KALYDECO® Results

Age 1 to less than 2 years old

 

Study 8

Study 8 was a 24-week safety study of KALYDECO in children less than 2 years old. Nineteen children age 1 to less than 2 years old and 11 children age 6 months to less than 1 year old with cystic fibrosis (CF) were evaluated in this study. All patients enrolled took KALYDECO. No one in the study took placebo. The primary purpose of this study was to determine the safety and tolerability of KALYDECO.

See the results for children age 6 months to less than 1 year old.

 

Mutations eligible to enroll in this study were:

G1244E, G1349D, G178R, G551D, G551S, R117H, S1251N, S1255P, S549N, S549R.

 

Mutations included in this study:

16 children had the G551D mutation, 2 had the S549N mutation, and 1 had the G178R mutation.

 

How KALYDECO was given

Children who weighed ~15 pounds (7 kg) to less than ~31 pounds (14 kg) received 50 mg of KALYDECO oral granules every 12 hours.

Children who weighed ~31 pounds (14 kg) or more received 75 mg of KALYDECO oral granules every 12 hours.

All children took KALYDECO oral granules mixed with 1 teaspoon of age-appropriate soft food or liquid every 12 hours with fat-containing food. All children continued to take their other CF treatments.

Learn how to give your child KALYDECO oral granules.

 

Study results

Safety

The safety of KALYDECO, observed in this study, was similar to what was observed in KALYDECO studies in people with CF age 2 years and older.

See additional safety information.

 

Other study results

Sweat chloride

For children age 1 to less than 2 years old who took KALYDECO:
Sweat chloride decreased on average by -73.5 mmol/L at Week 24.

(average mmol/L beginning of study was 104.1)

Sweat chloride is a measure of the amount of salt in your child's sweat. People with CF have high levels of sweat chloride. A decrease in sweat chloride levels does not mean there will be an improvement in lung function (FEV1).

Because no one in this study took placebo, it is not known if changes in sweat chloride were due to KALYDECO.

 

Study limitations

KALYDECO was approved in people age 1 to less than 2 years old based on the benefits shown in studies of KALYDECO in older people as well as the safety assessment in this study.

Talk to your healthcare provider to learn more about how KALYDECO was approved in this age group.

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