KALYDECO® Results

Age 6 months to less than 1 year old

Study 8

The safety and tolerability of KALYDECO were evaluated in 36 children age 4 months to less than 2 years old with cystic fibrosis (CF).
All the children in this study took KALYDECO.

One part of the study evaluated 11 children age 6 months to less than 1 year old.

See the results for children age 4 months to less than 6 months old and 1 year to less than 2 years old.

 

Mutations eligible to enroll in this study were:

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

 

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.

In the clinical study, instruction was provided to give the dose of KALYDECO oral granules mixed into 1 teaspoon of an age-appropriate soft food or liquid, either orally with a syringe or with a spoon, every 12 hours along with a fat-containing food. 

Direction was given to ensure the full contents of the syringe were administered.

KALYDECO granules should not be given with a bottle.

Please note: A 25-mg dose is available for children who weigh 11 pounds (5 kg) to less than 15 pounds (7 kg).

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 6 months to less than 1 year old who took KALYDECO:
Sweat chloride decreased on average by -58.6 mmol/L at Week 24.

Sweat chloride is the amount of salt in your child’s sweat. 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 6 months to less than 1 year old based on the efficacy 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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