Trial Outcomes & Findings for Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype (NCT NCT04167345)
NCT ID: NCT04167345
Last Updated: 2022-02-02
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
48 participants
Primary outcome timeframe
From Baseline at Day 28
Results posted on
2022-02-02
Participant Flow
There were 3 parts in the study: Parts A1, A2 and B. As pre-specified in SAP, data collected for the placebo group for Parts A1, A2 and B were pooled and reported as a single combined arm (Parts A1, A2 and B Combined: Placebo) and data collected for VX-814 400 mg for Parts A1 and A2 were pooled and reported as a single combined arm (Parts A1 and A2 Combined: VX-814 400 milligrams \[mg\]) in the below presented results.
This study was conducted in participants 18 through 80 of years of age, inclusive with the PiZZ genotype.
Participant milestones
| Measure |
Parts A1, A2 and B Combined: Placebo
Participants received placebo matched to VX-814 in the treatment period for 28 days.
|
Part A1: VX-814 100 mg
Participants received VX-814 100 mg once every 12 hours (q12h) in the treatment period for 28 days.
|
Part A1: VX-814 200 mg
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
|
Parts A1 and A2 Combined: VX-814 400 mg
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
|
Part B: VX-814 600 mg
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
|
|---|---|---|---|---|---|
|
Overall Study
STARTED
|
10
|
4
|
3
|
13
|
18
|
|
Overall Study
COMPLETED
|
9
|
3
|
3
|
13
|
18
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
Parts A1, A2 and B Combined: Placebo
Participants received placebo matched to VX-814 in the treatment period for 28 days.
|
Part A1: VX-814 100 mg
Participants received VX-814 100 mg once every 12 hours (q12h) in the treatment period for 28 days.
|
Part A1: VX-814 200 mg
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
|
Parts A1 and A2 Combined: VX-814 400 mg
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
|
Part B: VX-814 600 mg
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
|
|---|---|---|---|---|---|
|
Overall Study
Other
|
1
|
1
|
0
|
0
|
0
|
Baseline Characteristics
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
Baseline characteristics by cohort
| Measure |
Parts A1, A2 and B Combined: Placebo
n=10 Participants
Participants received placebo matched to VX-814 in the treatment period for 28 days.
|
Part A1: VX-814 100 mg
n=4 Participants
Participants received VX-814 100 mg q12h in the treatment period for 28 days.
|
Part A1: VX-814 200 mg
n=3 Participants
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
|
Parts A1 and A2 Combined: VX-814 400 mg
n=13 Participants
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
|
Part B: VX-814 600 mg
n=18 Participants
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
|
Total
n=48 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Age, Continuous
|
54.6 years
STANDARD_DEVIATION 11.3 • n=93 Participants
|
51.8 years
STANDARD_DEVIATION 14.8 • n=4 Participants
|
55.5 years
STANDARD_DEVIATION 7.8 • n=27 Participants
|
57.9 years
STANDARD_DEVIATION 13.2 • n=483 Participants
|
57.9 years
STANDARD_DEVIATION 9.1 • n=36 Participants
|
56.5 years
STANDARD_DEVIATION 10.9 • n=10 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
7 Participants
n=483 Participants
|
12 Participants
n=36 Participants
|
27 Participants
n=10 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
6 Participants
n=483 Participants
|
6 Participants
n=36 Participants
|
21 Participants
n=10 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
2 Participants
n=10 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
12 Participants
n=483 Participants
|
14 Participants
n=36 Participants
|
41 Participants
n=10 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
1 Participants
n=483 Participants
|
4 Participants
n=36 Participants
|
5 Participants
n=10 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
0 Participants
n=10 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
0 Participants
n=10 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
0 Participants
n=10 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
0 Participants
n=10 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
13 Participants
n=483 Participants
|
18 Participants
n=36 Participants
|
48 Participants
n=10 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
0 Participants
n=10 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
0 Participants
n=10 Participants
|
|
Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
|
4.2 micromole per liter
STANDARD_DEVIATION 1.2 • n=93 Participants
|
5.1 micromole per liter
STANDARD_DEVIATION 2.3 • n=4 Participants
|
3.9 micromole per liter
STANDARD_DEVIATION 0.4 • n=27 Participants
|
3.9 micromole per liter
STANDARD_DEVIATION 0.7 • n=483 Participants
|
4.5 micromole per liter
STANDARD_DEVIATION 1.0 • n=36 Participants
|
4.3 micromole per liter
STANDARD_DEVIATION 1.1 • n=10 Participants
|
PRIMARY outcome
Timeframe: From Baseline at Day 28Population: Full analysis set (FAS) included all randomized participants who received at least 1 dose of study drug. The "overall number of participants analyzed" signifies participants who were evaluable at the specified time point. As pre-specified in SAP, statistical comparison with placebo were planned only for VX-814 400 mg and 600 mg treatment arms.
Outcome measures
| Measure |
Parts A1, A2 and B Combined: Placebo
n=6 Participants
Participants received placebo matched to VX-814 in the treatment period for 28 days.
|
Part A1: VX-814 100 mg
n=2 Participants
Participants received VX-814 100 mg q12h in the treatment period for 28 days.
|
Part A1: VX-814 200 mg
n=3 Participants
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
|
Parts A1 and A2 Combined: VX-814 400 mg
n=8 Participants
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
|
Part B: VX-814 600 mg
n=15 Participants
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
|
|---|---|---|---|---|---|
|
Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
|
-0.4 micromole per liter
Standard Deviation 0.3
|
0.2 micromole per liter
Standard Deviation 0.1
|
0.3 micromole per liter
Standard Deviation 0.5
|
1.4 micromole per liter
Standard Deviation 0.6
|
1.6 micromole per liter
Standard Deviation 1.0
|
PRIMARY outcome
Timeframe: Day 1 up to Week 8Population: The safety set included all participants who received at least 1 dose of study drug.
Outcome measures
| Measure |
Parts A1, A2 and B Combined: Placebo
n=10 Participants
Participants received placebo matched to VX-814 in the treatment period for 28 days.
|
Part A1: VX-814 100 mg
n=4 Participants
Participants received VX-814 100 mg q12h in the treatment period for 28 days.
|
Part A1: VX-814 200 mg
n=3 Participants
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
|
Parts A1 and A2 Combined: VX-814 400 mg
n=13 Participants
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
|
Part B: VX-814 600 mg
n=18 Participants
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
|
|---|---|---|---|---|---|
|
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants With AEs
|
4 participants
|
3 participants
|
1 participants
|
10 participants
|
14 participants
|
|
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants With SAEs
|
0 participants
|
2 participants
|
1 participants
|
1 participants
|
0 participants
|
SECONDARY outcome
Timeframe: From Baseline at Day 28Population: FAS. The "overall number of participants analyzed" signifies participants who were evaluable at the specified time point. As pre-specified in SAP, statistical comparisons with placebo were planned only for VX-814 400 mg and 600 mg treatment arms.
Outcome measures
| Measure |
Parts A1, A2 and B Combined: Placebo
n=6 Participants
Participants received placebo matched to VX-814 in the treatment period for 28 days.
|
Part A1: VX-814 100 mg
n=2 Participants
Participants received VX-814 100 mg q12h in the treatment period for 28 days.
|
Part A1: VX-814 200 mg
n=3 Participants
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
|
Parts A1 and A2 Combined: VX-814 400 mg
n=8 Participants
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
|
Part B: VX-814 600 mg
n=15 Participants
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
|
|---|---|---|---|---|---|
|
Change in Plasma Antigenic AAT Levels
|
0.4 micromole per liter
Standard Deviation 1.3
|
0.1 micromole per liter
Standard Deviation 0.2
|
0.7 micromole per liter
Standard Deviation 0.7
|
2.4 micromole per liter
Standard Deviation 1.2
|
2.6 micromole per liter
Standard Deviation 1.1
|
SECONDARY outcome
Timeframe: Pre-dose at Day 7, Day 14, Day 21, and Day 28Population: Pharmacokinetic analysis included all randomized participants who received at least 1 dose of study drug. Here "number analyzed" signifies participants who were evaluable at the specified time point.
Outcome measures
| Measure |
Parts A1, A2 and B Combined: Placebo
n=4 Participants
Participants received placebo matched to VX-814 in the treatment period for 28 days.
|
Part A1: VX-814 100 mg
n=3 Participants
Participants received VX-814 100 mg q12h in the treatment period for 28 days.
|
Part A1: VX-814 200 mg
n=13 Participants
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
|
Parts A1 and A2 Combined: VX-814 400 mg
n=18 Participants
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
|
Part B: VX-814 600 mg
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
|
|---|---|---|---|---|---|
|
Observed Pre-dose Plasma Concentration of VX-814
Day 21
|
0.700 microgram per milliliter
|
1.45 microgram per milliliter
|
3.68 microgram per milliliter
Standard Deviation 3.40
|
7.20 microgram per milliliter
Standard Deviation 6.05
|
—
|
|
Observed Pre-dose Plasma Concentration of VX-814
Day 28
|
0.326 microgram per milliliter
Standard Deviation 0.0282
|
0.993 microgram per milliliter
Standard Deviation 0.0300
|
3.23 microgram per milliliter
Standard Deviation 2.82
|
5.80 microgram per milliliter
Standard Deviation 3.73
|
—
|
|
Observed Pre-dose Plasma Concentration of VX-814
Day 7
|
0.476 microgram per milliliter
Standard Deviation 0.375
|
0.948 microgram per milliliter
Standard Deviation 0.512
|
3.53 microgram per milliliter
Standard Deviation 1.72
|
10.3 microgram per milliliter
Standard Deviation 6.55
|
—
|
|
Observed Pre-dose Plasma Concentration of VX-814
Day 14
|
0.244 microgram per milliliter
Standard Deviation 0.0919
|
1.07 microgram per milliliter
Standard Deviation 0.152
|
3.25 microgram per milliliter
Standard Deviation 2.23
|
8.53 microgram per milliliter
Standard Deviation 11.8
|
—
|
Adverse Events
Parts A1, A2 and B Combined: Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Part A1: VX-814 100 mg
Serious events: 2 serious events
Other events: 1 other events
Deaths: 0 deaths
Part A1: VX-814 200 mg
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Parts A1 and A2 Combined: VX-814 400 mg
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Part B: VX-814 600 mg
Serious events: 0 serious events
Other events: 14 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Parts A1, A2 and B Combined: Placebo
n=10 participants at risk
Participants received placebo matched to VX-814 in the treatment period for 28 days.
|
Part A1: VX-814 100 mg
n=4 participants at risk
Participants received VX-814 100 mg q12h in the treatment period for 28 days.
|
Part A1: VX-814 200 mg
n=3 participants at risk
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
|
Parts A1 and A2 Combined: VX-814 400 mg
n=13 participants at risk
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
|
Part B: VX-814 600 mg
n=18 participants at risk
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
|
|---|---|---|---|---|---|
|
Infections and infestations
Gastrointestinal infection
|
0.00%
0/10 • Day 1 up to Week 8
|
25.0%
1/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
33.3%
1/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
33.3%
1/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
0.00%
0/10 • Day 1 up to Week 8
|
25.0%
1/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
Other adverse events
| Measure |
Parts A1, A2 and B Combined: Placebo
n=10 participants at risk
Participants received placebo matched to VX-814 in the treatment period for 28 days.
|
Part A1: VX-814 100 mg
n=4 participants at risk
Participants received VX-814 100 mg q12h in the treatment period for 28 days.
|
Part A1: VX-814 200 mg
n=3 participants at risk
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
|
Parts A1 and A2 Combined: VX-814 400 mg
n=13 participants at risk
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
|
Part B: VX-814 600 mg
n=18 participants at risk
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
|
|---|---|---|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
20.0%
2/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
16.7%
3/18 • Day 1 up to Week 8
|
|
Gastrointestinal disorders
Dyspepsia
|
10.0%
1/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Gastrointestinal disorders
Frequent bowel movements
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
11.1%
2/18 • Day 1 up to Week 8
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
General disorders
Fatigue
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
11.1%
2/18 • Day 1 up to Week 8
|
|
Infections and infestations
Ear infection
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Infections and infestations
Respiratory tract infection
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Infections and infestations
Rhinitis
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Cardiac disorders
Palpitations
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Gastrointestinal disorders
Abdominal distension
|
10.0%
1/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
16.7%
3/18 • Day 1 up to Week 8
|
|
Infections and infestations
Tooth abscess
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
11.1%
2/18 • Day 1 up to Week 8
|
|
Infections and infestations
Vulvovaginal candidiasis
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Injury, poisoning and procedural complications
Dental restoration failure
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
15.4%
2/13 • Day 1 up to Week 8
|
33.3%
6/18 • Day 1 up to Week 8
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
15.4%
2/13 • Day 1 up to Week 8
|
33.3%
6/18 • Day 1 up to Week 8
|
|
Investigations
Blood cholesterol increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Investigations
Blood pressure increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
11.1%
2/18 • Day 1 up to Week 8
|
|
Investigations
Crystal urine present
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Investigations
Eosinophil count increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Investigations
Gamma-glutamyltransferase increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Investigations
International normalised ratio increased
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Investigations
Protein urine present
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Investigations
Prothrombin time prolonged
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
10.0%
1/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Nervous system disorders
Headache
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
15.4%
2/13 • Day 1 up to Week 8
|
11.1%
2/18 • Day 1 up to Week 8
|
|
Nervous system disorders
Restless legs syndrome
|
10.0%
1/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Psychiatric disorders
Nightmare
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Renal and urinary disorders
Pollakiuria
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
10.0%
1/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Respiratory, thoracic and mediastinal disorders
Respiration abnormal
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
5.6%
1/18 • Day 1 up to Week 8
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
0.00%
0/10 • Day 1 up to Week 8
|
25.0%
1/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
0.00%
0/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
|
Vascular disorders
Hot flush
|
0.00%
0/10 • Day 1 up to Week 8
|
0.00%
0/4 • Day 1 up to Week 8
|
0.00%
0/3 • Day 1 up to Week 8
|
7.7%
1/13 • Day 1 up to Week 8
|
0.00%
0/18 • Day 1 up to Week 8
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER