Study 6 was a 24-week study in which the safety of KALYDECO was evaluated in 34 children age 2 to less than 6 years old with cystic fibrosis (CF). All children in this study took KALYDECO. No one in this study took placebo. The primary purpose of this study was to determine the safety and tolerability of KALYDECO.
Mutations eligible to enroll in this study were:
G1244E, G1349D, G178R, G551D, G551S, G970R,* S1251N, S1255P, S549N, S549R.
*KALYDECO is not indicated for people with CF who have the G970R mutation.
Mutations included in this study:
32 children had the G551D mutation and 2 had the S549N mutation.
How KALYDECO was given
Other study results
KALYDECO was approved in people age 2 to less than 6 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.