Trial Outcomes & Findings for A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF) (NCT NCT02953314)
NCT ID: NCT02953314
Last Updated: 2020-03-04
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
83 participants
Primary outcome timeframe
Day 1 and Day 14
Results posted on
2020-03-04
Participant Flow
This study consisted of 2-parts (Part A and B). The planned primary analysis was designed to assess overall treatment arm "TEZ/IVA", irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm "TEZ/IVA".
Participant milestones
| Measure |
Part A
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days.
Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
Part B
Participants weighing \<40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg orally in the evening for 24 weeks.
Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
13
|
70
|
|
Overall Study
COMPLETED
|
13
|
67
|
|
Overall Study
NOT COMPLETED
|
0
|
3
|
Reasons for withdrawal
| Measure |
Part A
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days.
Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
Part B
Participants weighing \<40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg orally in the evening for 24 weeks.
Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks.
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
|
Overall Study
Withdrawal of consent (not due to AE)
|
0
|
2
|
Baseline Characteristics
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF)
Baseline characteristics by cohort
| Measure |
Part A
n=13 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
Part B
n=70 Participants
Participants weighing \<40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks.
Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks.
|
Total
n=83 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
13 Participants
n=5 Participants
|
70 Participants
n=7 Participants
|
83 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
41 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
36 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=5 Participants
|
67 Participants
n=7 Participants
|
80 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=5 Participants
|
68 Participants
n=7 Participants
|
80 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1 and Day 14Population: Pharmacokinetic (PK) set included participants who received at least 1 dose of study drug and for whom the primary PK data were considered to be sufficient and interpretable. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points.
Outcome measures
| Measure |
Part A
n=13 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Day 1: TEZ (<25 Kg)
|
6630 nanogram per milliliter (ng/mL)
Geometric Coefficient of Variation 10.3
|
|
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Day 1: TEZ (≥25 Kg)
|
4310 nanogram per milliliter (ng/mL)
Geometric Coefficient of Variation 42.6
|
|
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Day 14: TEZ (<25 Kg)
|
6300 nanogram per milliliter (ng/mL)
Geometric Coefficient of Variation 10.3
|
|
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Day 14: TEZ (≥25 Kg)
|
5340 nanogram per milliliter (ng/mL)
Geometric Coefficient of Variation 49.0
|
|
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Day 1: IVA (<25 Kg)
|
656 nanogram per milliliter (ng/mL)
|
|
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Day 1: IVA (≥25 Kg)
|
1010 nanogram per milliliter (ng/mL)
Geometric Coefficient of Variation 64.3
|
|
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Day 14: IVA (<25 Kg)
|
578 nanogram per milliliter (ng/mL)
Geometric Coefficient of Variation 60.4
|
|
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Day 14: IVA (≥25 Kg)
|
1490 nanogram per milliliter (ng/mL)
Geometric Coefficient of Variation 105
|
PRIMARY outcome
Timeframe: Day 1 and Day 14Population: PK set. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points."Number Analyzed=0" signified no subjects were evaluated for the specified parameter at that time point.
Outcome measures
| Measure |
Part A
n=13 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part A: Area Under the Concentration Versus Time Curve During Dosing Interval (AUCtau) of TEZ and IVA
Day 1: TEZ (<25 Kg)
|
54300 hour*nanogram per milliliter (hr*ng/mL)
Geometric Coefficient of Variation 16.2
|
|
Part A: Area Under the Concentration Versus Time Curve During Dosing Interval (AUCtau) of TEZ and IVA
Day 1: TEZ (≥25 Kg)
|
41600 hour*nanogram per milliliter (hr*ng/mL)
Geometric Coefficient of Variation 36.2
|
|
Part A: Area Under the Concentration Versus Time Curve During Dosing Interval (AUCtau) of TEZ and IVA
Day 14: TEZ (<25 Kg)
|
66500 hour*nanogram per milliliter (hr*ng/mL)
Geometric Coefficient of Variation 30.5
|
|
Part A: Area Under the Concentration Versus Time Curve During Dosing Interval (AUCtau) of TEZ and IVA
Day 14: TEZ (≥25 Kg)
|
71600 hour*nanogram per milliliter (hr*ng/mL)
Geometric Coefficient of Variation 61.1
|
|
Part A: Area Under the Concentration Versus Time Curve During Dosing Interval (AUCtau) of TEZ and IVA
Day 14: IVA (<25 Kg)
|
5050 hour*nanogram per milliliter (hr*ng/mL)
Geometric Coefficient of Variation 49.1
|
|
Part A: Area Under the Concentration Versus Time Curve During Dosing Interval (AUCtau) of TEZ and IVA
Day 14: IVA (≥25 Kg)
|
12400 hour*nanogram per milliliter (hr*ng/mL)
Geometric Coefficient of Variation 118
|
PRIMARY outcome
Timeframe: Day 1 up to Week 28Population: Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with AEs
|
65 participants
|
|
Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
6 participants
|
SECONDARY outcome
Timeframe: Day 1 and Day 14Population: PK set. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points.
Outcome measures
| Measure |
Part A
n=13 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M1-TEZ (<25 kg)
|
1720 ng/mL
Geometric Coefficient of Variation 3.29
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M1-TEZ (≥25 kg)
|
1530 ng/mL
Geometric Coefficient of Variation 22.0
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M1-TEZ (<25 kg)
|
8360 ng/mL
Geometric Coefficient of Variation 22
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M1-TEZ (≥25 kg)
|
5930 ng/mL
Geometric Coefficient of Variation 19.9
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M2-TEZ (<25 kg)
|
1130 ng/mL
Geometric Coefficient of Variation 4.36
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M2-TEZ (≥25 kg)
|
922 ng/mL
Geometric Coefficient of Variation 25.8
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M2-TEZ (<25 kg)
|
6180 ng/mL
Geometric Coefficient of Variation 27.2
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M2-TEZ (≥25 kg)
|
5350 ng/mL
Geometric Coefficient of Variation 27.2
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M1-IVA (<25 kg)
|
2320 ng/mL
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M1-IVA (≥25 kg)
|
2430 ng/mL
Geometric Coefficient of Variation 57.1
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M1-IVA (<25 kg)
|
1460 ng/mL
Geometric Coefficient of Variation 34.6
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M1-IVA (≥25 kg)
|
3420 ng/mL
Geometric Coefficient of Variation 72.7
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M6-IVA (<25 kg)
|
849 ng/mL
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M6-IVA (≥25 kg)
|
1070 ng/mL
Geometric Coefficient of Variation 55.7
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M6-IVA (<25 kg)
|
1090 ng/mL
Geometric Coefficient of Variation 31.4
|
|
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M6-IVA (≥25 kg)
|
2720 ng/mL
Geometric Coefficient of Variation 59.2
|
SECONDARY outcome
Timeframe: Day 1 and Day 14Population: PK set. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. "Number Analyzed=0" signified no subjects were evaluated for the specified parameter at that time point.
Outcome measures
| Measure |
Part A
n=13 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M1-TEZ (<25 kg)
|
36500 hr*ng/mL
Geometric Coefficient of Variation 5.80
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M1-TEZ (≥25 kg)
|
27400 hr*ng/mL
Geometric Coefficient of Variation 26.3
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M1-TEZ (<25 kg)
|
160000 hr*ng/mL
Geometric Coefficient of Variation 15.5
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M1-TEZ (≥25 kg)
|
121000 hr*ng/mL
Geometric Coefficient of Variation 17.1
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M2-TEZ (<25 kg)
|
14200 hr*ng/mL
Geometric Coefficient of Variation 12.3
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 1: M2-TEZ (≥25 kg)
|
11100 hr*ng/mL
Geometric Coefficient of Variation 25.9
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M2-TEZ (<25 kg)
|
137000 hr*ng/mL
Geometric Coefficient of Variation 32.7
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M2-TEZ (≥25 kg)
|
119000 hr*ng/mL
Geometric Coefficient of Variation 27.7
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M1-IVA (<25 kg)
|
13700 hr*ng/mL
Geometric Coefficient of Variation 52.1
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M1-IVA (≥25 kg)
|
30300 hr*ng/mL
Geometric Coefficient of Variation 81.1
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M6-IVA (<25 kg)
|
10200 hr*ng/mL
Geometric Coefficient of Variation 58.5
|
|
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Day 14: M6-IVA (≥25 kg)
|
26000 hr*ng/mL
Geometric Coefficient of Variation 70.6
|
SECONDARY outcome
Timeframe: Day 1 up to Day 28Population: Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm.
Outcome measures
| Measure |
Part A
n=13 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part A: Number of Participants With AEs and SAEs
Participants with AEs
|
12 participants
|
|
Part A: Number of Participants With AEs and SAEs
Participants with SAEs
|
0 participants
|
SECONDARY outcome
Timeframe: Week 16Population: PK set. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure.
Outcome measures
| Measure |
Part A
n=69 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
TEZ (<40 kg)
|
4800 ng/mL
Geometric Coefficient of Variation 33.7
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
TEZ (≥40 kg)
|
5870 ng/mL
Geometric Coefficient of Variation 46.5
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
M1-TEZ (<40 kg)
|
5310 ng/mL
Geometric Coefficient of Variation 36.0
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
M1-TEZ (≥40 kg)
|
5440 ng/mL
Geometric Coefficient of Variation 61.5
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
M2-TEZ (<40 kg)
|
4170 ng/mL
Geometric Coefficient of Variation 47.4
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
M2-TEZ (≥40 kg)
|
5210 ng/mL
Geometric Coefficient of Variation 55.0
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
IVA (<40 kg)
|
725 ng/mL
Geometric Coefficient of Variation 56.9
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
IVA (≥40 kg)
|
886 ng/mL
Geometric Coefficient of Variation 58.7
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
M1-IVA (<40 kg)
|
1560 ng/mL
Geometric Coefficient of Variation 54.8
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
M1-IVA (≥40 kg)
|
1870 ng/mL
Geometric Coefficient of Variation 50.2
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
M6-IVA (<40 kg)
|
870 ng/mL
Geometric Coefficient of Variation 69.2
|
|
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
M6-IVA (≥40 kg)
|
1120 ng/mL
Geometric Coefficient of Variation 29.5
|
SECONDARY outcome
Timeframe: Week 16Population: PK set. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points.
Outcome measures
| Measure |
Part A
n=69 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
M1-TEZ (<40 kg)
|
104000 hr*ng/mL
Geometric Coefficient of Variation 44.2
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
TEZ (<40 kg)
|
50300 hr*ng/mL
Geometric Coefficient of Variation 36.3
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
TEZ (≥40 kg)
|
60900 hr*ng/mL
Geometric Coefficient of Variation 50.6
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
M1-TEZ (≥40 kg)
|
100000 hr*ng/mL
Geometric Coefficient of Variation 87.2
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
M2-TEZ (<40 kg)
|
88400 hr*ng/mL
Geometric Coefficient of Variation 57.0
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
M2-TEZ (≥40 kg)
|
93600 hr*ng/mL
Geometric Coefficient of Variation 46.5
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
IVA (<40 kg)
|
5330 hr*ng/mL
Geometric Coefficient of Variation 62.2
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
IVA (≥40 kg)
|
7410 hr*ng/mL
Geometric Coefficient of Variation 53.8
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
M1-IVA (<40 kg)
|
12700 hr*ng/mL
Geometric Coefficient of Variation 55.9
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
M1-IVA (≥40 kg)
|
17200 hr*ng/mL
Geometric Coefficient of Variation 40.4
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
M6-IVA (<40 kg)
|
8140 hr*ng/mL
Geometric Coefficient of Variation 70.2
|
|
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
M6-IVA (≥40 kg)
|
11100 hr*ng/mL
Geometric Coefficient of Variation 39.4
|
SECONDARY outcome
Timeframe: From Baseline through Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
|
0.9 percentage points
Interval -0.6 to 2.3
|
SECONDARY outcome
Timeframe: From Baseline through Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Relative Change in ppFEV1
|
1.4 percent change
Interval -0.4 to 3.1
|
SECONDARY outcome
Timeframe: From Baseline at Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in Weight
|
1.7 kg
Interval 1.3 to 2.0
|
SECONDARY outcome
Timeframe: From Baseline at Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
z-score is a statistical measure to describe whether a mean was above or below the standard. Weight, adjusted for age and sex, was analyzed as weight-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher weight.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in Weight-for-age Z-Score
|
0.00 z-score
Interval -0.05 to 0.05
|
SECONDARY outcome
Timeframe: From Baseline at Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in Height
|
2.7 centimeter (cm)
Interval 2.4 to 2.9
|
SECONDARY outcome
Timeframe: From Baseline at Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
z-score is a statistical measure to describe whether a mean was above or below the standard. Height, adjusted for age and sex, was analyzed as height-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher height.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in Height-for-age z-Score
|
0.00 z-score
Interval -0.05 to 0.05
|
SECONDARY outcome
Timeframe: From Baseline at Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
BMI was defined as weight in kg divided by height in square meter (m\^2).
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in Body Mass Index (BMI)
|
0.23 kg/m^2
Interval 0.06 to 0.4
|
SECONDARY outcome
Timeframe: From Baseline at Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
BMI was defined as weight in kg divided by height in m\^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in BMI-for-age z-Score
|
-0.03 z-score
Interval -0.1 to 0.04
|
SECONDARY outcome
Timeframe: From Baseline through Week 4Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
Sweat samples were collected using an approved collection device.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in Sweat Chloride
|
-13.0 millimole per liter (mmol/L)
Interval -16.2 to -9.9
|
SECONDARY outcome
Timeframe: From Baseline through Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
Sweat samples were collected using an approved collection device.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in Sweat Chloride
|
-14.5 mmol/L
Interval -17.4 to -11.6
|
SECONDARY outcome
Timeframe: From Baseline through Week 24Population: FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen.
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Outcome measures
| Measure |
Part A
n=70 Participants
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
|---|---|
|
Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
|
3.4 units on a scale
Interval 1.4 to 5.5
|
Adverse Events
Part A
Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths
Part B
Serious events: 6 serious events
Other events: 62 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part A
n=13 participants at risk
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
Part B
n=70 participants at risk
Participants weighing \<40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg orally in the evening for 24 weeks.
Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
1.4%
1/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Gastrointestinal disorders
Breath odour
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
1.4%
1/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
2.9%
2/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Sinusitis
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
1.4%
1/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
1.4%
1/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Snoring
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
1.4%
1/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
Other adverse events
| Measure |
Part A
n=13 participants at risk
Participants weighing \<25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days.
|
Part B
n=70 participants at risk
Participants weighing \<40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg orally in the evening for 24 weeks.
Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
14.3%
10/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
7.1%
5/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
10.0%
7/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Gastrointestinal disorders
Constipation
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
2.9%
2/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Nasopharyngitis
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
8.6%
6/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
21.4%
15/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Influenza
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
7.1%
5/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
8.6%
6/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Acute sinusitis
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
0.00%
0/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Ear infection
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
7.1%
5/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Urinary tract infection
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
1.4%
1/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
5.7%
4/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
23.1%
3/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
35.7%
25/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
8.6%
6/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
4.3%
3/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
15.4%
2/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
14.3%
10/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
8.6%
6/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
10.0%
7/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
General disorders
Pyrexia
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
18.6%
13/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
8.6%
6/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Investigations
Activated partial thromboplastin time prolonged
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
0.00%
0/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Investigations
International normalised ratio increased
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
0.00%
0/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Investigations
Prothrombin time prolonged
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
0.00%
0/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
2.9%
2/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
1.4%
1/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
2.9%
2/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Nervous system disorders
Headache
|
23.1%
3/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
8.6%
6/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Psychiatric disorders
Abnormal behaviour
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
0.00%
0/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
0.00%
0/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
|
Vascular disorders
Pallor
|
7.7%
1/13 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
0.00%
0/70 • Part A: Day 1 up to Day 28; Part B: Day 1 up to Week 28
Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm for Part A and Part B.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER