Trial Outcomes & Findings for Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del (NCT NCT03601637)
NCT ID: NCT03601637
Last Updated: 2023-01-06
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
61 participants
Primary outcome timeframe
Day 1 and Day 15
Results posted on
2023-01-06
Participant Flow
This study was conducted in participants with cystic fibrosis (CF) aged 1 through less than 2 years of age who are homozygous for F508del. A total of 61 participants were enrolled in Parts A and B of the study. One participant in Part B was enrolled but not dosed in this study. Therefore, data for 60 participants are reported in the participant flow and baseline characteristics sections.
Participant milestones
| Measure |
Part A: LUM/IVA
Participants weighing 7 to less than( \<)10 kilograms (kg) at screening received lumacaftor (LUM) 75 milligrams (mg)/ ivacaftor (IVA) 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to \<14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
|
Part B: LUM/IVA
Participants weighing 7 to \<9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
|
|---|---|---|
|
Overall Study
STARTED
|
14
|
46
|
|
Overall Study
COMPLETED
|
13
|
43
|
|
Overall Study
NOT COMPLETED
|
1
|
3
|
Reasons for withdrawal
| Measure |
Part A: LUM/IVA
Participants weighing 7 to less than( \<)10 kilograms (kg) at screening received lumacaftor (LUM) 75 milligrams (mg)/ ivacaftor (IVA) 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to \<14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
|
Part B: LUM/IVA
Participants weighing 7 to \<9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
|
|---|---|---|
|
Overall Study
Withdrawal of Consent (not due to adverse event)
|
0
|
1
|
|
Overall Study
Adverse Event
|
1
|
1
|
|
Overall Study
Other
|
0
|
1
|
Baseline Characteristics
Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
Baseline characteristics by cohort
| Measure |
Part A: LUM/IVA
n=14 Participants
Participants weighing 7 to less than(\<)10 kilograms (kg) at screening received lumacaftor (LUM) 75 milligrams (mg)/ ivacaftor (IVA) 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to \<14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
|
Part B: LUM/IVA
n=46 Participants
Participants weighing 7 to \<9 kg at screening received LUM 75 mg/ IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
|
Total
n=60 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
14 Participants
n=5 Participants
|
46 Participants
n=7 Participants
|
60 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
|
24 Participants
n=7 Participants
|
31 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
50 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
14 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
48 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not collected per local regulations
|
0 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1 and Day 15Population: Pharmacokinetic (PK) set included participants who received at least 1 dose of study drug. Here the "Number Analyzed" signifies those participants who were evaluable at specified time point.
Outcome measures
| Measure |
Part A: LUM 75 mg/IVA 94 mg
n=7 Participants
Participants received LUM 75 mg /IVA 94 mg dose every 12 hours for 15 days.
|
Part A: LUM 100 mg/IVA 125 mg
n=7 Participants
Participants received LUM 100 mg/IVA 125 mg dose 2 every 12 hours for 15 days.
|
Part B: LUM 150 mg/IVA 188 mg
Participants received LUM 150 mg/IVA 188 mg dose every 12 hours for 24 weeks.
|
|---|---|---|---|
|
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Day 1: LUM
|
14600 nanograms per milliliter (ng/mL)
Standard Deviation 5560
|
12600 nanograms per milliliter (ng/mL)
Standard Deviation 7190
|
—
|
|
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Day 15: LUM
|
16600 nanograms per milliliter (ng/mL)
Standard Deviation 9590
|
13900 nanograms per milliliter (ng/mL)
Standard Deviation 5800
|
—
|
|
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Day 1: IVA
|
1620 nanograms per milliliter (ng/mL)
Standard Deviation 648
|
1320 nanograms per milliliter (ng/mL)
Standard Deviation 804
|
—
|
|
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Day 15: IVA
|
718 nanograms per milliliter (ng/mL)
Standard Deviation 352
|
496 nanograms per milliliter (ng/mL)
Standard Deviation 268
|
—
|
PRIMARY outcome
Timeframe: Pre-dose at Day 8 and Day 15Population: PK set included participants who received at least 1 dose of study drug. Here the "Number Analyzed" signifies those participants who were evaluable at specified time point.
Outcome measures
| Measure |
Part A: LUM 75 mg/IVA 94 mg
n=7 Participants
Participants received LUM 75 mg /IVA 94 mg dose every 12 hours for 15 days.
|
Part A: LUM 100 mg/IVA 125 mg
n=7 Participants
Participants received LUM 100 mg/IVA 125 mg dose 2 every 12 hours for 15 days.
|
Part B: LUM 150 mg/IVA 188 mg
Participants received LUM 150 mg/IVA 188 mg dose every 12 hours for 24 weeks.
|
|---|---|---|---|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
Day 8: LUM
|
12000 ng/mL
Standard Deviation 8880
|
12800 ng/mL
Standard Deviation 3900
|
—
|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
Day 15: LUM
|
8380 ng/mL
Standard Deviation 7790
|
10500 ng/mL
Standard Deviation 3070
|
—
|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
Day 8: IVA
|
169 ng/mL
Standard Deviation 75.5
|
185 ng/mL
Standard Deviation 101
|
—
|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
Day 15: IVA
|
78.9 ng/mL
Standard Deviation 19.1
|
120 ng/mL
Standard Deviation 60.5
|
—
|
PRIMARY outcome
Timeframe: From Day 1 up to Week 26Population: Safety set included all participants who received at least 1 dose of study drug in the treatment period. The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single Part B: LUM/IVA arm.
Outcome measures
| Measure |
Part A: LUM 75 mg/IVA 94 mg
n=46 Participants
Participants received LUM 75 mg /IVA 94 mg dose every 12 hours for 15 days.
|
Part A: LUM 100 mg/IVA 125 mg
Participants received LUM 100 mg/IVA 125 mg dose 2 every 12 hours for 15 days.
|
Part B: LUM 150 mg/IVA 188 mg
Participants received LUM 150 mg/IVA 188 mg dose every 12 hours for 24 weeks.
|
|---|---|---|---|
|
Part B : Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Participants with TEAEs
|
44 participants
|
—
|
—
|
|
Part B : Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
5 participants
|
—
|
—
|
SECONDARY outcome
Timeframe: From Day 1 up to Day 25Population: Safety set included all participants who received at least 1 dose of study drug in the treatment period. The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single Part A: LUM/IVA arm.
Outcome measures
| Measure |
Part A: LUM 75 mg/IVA 94 mg
n=14 Participants
Participants received LUM 75 mg /IVA 94 mg dose every 12 hours for 15 days.
|
Part A: LUM 100 mg/IVA 125 mg
Participants received LUM 100 mg/IVA 125 mg dose 2 every 12 hours for 15 days.
|
Part B: LUM 150 mg/IVA 188 mg
Participants received LUM 150 mg/IVA 188 mg dose every 12 hours for 24 weeks.
|
|---|---|---|---|
|
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Participants with TEAEs
|
12 participants
|
—
|
—
|
|
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
0 participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Pre-dose at Day 8 and Day 15Population: PK set included participants who received at least 1 dose of study drug. Here the "Number Analyzed signifies those participants who were evaluable at specified time point.
Outcome measures
| Measure |
Part A: LUM 75 mg/IVA 94 mg
n=7 Participants
Participants received LUM 75 mg /IVA 94 mg dose every 12 hours for 15 days.
|
Part A: LUM 100 mg/IVA 125 mg
n=7 Participants
Participants received LUM 100 mg/IVA 125 mg dose 2 every 12 hours for 15 days.
|
Part B: LUM 150 mg/IVA 188 mg
Participants received LUM 150 mg/IVA 188 mg dose every 12 hours for 24 weeks.
|
|---|---|---|---|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 8: M28-LUM
|
1410 ng/mL
Standard Deviation 791
|
1470 ng/mL
Standard Deviation 365
|
—
|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 15: M28-LUM
|
1460 ng/mL
Standard Deviation 1060
|
1370 ng/mL
Standard Deviation 468
|
—
|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 8: M1-IVA
|
606 ng/mL
Standard Deviation 298
|
842 ng/mL
Standard Deviation 527
|
—
|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 15: M1-IVA
|
284 ng/mL
Standard Deviation 70.2
|
604 ng/mL
Standard Deviation 394
|
—
|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 8: M6-IVA
|
2380 ng/mL
Standard Deviation 843
|
3290 ng/mL
Standard Deviation 1830
|
—
|
|
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 15: M6-IVA
|
1460 ng/mL
Standard Deviation 763
|
2800 ng/mL
Standard Deviation 2300
|
—
|
SECONDARY outcome
Timeframe: From Baseline at Week 24Population: The Full Analysis Set (FAS) included all enrolled participants who were exposed to any amount of study drug in Part B.The pharmacodynamic analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single Part B: LUM/IVA arm.
Outcome measures
| Measure |
Part A: LUM 75 mg/IVA 94 mg
n=46 Participants
Participants received LUM 75 mg /IVA 94 mg dose every 12 hours for 15 days.
|
Part A: LUM 100 mg/IVA 125 mg
Participants received LUM 100 mg/IVA 125 mg dose 2 every 12 hours for 15 days.
|
Part B: LUM 150 mg/IVA 188 mg
Participants received LUM 150 mg/IVA 188 mg dose every 12 hours for 24 weeks.
|
|---|---|---|---|
|
Part B: Absolute Change in Sweat Chloride
|
-29.1 Millimole per liter (mmol/L)
Standard Deviation 13.5
|
—
|
—
|
SECONDARY outcome
Timeframe: Pre-dose at Day 15, Week 4, Week 12 and Week 24Population: PK set included participants who received at least 1 dose of study drug. Here the "Number Analyzed" signifies those participants who were evaluable at specified time point.
Outcome measures
| Measure |
Part A: LUM 75 mg/IVA 94 mg
n=1 Participants
Participants received LUM 75 mg /IVA 94 mg dose every 12 hours for 15 days.
|
Part A: LUM 100 mg/IVA 125 mg
n=44 Participants
Participants received LUM 100 mg/IVA 125 mg dose 2 every 12 hours for 15 days.
|
Part B: LUM 150 mg/IVA 188 mg
n=1 Participants
Participants received LUM 150 mg/IVA 188 mg dose every 12 hours for 24 weeks.
|
|---|---|---|---|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 15: LUM
|
13500 ng/mL
|
9030 ng/mL
Standard Deviation 4390
|
887 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 4: LUM
|
21900 ng/mL
|
9370 ng/mL
Standard Deviation 5030
|
1000 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 12: LUM
|
12100 ng/mL
|
9160 ng/mL
Standard Deviation 4400
|
566 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 24: LUM
|
11200 ng/mL
|
8930 ng/mL
Standard Deviation 5310
|
288 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 15: M28-LUM
|
1460 ng/mL
|
1260 ng/mL
Standard Deviation 528
|
786 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 4: M28-LUM
|
1570 ng/mL
|
1340 ng/mL
Standard Deviation 579
|
602 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 12: M28-LUM
|
1740 ng/mL
|
1440 ng/mL
Standard Deviation 599
|
501 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 24: M28-LUM
|
1870 ng/mL
|
1470 ng/mL
Standard Deviation 548
|
569 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 15: IVA
|
213 ng/mL
|
112 ng/mL
Standard Deviation 68.3
|
3.68 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 4: IVA
|
267 ng/mL
|
112 ng/mL
Standard Deviation 82.9
|
11.4 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 12: IVA
|
286 ng/mL
|
91.7 ng/mL
Standard Deviation 44.7
|
2.18 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 24: IVA
|
37.1 ng/mL
|
144 ng/mL
Standard Deviation 296
|
2.86 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 15: M1-IVA
|
1420 ng/mL
|
437 ng/mL
Standard Deviation 291
|
18.1 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 4: M1-IVA
|
1930 ng/mL
|
466 ng/mL
Standard Deviation 359
|
58.0 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 12: M1-IVA
|
759 ng/mL
|
418 ng/mL
Standard Deviation 236
|
18.3 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 24: M1-IVA
|
256 ng/mL
|
499 ng/mL
Standard Deviation 613
|
18.5 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Day 15: M6-IVA
|
4740 ng/mL
|
1930 ng/mL
Standard Deviation 1460
|
43.9 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 4: M6-IVA
|
9090 ng/mL
|
1970 ng/mL
Standard Deviation 1490
|
131 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 12: M6-IVA
|
3520 ng/mL
|
1950 ng/mL
Standard Deviation 1430
|
46.5 ng/mL
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Week 24: M6-IVA
|
1320 ng/mL
|
1740 ng/mL
Standard Deviation 1130
|
63.3 ng/mL
|
Adverse Events
Part A: LUM/IVA
Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths
Part B: LUM/IVA
Serious events: 5 serious events
Other events: 42 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part A: LUM/IVA
n=14 participants at risk
Participants weighing 7 to \<10 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 10 to \<14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
|
Part B: LUM/IVA
n=46 participants at risk
Participants weighing 7 to \<9 kg at screening received LUM 75 mg/ IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg FDC q12h for 24 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Post procedural fever
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
2.2%
1/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Gastrointestinal disorders
Distal intestinal obstruction syndrome
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
2.2%
1/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
6.5%
3/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
Other adverse events
| Measure |
Part A: LUM/IVA
n=14 participants at risk
Participants weighing 7 to \<10 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 10 to \<14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
|
Part B: LUM/IVA
n=46 participants at risk
Participants weighing 7 to \<9 kg at screening received LUM 75 mg/ IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg FDC q12h for 24 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Lip injury
|
7.1%
1/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
0.00%
0/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
8.7%
4/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Investigations
Pseudomonas test positive
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
10.9%
5/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
28.6%
4/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
34.8%
16/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
7.1%
1/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
8.7%
4/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
35.7%
5/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
10.9%
5/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
General disorders
Crying
|
7.1%
1/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
0.00%
0/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
General disorders
Pyrexia
|
7.1%
1/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
21.7%
10/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Psychiatric disorders
Insomnia
|
7.1%
1/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
2.2%
1/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
6.5%
3/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Gastrointestinal disorders
Constipation
|
7.1%
1/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
10.9%
5/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
6.5%
3/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Gastrointestinal disorders
Flatulence
|
7.1%
1/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
2.2%
1/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
17.4%
8/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Skin and subcutaneous tissue disorders
Rash
|
21.4%
3/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
8.7%
4/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Metabolism and nutrition disorders
Decreased appetite
|
7.1%
1/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
2.2%
1/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Infections and infestations
Ear infection
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
10.9%
5/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
17.4%
8/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Infections and infestations
Influenza
|
14.3%
2/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
0.00%
0/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
8.7%
4/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Infections and infestations
Viral upper respiratory tract infection
|
0.00%
0/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
10.9%
5/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
|
Infections and infestations
Upper respiratory tract infection
|
7.1%
1/14 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
13.0%
6/46 • From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place