Trial Outcomes & Findings for A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del (NCT NCT03625466)
NCT ID: NCT03625466
Last Updated: 2022-11-02
Results Overview
MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = \<50% of lobe involved and 2 = \>=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
51 participants
Primary outcome timeframe
From Baseline at Week 48
Results posted on
2022-11-02
Participant Flow
This study was planned in 2 parts: Part 1 (Placebo-controlled Period) and Part 2 (Open-label Period). The primary and secondary efficacy analyses were planned for only Part 1.
This study was conducted in participants with cystic fibrosis (CF) aged 2 to 5 years.
Participant milestones
| Measure |
Part 1: Placebo
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
Participants weighing less than (\<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
Part 2: Overall LUM/IVA
Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \>=6 years of age at week 48) in open-label period for 48 weeks.
|
|---|---|---|---|
|
Placebo-controlled Period (48 Weeks)
STARTED
|
16
|
35
|
0
|
|
Placebo-controlled Period (48 Weeks)
COMPLETED
|
16
|
33
|
0
|
|
Placebo-controlled Period (48 Weeks)
NOT COMPLETED
|
0
|
2
|
0
|
|
Open-label Period (48 Weeks)
STARTED
|
0
|
0
|
49
|
|
Open-label Period (48 Weeks)
COMPLETED
|
0
|
0
|
48
|
|
Open-label Period (48 Weeks)
NOT COMPLETED
|
0
|
0
|
1
|
Reasons for withdrawal
| Measure |
Part 1: Placebo
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
Participants weighing less than (\<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
Part 2: Overall LUM/IVA
Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \>=6 years of age at week 48) in open-label period for 48 weeks.
|
|---|---|---|---|
|
Placebo-controlled Period (48 Weeks)
Adverse Event
|
0
|
1
|
0
|
|
Placebo-controlled Period (48 Weeks)
Withdrawal of Consent (not due to AE)
|
0
|
1
|
0
|
|
Open-label Period (48 Weeks)
Withdrawal of Consent (not due to AE)
|
0
|
0
|
1
|
Baseline Characteristics
Baseline population included all randomized participants who have received at least 1 dose of study drug. Here "number analyzed" signifies participants who were evaluable for this outcome measure at the specified time point.
Baseline characteristics by cohort
| Measure |
Part 1: Placebo
n=16 Participants
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
n=35 Participants
Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg FDC every q12h in placebo-controlled period for 48 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
Total
n=51 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
4.2 years
STANDARD_DEVIATION 1.0 • n=16 Participants
|
4.2 years
STANDARD_DEVIATION 1.0 • n=35 Participants
|
4.2 years
STANDARD_DEVIATION 1.0 • n=51 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=16 Participants
|
11 Participants
n=35 Participants
|
18 Participants
n=51 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=16 Participants
|
24 Participants
n=35 Participants
|
33 Participants
n=51 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=16 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=51 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=16 Participants
|
35 Participants
n=35 Participants
|
51 Participants
n=51 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=16 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=16 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=16 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=16 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=16 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
White
|
16 Participants
n=16 Participants
|
35 Participants
n=35 Participants
|
51 Participants
n=51 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=16 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=16 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=51 Participants
|
|
Magnetic Resonance Imaging (MRI) Global Chest Score
|
21.4 score on a scale
STANDARD_DEVIATION 9.3 • n=15 Participants • Baseline population included all randomized participants who have received at least 1 dose of study drug. Here "number analyzed" signifies participants who were evaluable for this outcome measure at the specified time point.
|
17.6 score on a scale
STANDARD_DEVIATION 9.7 • n=34 Participants • Baseline population included all randomized participants who have received at least 1 dose of study drug. Here "number analyzed" signifies participants who were evaluable for this outcome measure at the specified time point.
|
18.8 score on a scale
STANDARD_DEVIATION 9.6 • n=49 Participants • Baseline population included all randomized participants who have received at least 1 dose of study drug. Here "number analyzed" signifies participants who were evaluable for this outcome measure at the specified time point.
|
PRIMARY outcome
Timeframe: From Baseline at Week 48Population: Full Analysis Set (FAS) included all randomized participants who carried the intended CF transmembrane conductance regulator gene (CFTR) allele mutation and received at least 1 dose of study drug in Part 1. Here "overall number of participants analyzed" signifies participants who were evaluable for this outcome measure.
MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = \<50% of lobe involved and 2 = \>=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement.
Outcome measures
| Measure |
Part 1: Placebo
n=15 Participants
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
n=32 Participants
Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
|---|---|---|
|
Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48
|
-0.3 score on a scale
Standard Deviation 6.1
|
-1.7 score on a scale
Standard Deviation 6.6
|
SECONDARY outcome
Timeframe: From Baseline Through Week 48Population: FAS.
LCI2.5 represents the number of lung turnovers required to reduce the end-tidal inert gas concentration to 1/40th of its starting value.
Outcome measures
| Measure |
Part 1: Placebo
n=16 Participants
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
n=35 Participants
Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
|---|---|---|
|
Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48
|
0.32 lung clearance index
Interval -0.2 to 0.84
|
-0.37 lung clearance index
Interval -0.85 to 0.1
|
SECONDARY outcome
Timeframe: From Baseline at Week 48Population: FAS.
The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.
Outcome measures
| Measure |
Part 1: Placebo
n=16 Participants
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
n=35 Participants
Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
|---|---|---|
|
Part 1: Absolute Change in Weight-for-age Z-score at Week 48
|
-0.07 z-score
Interval -0.24 to 0.11
|
0.13 z-score
Interval -0.01 to 0.27
|
SECONDARY outcome
Timeframe: From Baseline at Week 48Population: FAS.
The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.
Outcome measures
| Measure |
Part 1: Placebo
n=16 Participants
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
n=35 Participants
Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
|---|---|---|
|
Part 1: Absolute Change in Stature-for-age Z-score at Week 48
|
0.10 z-score
Interval -0.04 to 0.24
|
0.09 z-score
Interval -0.05 to 0.22
|
SECONDARY outcome
Timeframe: From Baseline at Week 48Population: FAS.
BMI was defined as weight in kilogram (kg) divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.
Outcome measures
| Measure |
Part 1: Placebo
n=16 Participants
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
n=35 Participants
Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
|---|---|---|
|
Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48
|
-0.24 z-score
Interval -0.55 to 0.07
|
0.20 z-score
Interval -0.02 to 0.41
|
Adverse Events
Part 1: Placebo
Serious events: 2 serious events
Other events: 16 other events
Deaths: 0 deaths
Part 1: LUM/IVA
Serious events: 7 serious events
Other events: 33 other events
Deaths: 0 deaths
Part 2: Overall LUM/IVA
Serious events: 12 serious events
Other events: 41 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part 1: Placebo
n=16 participants at risk
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
n=35 participants at risk
Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
Part 2: Overall LUM/IVA
n=49 participants at risk
Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \>=6 years of age at week 48) in open-label period for 48 weeks.
|
|---|---|---|---|
|
Gastrointestinal disorders
Coeliac disease
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Distal intestinal obstruction syndrome
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Bronchopulmonary aspergillosis allergic
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Chronic sinusitis
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.6%
3/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
6.1%
3/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Otitis media
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Pseudomonas bronchitis
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Tonsillitis
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Injury, poisoning and procedural complications
Sedation complication
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Lung infiltration
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Haematemesis
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Intussusception
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
Other adverse events
| Measure |
Part 1: Placebo
n=16 participants at risk
Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
|
Part 1: LUM/IVA
n=35 participants at risk
Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.
|
Part 2: Overall LUM/IVA
n=49 participants at risk
Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \>=6 years of age at week 48) in open-label period for 48 weeks.
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
20.0%
7/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
12.2%
6/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
11.4%
4/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Diarrhoea
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
11.4%
4/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
6.1%
3/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Post-tussive vomiting
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Vomiting
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
5.7%
2/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.2%
4/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
General disorders
Pyrexia
|
18.8%
3/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
17.1%
6/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Bacterial disease carrier
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
6.1%
3/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Bronchitis
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
5.7%
2/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
12.2%
6/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Conjunctivitis
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Enterobiasis
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Gastroenteritis
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.6%
3/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
56.2%
9/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
42.9%
15/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
22.4%
11/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Nasopharyngitis
|
50.0%
8/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
62.9%
22/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
38.8%
19/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Otitis media
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.6%
3/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Rhinitis
|
37.5%
6/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
25.7%
9/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
16.3%
8/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Sinusitis
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.2%
4/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Upper respiratory tract infection
|
18.8%
3/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Viral infection
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
6.1%
3/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Injury, poisoning and procedural complications
Traumatic haematoma
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.6%
3/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.2%
4/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
5.7%
2/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Investigations
Staphylococcus test positive
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
6.1%
3/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Nervous system disorders
Headache
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.6%
3/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
6.1%
3/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
31.2%
5/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
28.6%
10/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
22.4%
11/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
5.7%
2/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
25.0%
4/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
16.3%
8/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.6%
3/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
4.1%
2/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Cardiac disorders
Tachycardia
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Ear and labyrinth disorders
Otorrhoea
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Eye disorders
Astigmatism
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
5.7%
2/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Eye disorders
Blepharospasm
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Abnormal faeces
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Faeces pale
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Gastrointestinal disorders
Frequent bowel movements
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
General disorders
Peripheral swelling
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Diarrhoea infectious
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Febrile infection
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Otitis media acute
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Pharyngitis streptococcal
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
5.7%
2/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Pneumonia pseudomonal
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Injury, poisoning and procedural complications
Skin laceration
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
5.7%
2/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Investigations
Pseudomonas test positive
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Lung infiltration
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal polyps
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
8.6%
3/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
12.5%
2/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
5.7%
2/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
0.00%
0/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
|
Infections and infestations
Tonsillitis
|
6.2%
1/16 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.9%
1/35 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
2.0%
1/49 • From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place