Trial Outcomes & Findings for A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis (NCT NCT03911713)
NCT ID: NCT03911713
Last Updated: 2022-01-25
Results Overview
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
77 participants
Primary outcome timeframe
From Baseline at Week 12
Results posted on
2022-01-25
Participant Flow
This study was conducted in cystic fibrosis (CF) participants aged 18 years or older who have a gating mutation and were previously taking stable dose of ivacaftor (IVA).
Participant milestones
| Measure |
Ivacaftor
Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.
|
VX-561: 25 mg
Participants received VX-561 25 mg orally once daily (qd) in the treatment period for 12 weeks.
|
VX-561: 50 mg
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 150 mg
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
|---|---|---|---|---|---|
|
Overall Study
STARTED
|
12
|
6
|
11
|
24
|
24
|
|
Overall Study
Full Analysis Set
|
11
|
6
|
11
|
23
|
24
|
|
Overall Study
COMPLETED
|
11
|
4
|
11
|
22
|
24
|
|
Overall Study
NOT COMPLETED
|
1
|
2
|
0
|
2
|
0
|
Reasons for withdrawal
| Measure |
Ivacaftor
Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.
|
VX-561: 25 mg
Participants received VX-561 25 mg orally once daily (qd) in the treatment period for 12 weeks.
|
VX-561: 50 mg
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 150 mg
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
|---|---|---|---|---|---|
|
Overall Study
Lost to Follow-up
|
0
|
2
|
0
|
0
|
0
|
|
Overall Study
Other
|
0
|
0
|
0
|
1
|
0
|
|
Overall Study
Randomized, Never Dosed
|
1
|
0
|
0
|
1
|
0
|
Baseline Characteristics
A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Ivacaftor
n=11 Participants
Participants received IVA 150 mg orally q12h in the treatment period for 12 weeks.
|
VX-561: 25 mg
n=6 Participants
Participants received VX-561 25 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 50 mg
n=11 Participants
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 150 mg
n=23 Participants
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
n=24 Participants
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
Total
n=75 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Age, Continuous
|
33.3 years
STANDARD_DEVIATION 11.7 • n=5 Participants
|
33.0 years
STANDARD_DEVIATION 10.6 • n=7 Participants
|
27.8 years
STANDARD_DEVIATION 9.0 • n=5 Participants
|
32.5 years
STANDARD_DEVIATION 8.5 • n=4 Participants
|
37.4 years
STANDARD_DEVIATION 11.4 • n=21 Participants
|
33.5 years
STANDARD_DEVIATION 10.4 • n=8 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
26 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
15 Participants
n=4 Participants
|
15 Participants
n=21 Participants
|
49 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
2 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
22 Participants
n=4 Participants
|
24 Participants
n=21 Participants
|
73 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
1 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
23 Participants
n=4 Participants
|
24 Participants
n=21 Participants
|
74 Participants
n=8 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
|
74.0 percentage points
STANDARD_DEVIATION 21.2 • n=5 Participants
|
63.6 percentage points
STANDARD_DEVIATION 22.4 • n=7 Participants
|
66.8 percentage points
STANDARD_DEVIATION 17.4 • n=5 Participants
|
72.6 percentage points
STANDARD_DEVIATION 17.3 • n=4 Participants
|
73.9 percentage points
STANDARD_DEVIATION 17.0 • n=21 Participants
|
71.6 percentage points
STANDARD_DEVIATION 18.0 • n=8 Participants
|
PRIMARY outcome
Timeframe: From Baseline at Week 12Population: Full analysis set (FAS) included all randomized participants who have intended CF transmembrane conductance regulator gene (CFTR) genotype and received at least 1 dose of study drug in treatment period. VX-561:25 mg and VX-561:50 mg arms were discontinued at sponsor's discretion and it was specified in statistical plan that data will be reported for only IVA, VX-561:150 mg and VX-561:250 mg arms for this outcome measure.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Outcome measures
| Measure |
Ivacaftor
n=11 Participants
Participants received IVA 150 mg orally q12h in the treatment period for 12 weeks.
|
VX-561: 150 mg
n=23 Participants
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
n=24 Participants
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 150 mg
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
|---|---|---|---|---|---|
|
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
|
-0.8 percentage points
Interval -6.2 to 4.7
|
3.1 percentage points
Interval -0.8 to 7.0
|
2.7 percentage points
Interval -1.0 to 6.5
|
—
|
—
|
SECONDARY outcome
Timeframe: From Baseline at Week 12Population: FAS. VX-561:25 mg and VX-561:50 mg arms were discontinued at sponsor's discretion and it was specified in statistical plan that data will be reported for only IVA, VX-561:150 mg and VX-561:250 mg arms for this outcome measure.
Sweat samples were collected using an approved collection device.
Outcome measures
| Measure |
Ivacaftor
n=11 Participants
Participants received IVA 150 mg orally q12h in the treatment period for 12 weeks.
|
VX-561: 150 mg
n=23 Participants
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
n=24 Participants
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 150 mg
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
|---|---|---|---|---|---|
|
Absolute Change in Sweat Chloride (SwCl)
|
0.9 millimole per liter (mmol/L)
Interval -9.5 to 11.3
|
3.3 millimole per liter (mmol/L)
Interval -4.6 to 11.2
|
-6.5 millimole per liter (mmol/L)
Interval -14.1 to 1.2
|
—
|
—
|
SECONDARY outcome
Timeframe: At Week 4Population: Pharmacokinetic (PK) set included all participants who received at least 1 dose of study drug and for whom the primary PK data were considered to be sufficient and interpretable. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number Analyzed" signifies those who were evaluable for the specific category.
Outcome measures
| Measure |
Ivacaftor
n=9 Participants
Participants received IVA 150 mg orally q12h in the treatment period for 12 weeks.
|
VX-561: 150 mg
n=4 Participants
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
n=7 Participants
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 150 mg
n=20 Participants
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
n=20 Participants
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
|---|---|---|---|---|---|
|
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
VX-561: Week 4
|
—
|
26.1 nanogram per milliliter (ng/mL)
Standard Deviation 24.7
|
123 nanogram per milliliter (ng/mL)
Standard Deviation 61.6
|
458 nanogram per milliliter (ng/mL)
Standard Deviation 273
|
1100 nanogram per milliliter (ng/mL)
Standard Deviation 856
|
|
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
M1-VX-561: Week 4
|
—
|
18.1 nanogram per milliliter (ng/mL)
Standard Deviation 17.7
|
108 nanogram per milliliter (ng/mL)
Standard Deviation 58.6
|
378 nanogram per milliliter (ng/mL)
Standard Deviation 213
|
739 nanogram per milliliter (ng/mL)
Standard Deviation 407
|
|
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
M6-VX-561: Week 4
|
—
|
NA nanogram per milliliter (ng/mL)
Standard Deviation NA
NA indicates that summary statistics were not reported as the number of observations below the limit of quantification was greater than 50% of the total number of observations at the specified time points.
|
59.8 nanogram per milliliter (ng/mL)
Standard Deviation 34.0
|
211 nanogram per milliliter (ng/mL)
Standard Deviation 189
|
370 nanogram per milliliter (ng/mL)
Standard Deviation 233
|
|
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
IVA: Week 4
|
952 nanogram per milliliter (ng/mL)
Standard Deviation 766
|
—
|
—
|
—
|
—
|
|
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
M1-IVA: Week 4
|
1330 nanogram per milliliter (ng/mL)
Standard Deviation 774
|
—
|
—
|
—
|
—
|
|
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
M6-IVA: Week 4
|
662 nanogram per milliliter (ng/mL)
Standard Deviation 398
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 16Population: Safety Set included all participants who received at least 1 dose of study drug.
Outcome measures
| Measure |
Ivacaftor
n=11 Participants
Participants received IVA 150 mg orally q12h in the treatment period for 12 weeks.
|
VX-561: 150 mg
n=6 Participants
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
n=11 Participants
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 150 mg
n=23 Participants
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
n=24 Participants
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
|---|---|---|---|---|---|
|
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with AEs
|
8 participants
|
4 participants
|
8 participants
|
21 participants
|
23 participants
|
|
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
1 participants
|
2 participants
|
2 participants
|
2 participants
|
1 participants
|
Adverse Events
Ivacaftor
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
VX-561: 25 mg
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
VX-561: 50 mg
Serious events: 2 serious events
Other events: 8 other events
Deaths: 0 deaths
VX-561: 150 mg
Serious events: 2 serious events
Other events: 17 other events
Deaths: 0 deaths
VX-561: 250 mg
Serious events: 1 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Ivacaftor
n=11 participants at risk
Participants received IVA 150 mg orally q12h in the treatment period for 12 weeks.
|
VX-561: 25 mg
n=6 participants at risk
Participants received VX-561 25 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 50 mg
n=11 participants at risk
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 150 mg
n=23 participants at risk
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
n=24 participants at risk
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
|---|---|---|---|---|---|
|
Gastrointestinal disorders
Distal intestinal obstruction syndrome
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
9.1%
1/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
18.2%
2/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Investigations
Forced expiratory volume decreased
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
Other adverse events
| Measure |
Ivacaftor
n=11 participants at risk
Participants received IVA 150 mg orally q12h in the treatment period for 12 weeks.
|
VX-561: 25 mg
n=6 participants at risk
Participants received VX-561 25 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 50 mg
n=11 participants at risk
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 150 mg
n=23 participants at risk
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
|
VX-561: 250 mg
n=24 participants at risk
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
|
|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Cardiac disorders
Palpitations
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Ear and labyrinth disorders
Ear pain
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Ear and labyrinth disorders
Tinnitus
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Eye disorders
Glaucoma
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Gastrointestinal disorders
Abdominal pain lower
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Gastrointestinal disorders
Constipation
|
9.1%
1/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Gastrointestinal disorders
Duodenitis
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Gastrointestinal disorders
Nausea
|
9.1%
1/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
General disorders
Chest discomfort
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
General disorders
Fatigue
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
General disorders
Malaise
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
General disorders
Pyrexia
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Infections and infestations
Bronchopulmonary aspergillosis allergic
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Infections and infestations
Epididymitis
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Infections and infestations
Fungal skin infection
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
18.2%
2/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
18.2%
2/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
16.7%
4/24 • Baseline up to Week 16
|
|
Infections and infestations
Nasopharyngitis
|
18.2%
2/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
12.5%
3/24 • Baseline up to Week 16
|
|
Infections and infestations
Oral candidiasis
|
9.1%
1/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Infections and infestations
Sinusitis
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
8.3%
2/24 • Baseline up to Week 16
|
|
Infections and infestations
Upper respiratory tract infection
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
4.3%
1/23 • Baseline up to Week 16
|
8.3%
2/24 • Baseline up to Week 16
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
18.2%
2/11 • Baseline up to Week 16
|
4.3%
1/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Investigations
Atypical mycobacterium test positive
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Investigations
Bacterial test positive
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Investigations
Blood creatine phosphokinase increased
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
18.2%
2/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Investigations
Blood glucose increased
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Investigations
Coronavirus test positive
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Investigations
Forced expiratory volume decreased
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Investigations
Protein urine present
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Investigations
Pulmonary function test decreased
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Investigations
Urine ketone body present
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Investigations
Weight decreased
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Nervous system disorders
Dizziness
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Nervous system disorders
Headache
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
4.3%
1/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Nervous system disorders
Migraine
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Nervous system disorders
Sinus headache
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.2%
2/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
26.1%
6/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/11 • Baseline up to Week 16
|
33.3%
2/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
13.0%
3/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Increased viscosity of bronchial secretion
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Lower respiratory tract congestion
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
18.2%
2/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
8.7%
2/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
18.2%
2/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
17.4%
4/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Paranasal sinus discomfort
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
4.3%
1/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary pain
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Rales
|
0.00%
0/11 • Baseline up to Week 16
|
16.7%
1/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
4.3%
1/23 • Baseline up to Week 16
|
4.2%
1/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Respiration abnormal
|
18.2%
2/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
17.4%
4/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
17.4%
4/23 • Baseline up to Week 16
|
12.5%
3/24 • Baseline up to Week 16
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
0.00%
0/24 • Baseline up to Week 16
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
8.3%
2/24 • Baseline up to Week 16
|
|
Skin and subcutaneous tissue disorders
Rash
|
9.1%
1/11 • Baseline up to Week 16
|
0.00%
0/6 • Baseline up to Week 16
|
0.00%
0/11 • Baseline up to Week 16
|
0.00%
0/23 • Baseline up to Week 16
|
8.3%
2/24 • Baseline up to Week 16
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place