Keep in mind, results shown are an average for all people studied and differed among individuals and mutations. You or your child may have a different experience.
Children age 6
through 11 years
People with
F508del/F508del
People with other
responsive mutations
Study details
In a safety study of 70 children with cystic fibrosis (CF), the safety of SYMDEKO was evaluated. Participants were age 6 through 11 years with 2 copies of the F508del mutation or 1 copy of the F508del mutation and a second mutation in the CF gene predicted to respond to SYMDEKO.
- Each child took SYMDEKO every 12 hours with fat-containing food for 24 weeks (~6 months)
- Participants continued to take their other prescribed CF therapies
- All patients knew they were taking SYMDEKO, and no children in the study took placebo
Safety study results
Safety through 24 weeks
The safety of SYMDEKO observed in the study was similar to what was observed in people age 12 years and older.
Please see Important Safety Information.
View Side Effects and Additional Safety Information in people age 12 years and older.
Safety study considerations
- Because no one took placebo in the safety study, it is not known if the changes in lung function and sweat chloride levels were due to SYMDEKO
- Changes in sweat chloride levels observed in the study are not related to changes in lung function
- Keep in mind, all results shown are an average of all people studied and differed among individuals and mutations. Your child may have a different experience
Additional safety study results
Sweat chloride levels through 24 weeks
Decreased sweat chloride levels by 14.5 mmol/L on average
Sweat chloride is a measure of the amount of salt in a child’s sweat (mmol/L). People with CF have high levels of sweat chloride.
mmol/L
Lung function (FEV1)* through 24 weeks
Lung function changed by 0.9 percentage points on average
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. Lung function was part of the safety assessment in this trial.
POINT CHANGE
*FEV1=percent predicted forced expiratory volume in 1 second.
Study details
In a clinical study of 504 people with cystic fibrosis (CF), SYMDEKO and placebo were compared to determine the possible benefits and risks of SYMDEKO. Participants were age 12 years and older with 2 copies of the F508del mutation in the CF gene.
- 248 people took SYMDEKO and 256 took placebo every 12 hours with fat-containing food for 24 weeks (~6 months)
- Participants continued to take their other prescribed CF therapies
SYMDEKO improved lung function and reduced pulmonary exacerbations
Lung function (FEV1)* through 24 weeks
Improved lung function by 4 percentage points on average compared with those who took placebo.
IMPROVEMENT
VS PLACEBO
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.
Keep in mind, results shown are an average for all people studied and differed among individuals. You or your child may have a different experience.
Pulmonary exacerbations through 24 weeks
Lowered the risk of having a pulmonary exacerbation by 35% on average compared with those who took placebo.
There were 78 pulmonary exacerbations in the SYMDEKO group and 122 in the placebo group.
Pulmonary exacerbations are defined as certain symptoms that require treatment with new oral, intravenous (IV), or inhaled antibiotics.
Additional clinical study results
Body Mass Index (BMI) at 24 weeks
Increased by 0.06 kg/m2 on average compared with those who took placebo.
- For example, a person who is 5’4” and 110 pounds would gain 0.35 pounds, or a person who is 5’10” and 160 pounds would gain 0.42 pounds
- It cannot be determined if this change was due to SYMDEKO
- BMI is a measure of someone’s weight in relation to his or her height
CF respiratory symptoms through 24 weeks
5.1 point change on average in certain CF respiratory symptoms compared with those who took placebo
- It cannot be determined if this change was due to SYMDEKO
A tool† was used to measure CF respiratory symptoms including cough, difficulty breathing, and amount of mucus coughed up.
*FEV1=percent predicted forced expiratory volume in 1 second.
†Respiratory symptoms were measured using a tool called the Cystic Fibrosis Questionnaire-Revised Respiratory Domain score.
Study details
In a clinical study of 244 people with cystic fibrosis (CF), SYMDEKO was compared with ivacaftor and placebo to determine the possible benefits and risks of SYMDEKO. Participants were age 12 years and older with the F508del mutation and a second mutation in the CF gene predicted to respond to SYMDEKO.
Mutations included in the clinical study
| 711+3AG c.579+3A>G |
A455E c.1364C>A |
E831X c.2491G>T |
R347H c.1040G>A |
S977F c.2930C>T |
| 2789+5GA c.2657+5G>A |
D110H c.328G>C |
L206W c.617T>G |
R352Q c.1055G>A |
|
| 3272-26AG c.3140-26A>G |
D579G c.1736A>G |
P67L c.200C>T |
R1070W c.3208C>T |
|
| 3849+10kbCT c.3718-2477C>T |
D1152H c.3454G>C |
R117C c.349C>T |
S945L c.2834C>T |
| 711+3AG c.579+3A>G |
L206W c.617T>G |
| 2789+5GA c.2657+5G>A |
P67L c.200C>T |
| 3272-26AG c.3140-26A>G |
R117C c.349C>T |
| 3849+10kbCT c.3718-2477C>T |
R347H c.1040G>A |
| A455E c.1364C>A |
R352Q c.1055G>A |
| D110H c.328G>C |
R1070W c.3208C>T |
| D579G c.1736A>G |
S945L c.2834C>T |
| D1152H c.3454G>C |
S977F c.2930C>T |
| E831X c.2491G>T |
View all mutations that are responsive to SYMDEKO.
- Each person had two 8-week treatment periods where they took either SYMDEKO, ivacaftor, or placebo every 12 hours with fat-containing food. Participants received 2 of the 3 possible treatments after completing both treatment periods
- 161 patients took SYMDEKO, 156 patients took ivacaftor, and 161 patients took placebo
- Between treatment periods, there was an 8-week washout period where they did not take SYMDEKO, ivacaftor, or placebo
- Participants continued to take their other prescribed CF therapies
SYMDEKO improved lung function
Lung function (FEV1)* average of Weeks 4 and 8
Improved lung function overall by an average of 6.8 percentage points compared with placebo. Additionally, improved lung function by an average of 2.1 percentage points compared with ivacaftor.
IMPROVEMENT
VS PLACEBO
IMPROVEMENT
VS IVACAFTOR
Changes in lung function varied by mutations (range -1.0 to 10.1 percentage points compared with placebo) and individuals.
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.
Keep in mind, all results shown are an average of all people studied and differed among individuals and mutations. You or your child may have a different experience.
Additional clinical study results
CF respiratory symptoms average of Weeks 4 and 8
11.1 point overall improvement on average in CF respiratory symptoms compared with placebo
1.4 point change on average in CF respiratory symptoms compared with ivacaftor
- It cannot be determined whether this change was due to SYMDEKO
Changes in CF respiratory symptoms varied by mutations (range -11.1 to 29.2 points compared with placebo).
A tool† was used to measure CF respiratory symptoms including cough, difficulty breathing, and amount of mucus coughed up.
