Trial Outcomes & Findings for Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme (NCT NCT00837824)
NCT ID: NCT00837824
Last Updated: 2015-04-07
Results Overview
The trial was terminated early due to inadequate study design. During the study period of 7 months, only 1 patient had a clinical event, a stroke, in the Fabrazyme 1 mg/kg treatment arm. The time to event was determined from first dose of Fabrazyme to the date of event.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
7 months
Results posted on
2015-04-07
Participant Flow
Because the study was ended early only 20 patients from 9 sites were randomized into this study using a 1:1 randomization scheme to receive biweekly infusions of either 1 mg/kg (11 patients) or 3 mg/kg (9 patients) of Fabrazyme.
Participant milestones
| Measure |
Fabrazyme 1mg/kg Every 2 Weeks
Fabrazyme 1.0 mg/kg every 2 weeks
|
Fabrazyme 3mg/kg Every 2 Weeks
Fabrazyme 3.0 mg/kg every 2 weeks
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
9
|
|
Overall Study
COMPLETED
|
11
|
9
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
Baseline characteristics by cohort
| Measure |
Fabrazyme 1mg/kg Every 2 Weeks
n=11 Participants
Fabrazyme 1.0 mg/kg every 2 weeks
|
Fabrazyme 3mg/kg Every 2 Weeks
n=9 Participants
Fabrazyme 3.0 mg/kg every 2 weeks
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
< 40 Years
|
1 Years
n=5 Participants
|
1 Years
n=7 Participants
|
2 Years
n=5 Participants
|
|
Age, Customized
≥ 40 Years
|
10 Years
n=5 Participants
|
8 Years
n=7 Participants
|
18 Years
n=5 Participants
|
|
Age, Continuous
|
49.4 years
STANDARD_DEVIATION 6.96 • n=5 Participants
|
48.9 years
STANDARD_DEVIATION 7.55 • n=7 Participants
|
49.2 years
STANDARD_DEVIATION 7.04 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
7 Number of Participants
n=5 Participants
|
7 Number of Participants
n=7 Participants
|
14 Number of Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black
|
3 Number of Participants
n=5 Participants
|
2 Number of Participants
n=7 Participants
|
5 Number of Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
1 Number of Participants
n=5 Participants
|
0 Number of Participants
n=7 Participants
|
1 Number of Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 7 monthsPopulation: Intent-to-Treat (ITT) Population-consisted of all 20 patients enrolled in the trial. The original sample size was 120 patients. Due to early termination, only 20 patients were enrolled in this trial. No imputation of data was performed. During the 7 months study period, only 1 patient had a clinical event in the Fabrazyme 1 mg/kg treatment arm.
The trial was terminated early due to inadequate study design. During the study period of 7 months, only 1 patient had a clinical event, a stroke, in the Fabrazyme 1 mg/kg treatment arm. The time to event was determined from first dose of Fabrazyme to the date of event.
Outcome measures
| Measure |
Fabrazyme 1mg/kg Every 2 Weeks
n=1 Participants
Fabrazyme 1.0 mg/kg every 2 weeks
|
Fabrazyme 3mg/kg Every 2 Weeks
Fabrazyme 3.0 mg/kg every 2 weeks
|
|---|---|---|
|
Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease
|
35 Days
|
—
|
SECONDARY outcome
Timeframe: Evaluated at Baseline, Month 3, and Final VisitPopulation: Fabrazyme 1mg/kg every 2 weeks: ITT population - 11 patients at baseline, 7 patients at Month 3, and 9 patients at Final Visit had Plasma GL-3 values. Fabrazyme 3mg/kg every 2 weeks: ITT population - 9 patients at baseline, 2 patients at Month 3, and 7 patients at Final Visit had Plasma GL-3 values
This outcome measure evaluated the mean plasma GL-3 values for all patients to see if it decreased while on Fabrazyme. Normal plasma GL-3 level is defined as ≤ 7.03 µg/mL.
Outcome measures
| Measure |
Fabrazyme 1mg/kg Every 2 Weeks
n=11 Participants
Fabrazyme 1.0 mg/kg every 2 weeks
|
Fabrazyme 3mg/kg Every 2 Weeks
n=9 Participants
Fabrazyme 3.0 mg/kg every 2 weeks
|
|---|---|---|
|
Plasma Globotriaosylceramide (GL-3)
Plasma GL-3 at Baseline
|
8.7 µg/mL
Standard Deviation 3.33
|
8.3 µg/mL
Standard Deviation 3.92
|
|
Plasma Globotriaosylceramide (GL-3)
Plasma GL-3 at Month 3
|
5.1 µg/mL
Standard Deviation 2.05
|
5.6 µg/mL
Standard Deviation 0.28
|
|
Plasma Globotriaosylceramide (GL-3)
Plasma GL-3 at Final Visit
|
5.1 µg/mL
Standard Deviation 1.53
|
4.9 µg/mL
Standard Deviation 1.74
|
Adverse Events
Fabrazyme 1mg/kg Every 2 Weeks
Serious events: 4 serious events
Other events: 10 other events
Deaths: 0 deaths
Fabrazyme 3mg/kg Every 2 Weeks
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Total
Serious events: 5 serious events
Other events: 19 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Fabrazyme 1mg/kg Every 2 Weeks
n=11 participants at risk
Fabrazyme 1.0 mg/kg every 2 weeks
|
Fabrazyme 3mg/kg Every 2 Weeks
n=9 participants at risk
Fabrazyme 3.0 mg/kg every 2 weeks
|
Total
n=20 participants at risk
|
|---|---|---|---|
|
Cardiac disorders
Atrial fibrillation
|
18.2%
2/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
10.0%
2/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Cardiac disorders
Atrial flutter
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Cardiac disorders
Coronary artery occlusion
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Pyrexia
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Sepsis
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Injury, poisoning and procedural complications
Arteriovenous fistula thrombosis
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Nervous system disorders
Cerebrovascular accident
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
Other adverse events
| Measure |
Fabrazyme 1mg/kg Every 2 Weeks
n=11 participants at risk
Fabrazyme 1.0 mg/kg every 2 weeks
|
Fabrazyme 3mg/kg Every 2 Weeks
n=9 participants at risk
Fabrazyme 3.0 mg/kg every 2 weeks
|
Total
n=20 participants at risk
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Ear and labyrinth disorders
Ear congestion
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Ear and labyrinth disorders
Ear discomfort
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Gastrointestinal disorders
Abdominal distension
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Gastrointestinal disorders
Abdominal pain
|
18.2%
2/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
10.0%
2/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Gastrointestinal disorders
Constipation
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Gastrointestinal disorders
Food poisoning
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Gastrointestinal disorders
Gastrointestinal hypermotility
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Gastrointestinal disorders
Vomiting
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Adverse event
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Chills
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Fatigue
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
10.0%
2/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Feeling hot
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Influenza like illness
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
10.0%
2/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Malaise
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Oedema peripheral
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
33.3%
3/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
15.0%
3/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Pain
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
10.0%
2/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Pyrexia
|
18.2%
2/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
10.0%
2/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Sluggishness
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Arteriovenous fistula site infection
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Upper respiratory tract infection
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Injury, poisoning and procedural complications
Excoriation
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Injury, poisoning and procedural complications
Joint sprain
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Injury, poisoning and procedural complications
Muscle strain
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Investigations
Blood bicarbonate decreased
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Investigations
Blood calcium decreased
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Investigations
Blood phosphorus increased
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Investigations
Breath sounds abnormal
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Investigations
Haematocrit decreased
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Investigations
Haemoglobin decreased
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Investigations
Red blood cell count decreased
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Metabolism and nutrition disorders
Gout
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Metabolism and nutrition disorders
Hypovolaemia
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Musculoskeletal and connective tissue disorders
Limb discomfort
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
22.2%
2/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
10.0%
2/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal discomfort
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Nervous system disorders
Dizziness
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Nervous system disorders
Headache
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
22.2%
2/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
15.0%
3/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Nervous system disorders
Restless legs syndrome
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Nervous system disorders
Sinus headache
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Nervous system disorders
Somnolence
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Nervous system disorders
Speech disorder
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Nervous system disorders
Transient ischaemic attack
|
18.2%
2/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
10.0%
2/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
10.0%
2/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
11.1%
1/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
9.1%
1/11
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
0.00%
0/9
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
5.0%
1/20
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee In multi-site studies, PI can publish after Genzyme publishes or 18 months after study completion. PI gives Genzyme a draft 60 days before publication. Genzyme can ask that confidential information be removed, and can defer publication another 60 days upon notifying PI that it will file a patent application on inventions contained in the draft.
- Publication restrictions are in place
Restriction type: OTHER