KALYDECO® Results

Age 6 years and older

 

Study 2

Study 2 was a 48-week study in which 52 people age 6 to less than 12 years old with a G551D mutation took KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours. Results were measured at different time points, including 24 and 48 weeks. All people took their other cystic fibrosis (CF) treatments except for hypertonic saline through the entire length of the study (48 weeks). The study evaluated lung function (FEV1). Other results studied were CF respiratory symptoms, weight, and sweat chloride. The study also evaluated the safety of KALYDECO.

 

Mutation eligible to enroll and included in this study was:

G551D.

 

Study results

Lung function (FEV1)

Lung function increased
12.5 percentage points greater average improvement, compared to placebo through 24 weeks.
10.0 percentage points greater average improvement, compared to placebo through 48 weeks.

Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second. 

 

Other study results

CF respiratory symptoms

No difference was seen in respiratory symptoms compared to placebo through Week 48.

Respiratory symptoms are measured by the CFQ-R Respiratory Domain score, which is a tool used to measure respiratory symptoms, including coughing, mucus, and trouble breathing.

Weight

Weight increased

About 6 pounds (2.8 kg) on average, compared to placebo at Week 48.

Sweat chloride

After taking KALYDECO:

Sweat chloride decreased on average by -53 mmol/L compared to placebo at Week 48.

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). 

 

KALYDECO has also been studied in people age 12 and older who have the G551D mutation. Be sure to review the information on this study in the Age 12 years and older section of this website.

 

 

Study 4

In Study 4, 39 people age 6 and older who had an eligible CF mutation (listed below) took KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours. All people took all their other CF treatments except for hypertonic saline. Each person had an 8-week treatment period with KALYDECO and an 8-week treatment period with placebo. The study evaluated lung function (FEV1). Other results studied were CF respiratory symptoms, body mass index (BMI), and sweat chloride. The study also evaluated the safety of KALYDECO.

 

Mutations eligible to enroll and included in this study were:

G1244E, G1349D, G178R, G551S, G970R,* S1251N, S1255P, S549N, S549R.

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

Important considerations
  • Results for the mutations studied varied by mutation. Not all mutations showed the same level of benefit when taking KALYDECO
  • Some people experienced less improvement compared to the average for all the people in the study. Others experienced more improvement
  • Talk to your healthcare provider to learn more about your specific mutation

 

Study results

Lung function (FEV1)

Lung function increased

In the overall population for the 9 mutations studied, people had a 10.7 percentage points average improvement in lung function compared to placebo, from the start of the study through Week 8.

Results varied by mutation.

Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second. 

 

Other study results

CF respiratory symptoms

CF respiratory symptoms improved

In the overall population for the 9 mutations studied, people had a 9.6-point average improvement in CF respiratory symptoms compared to placebo, from the start of the study through Week 8.

Results varied by mutation.

Respiratory symptoms are measured by the CFQ-R Respiratory Domain score, which is a tool used to measure respiratory symptoms, including coughing, mucus, and trouble breathing.

Body mass index (BMI)

BMI increased

In the overall population for the 9 mutations studied, people had a 0.66 kg/m2 on average increase in BMI compared to placebo at Week 8.

Results varied by mutation.

BMI is a measure of someone’s weight in relation to his or her height. For example, for a 5'4", 110-lb adult, a 0.66 kg/m2 increase in BMI would be a gain of about 4 lb; for a 4'6", 65-lb child, this would be a gain of about 3 lb.

Sweat chloride

Sweat chloride decreased

In the overall population for the 9 mutations studied, people had a -50 mmol/L average decrease in sweat chloride compared to placebo from the start of the study through Week 8.

Results varied by mutation.

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). 

 

Study 5

Study 5 was a 24-week study in which 69 people age 6 and older with an R117H mutation took either KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours, along with their other CF treatments. The study evaluated lung function (FEV1). Other results studied were sweat chloride, CF respiratory symptoms, body mass index (BMI), and pulmonary exacerbations. The study also evaluated the safety of KALYDECO.

 

Mutation eligible to enroll and included in this study was:

R117H.

 

What was studied

Lung function (FEV1)

Lung function (FEV1) was the main measure of this study 

Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second. 

Other measures studied

Sweat chloride
Sweat chloride is a measure of the amount of salt in your sweat. People with CF have high levels of sweat chloride.

Sweat chloride
Sweat chloride is a measure of the amount of salt in your sweat. People with CF have high levels of sweat chloride.

Body mass index (BMI)
BMI is a measure of someone’s weight in relation to his or her height.

Body mass index (BMI)
BMI is a measure of someone’s weight in relation to his or her height.

Safety
Safety is the evaluation of all side effects that occur during a study.

Safety
Safety is the evaluation of all side effects that occur during a study.

CF respiratory symptoms
Respiratory symptoms are measured by the CFQ-R Respiratory Domain score, which is a tool used to measure respiratory symptoms, including coughing, mucus, and trouble breathing.

CF respiratory symptoms
Respiratory symptoms are measured by the CFQ-R Respiratory Domain score, which is a tool used to measure respiratory symptoms, including coughing, mucus, and trouble breathing.

Pulmonary exacerbations
Pulmonary exacerbations are defined as changes in certain symptoms requiring changes in the use of oral, IV, or inhaled antibiotics.

Pulmonary exacerbations
Pulmonary exacerbations are defined as changes in certain symptoms requiring changes in the use of oral, IV, or inhaled antibiotics.

 

Some people in this study showed improvement in these measures, but others did not.

  • Please talk to your healthcare provider if you’d like to learn about the results of this study
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