receiving KALYDECO. Your doctor will do blood tests to check your liver:

  • before you start KALYDECO
  • every 3 months during your first year of taking KALYDECO
  • every year while you are taking KALYDECO

For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often.

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

Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving KALYDECO. Your doctor should perform eye examinations prior to and during treatment with KALYDECO to look for cataracts.

The most common side effects include:

  • headache
  • upper respiratory tract infection (common cold), including sore throat, nasal or sinus congestion, runny nose
  • stomach (abdominal) pain
  • diarrhea
  • rash
  • nausea
  • dizziness

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 KALYDECO. For more information, ask your doctor or pharmacist.

Call your doctor for medical advice about side effects. You are encouraged to report side effects to FDA at 1-800-FDA-1088.

Click here for KALYDECO full Prescribing Information, including Patient Information.