Trial Outcomes & Findings for A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease (NCT NCT00304512)
NCT ID: NCT00304512
Last Updated: 2018-10-03
Results Overview
TEAEs were defined as any adverse event with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe adverse event was defined as an adverse event that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through Week 48 is presented. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
9 participants
Primary outcome timeframe
Day 1 (after dosing) through Week 48
Results posted on
2018-10-03
Participant Flow
Before randomization, participants were stratified into 2 groups (high or low) by their baseline α-galactosidase A (α-Gal A) activity. High was defined as activity greater than 40% of normal; low was defined as activity less than or equal to 40% of normal.
Participant milestones
| Measure |
Migalastat Low Dose 50 mg
Migalastat 50 milligrams (mg) was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat Middle Dose 150 mg
Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat High Dose 250 mg
Migalastat 250 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
|---|---|---|---|
|
Treatment Period
STARTED
|
2
|
4
|
3
|
|
Treatment Period
Safety Population
|
2
|
4
|
3
|
|
Treatment Period
Pharmacokinetic (PK) Population
|
2
|
4
|
3
|
|
Treatment Period
Pharmacodynamic (PD) Population
|
2
|
4
|
3
|
|
Treatment Period
COMPLETED
|
2
|
4
|
3
|
|
Treatment Period
NOT COMPLETED
|
0
|
0
|
0
|
|
Extension Period
STARTED
|
2
|
4
|
3
|
|
Extension Period
COMPLETED
|
2
|
4
|
3
|
|
Extension Period
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
Baseline characteristics by cohort
| Measure |
Migalastat Low Dose 50 mg
n=2 Participants
Migalastat 50 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat Middle Dose 150 mg
n=4 Participants
Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat High Dose 250 mg
n=3 Participants
Migalastat 250 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
49.0 years
STANDARD_DEVIATION 18.38 • n=5 Participants
|
44.8 years
STANDARD_DEVIATION 9.95 • n=7 Participants
|
44.0 years
STANDARD_DEVIATION 2.65 • n=5 Participants
|
45.4 years
STANDARD_DEVIATION 9.23 • n=4 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Day 1 (after dosing) through Week 48Population: Safety Population: all participants who received at least 1 dose of study drug.
TEAEs were defined as any adverse event with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe adverse event was defined as an adverse event that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through Week 48 is presented. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Outcome measures
| Measure |
Migalastat Low Dose 50 mg
n=2 Participants
Migalastat 50 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat Middle Dose 150 mg
n=4 Participants
Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat High Dose 250 mg
n=3 Participants
Migalastat 250 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
|---|---|---|---|
|
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 0 (predose), 0.5, 1, 2, 3, 4, 5, 6, 8, and 10 hours (postdose)Population: PK Population: all participants who received study drug and had at least 1 postbaseline PK parameter recorded.
The AUC to the last measurable concentration (AUC0-t) was evaluated in plasma following a single oral dose of migalastat on Day 1 and following multiple oral doses on Day 14 (2 weeks) and Day 84 (12 weeks). All samples were collected on each dosing day.
Outcome measures
| Measure |
Migalastat Low Dose 50 mg
n=2 Participants
Migalastat 50 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat Middle Dose 150 mg
n=4 Participants
Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat High Dose 250 mg
n=3 Participants
Migalastat 250 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
|---|---|---|---|
|
PK: Area Under The Concentration Versus Time Curve (AUC) After Administration Of Migalastat
AUC0-t: Single Dose, Day 1
|
2628.9 nanograms*hours/milliliters (ng*hr/mL)
Geometric Coefficient of Variation 100.4
|
8941.6 nanograms*hours/milliliters (ng*hr/mL)
Geometric Coefficient of Variation 32.2
|
13217.2 nanograms*hours/milliliters (ng*hr/mL)
Geometric Coefficient of Variation 30.2
|
|
PK: Area Under The Concentration Versus Time Curve (AUC) After Administration Of Migalastat
AUC0-t: Multiple Dose, Day 14
|
3191.6 nanograms*hours/milliliters (ng*hr/mL)
Geometric Coefficient of Variation 64.9
|
10637.9 nanograms*hours/milliliters (ng*hr/mL)
Geometric Coefficient of Variation 35.6
|
14850.6 nanograms*hours/milliliters (ng*hr/mL)
Geometric Coefficient of Variation 9.6
|
|
PK: Area Under The Concentration Versus Time Curve (AUC) After Administration Of Migalastat
AUC0-t: Multiple Dose, Day 84
|
2300.3 nanograms*hours/milliliters (ng*hr/mL)
Geometric Coefficient of Variation 79.6
|
8581.9 nanograms*hours/milliliters (ng*hr/mL)
Geometric Coefficient of Variation 29.7
|
9970.3 nanograms*hours/milliliters (ng*hr/mL)
Geometric Coefficient of Variation 37.8
|
SECONDARY outcome
Timeframe: Baseline, Week 12 (end of treatment period), Week 48 (end of extension period)Population: PD Population: all participants who received study drug and had at least 1 postbaseline PD parameter recorded.
Leukocytes were isolated from whole blood and lysed, and α-Gal A activity was measured using a validated fluorometric assay, with catalysis to fluorescent 4-methylumbelliferone (4-MU) as the activity measure. The activity values obtained were normalized to protein (measured using a colorimetric assay) and reported as enzyme activity (nanomole \[nmol\] 4-MU/hr) per mg of protein. On Day 1 of the first visit and at every visit thereafter, the samples were collected prior to dosing with migalastat. α-Gal A activity in leukocytes are presented by individual participants.
Outcome measures
| Measure |
Migalastat Low Dose 50 mg
n=2 Participants
Migalastat 50 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat Middle Dose 150 mg
n=4 Participants
Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat High Dose 250 mg
n=3 Participants
Migalastat 250 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
|---|---|---|---|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 1: Baseline
|
13.4 nmol 4-MU/hr/mg protein
|
—
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 1: Week 12
|
2.46 nmol 4-MU/hr/mg protein
|
—
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 1: Week 48
|
26 nmol 4-MU/hr/mg protein
|
—
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 2: Baseline
|
24.5 nmol 4-MU/hr/mg protein
|
—
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 2: Week 12
|
35.8 nmol 4-MU/hr/mg protein
|
—
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 2: Week 48
|
39.6 nmol 4-MU/hr/mg protein
|
—
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 3: Baseline
|
—
|
25.1 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 3: Week 12
|
—
|
4.15 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 3: Week 48
|
—
|
NA nmol 4-MU/hr/mg protein
The 4-MU concentration at this time point was below the limit of quantitation
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 4: Baseline
|
—
|
6.39 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 4: Week 12
|
—
|
18.4 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 4: Week 48
|
—
|
16.1 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 5: Baseline
|
—
|
17.3 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 5: Week 12
|
—
|
5.85 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 5: Week 48
|
—
|
22.2 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 6: Baseline
|
—
|
24.6 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 6: Week 12
|
—
|
12.6 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 6: Week 48
|
—
|
46.5 nmol 4-MU/hr/mg protein
|
—
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 7: Baseline
|
—
|
—
|
3.25 nmol 4-MU/hr/mg protein
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 7: Week 12
|
—
|
—
|
6.56 nmol 4-MU/hr/mg protein
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 7: Week 48
|
—
|
—
|
4.83 nmol 4-MU/hr/mg protein
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 8: Baseline
|
—
|
—
|
14.7 nmol 4-MU/hr/mg protein
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 8: Week 12
|
—
|
—
|
23.3 nmol 4-MU/hr/mg protein
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 8: Week 48
|
—
|
—
|
29.2 nmol 4-MU/hr/mg protein
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 9: Baseline
|
—
|
—
|
13.1 nmol 4-MU/hr/mg protein
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 9: Week 12
|
—
|
—
|
8.86 nmol 4-MU/hr/mg protein
|
|
α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
Participant 9: Week 48
|
—
|
—
|
7.58 nmol 4-MU/hr/mg protein
|
Adverse Events
Migalastat Low Dose 50 mg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Migalastat Middle Dose 150 mg
Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths
Migalastat High Dose 250 mg
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Migalastat Low Dose 50 mg
n=2 participants at risk
Migalastat 50 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat Middle Dose 150 mg
n=4 participants at risk
Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat High Dose 250 mg
n=3 participants at risk
Migalastat 250 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
|---|---|---|---|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
Other adverse events
| Measure |
Migalastat Low Dose 50 mg
n=2 participants at risk
Migalastat 50 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat Middle Dose 150 mg
n=4 participants at risk
Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
Migalastat High Dose 250 mg
n=3 participants at risk
Migalastat 250 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Blood and lymphatic system disorders
Eosinophilia
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Atrioventricular block
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Bifascicular block
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Cardiomegaly
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Cardiomyopathy
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Diastolic dysfunction
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Mitral valve calcification
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Myocardial ischaemia
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Sinus arrhythmia
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Cardiac disorders
Ventricular hypertrophy
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Congenital, familial and genetic disorders
Fabry's disease
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Eye disorders
Visual acuity reduced
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Gastrointestinal disorders
Abdominal discomfort
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Gastrointestinal disorders
Abdominal mass
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Gastrointestinal disorders
Abdominal pain
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Gastrointestinal disorders
Diarrhoea
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Gastrointestinal disorders
Nausea
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
General disorders
Chest Pain
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
General disorders
Chills
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
General disorders
Face oedema
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
General disorders
Fatigue
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
General disorders
Feeling abnormal
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
General disorders
Oedema peripheral
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
General disorders
Pyrexia
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Bronchitis
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Bronchitis acute
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Fungal infection
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Impetigo
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Influenza
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Nasopharyngitis
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Sinusitis
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Viral infection
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Injury, poisoning and procedural complications
Subcutaneous haematoma
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Atrial pressure increased
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Blood glucose increased
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Blood urine present
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
C-reactive protein increased
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Cardiac murmur
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Echocardiogram abnormal
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Electrocardiogram abnormal
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Electrocardiogram PR shortened
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Nuclear magnetic resonance imaging abnormal
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Nuclear magnetic resonance imaging brain abnormal
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
QRS axis abnormal
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
50.0%
2/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Musculoskeletal and connective tissue disorders
Neck mass
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
50.0%
2/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Carotid artery stenosis
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Cerebral Artery occlusion
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Dizziness
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Gliosis
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Headache
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
75.0%
3/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Lethargy
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Memory impairment
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Vertebral artery stenosis
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Psychiatric disorders
Depressed mood
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Renal and urinary disorders
Dysuria
|
50.0%
1/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Renal and urinary disorders
Proteinuria
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Skin and subcutaneous tissue disorders
Rash macular
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
25.0%
1/4 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/3 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Vascular disorders
Aortic stenosis
|
0.00%
0/2 • Day 1 after dosing through Week 48 (end of extension period)
|
0.00%
0/4 • Day 1 after dosing through Week 48 (end of extension period)
|
33.3%
1/3 • Day 1 after dosing through Week 48 (end of extension period)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator can only publish the results from this trial provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review. If requested, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication for an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
- Publication restrictions are in place
Restriction type: OTHER