Trial Outcomes & Findings for A Study of Fabrazyme in Pediatric Patients With Fabry Disease (NCT NCT00074958)
NCT ID: NCT00074958
Last Updated: 2015-04-02
Results Overview
Skin biopsies were taken at Baseline, Week 24 and Week 48 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
16 participants
Primary outcome timeframe
Baseline, Week 24 and Week 48
Results posted on
2015-04-02
Participant Flow
Participant milestones
| Measure |
Fabrazyme
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
15
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Fabrazyme
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
A Study of Fabrazyme in Pediatric Patients With Fabry Disease
Baseline characteristics by cohort
| Measure |
Fabrazyme
n=16 Participants
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
|
|---|---|
|
Age, Continuous
|
12.1 years
STANDARD_DEVIATION 2.52 • n=93 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 24 and Week 48Population: Intent to Treat (ITT) population - male patients only. 14 patients had skin biopsies performed at Baseline and Week 24 but only 5 patients had skin biopsies performed at Week 48.
Skin biopsies were taken at Baseline, Week 24 and Week 48 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
Outcome measures
| Measure |
Fabrazyme
n=14 Participants
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
|
|---|---|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
None/Trace (0) Score at Baseline
|
2 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
None/Trace (0) Score at Week 24
|
14 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
None/Trace (0) Score at Week 48
|
5 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Mild (1) Score at Baseline
|
0 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Mild (1) Score at Week 24
|
0 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Mild (1) Score at Week 48
|
0 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Moderate (2) Score at Baseline
|
11 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Moderate (2) Score at Week 24
|
0 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Moderate (2) Score at Week 48
|
0 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Severe (3) Score at Baseline
|
1 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Severe (3) Score at Week 24
|
0 patients
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Severe (3) Score at Week 48
|
0 patients
|
SECONDARY outcome
Timeframe: Baseline, Week 24 and Week 48Population: ITT population. 16 male patients had plasma GL-3 values at Baseline and Week 24, while 15 male patients had plasma GL-3 values at Week 48. 2 female patients had plasma GL-3 values at Baseline, Week 24 and Week 48.
Plasma GL-3 values at Baseline, Week 24, and Week 48. Normal plasma GL-3 level is ≤ 7.03 µg/mL.
Outcome measures
| Measure |
Fabrazyme
n=16 Participants
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
|
|---|---|
|
Plasma GL-3
Overall at Week 48
|
4.9 µg/mL
Standard Deviation 1.01
|
|
Plasma GL-3
Male Patients at Baseline
|
15.4 µg/mL
Standard Deviation 4.10
|
|
Plasma GL-3
Female Patients at Baseline
|
4.9 µg/mL
Standard Deviation 0.64
|
|
Plasma GL-3
Overall at Baseline
|
14.1 µg/mL
Standard Deviation 5.25
|
|
Plasma GL-3
Males at Week 24
|
5.5 µg/mL
Standard Deviation 0.95
|
|
Plasma GL-3
Females at Week 24
|
3.6 µg/mL
Standard Deviation 0.57
|
|
Plasma GL-3
Overall at Week 24
|
5.2 µg/mL
Standard Deviation 1.10
|
|
Plasma GL-3
Males at Week 48
|
5.0 µg/mL
Standard Deviation 1.00
|
|
Plasma GL-3
Females at Week 48
|
4.4 µg/mL
Standard Deviation 1.27
|
Adverse Events
Fabrazyme
Serious events: 1 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Fabrazyme
n=16 participants at risk
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
|
|---|---|
|
Immune system disorders
Anaphylactic shock
|
6.2%
1/16
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
n=16 participants at risk
1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
6.2%
1/16
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.
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
12.5%
2/16
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
Angina pectoris
|
6.2%
1/16
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
Arrhythmia
|
6.2%
1/16
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
Tachycardia
|
6.2%
1/16
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 pain
|
6.2%
1/16
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
Tinnitus
|
6.2%
1/16
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
Vertigo
|
6.2%
1/16
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.
|
|
Eye disorders
Eye oedema
|
6.2%
1/16
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.
|
|
Eye disorders
Lacrimation increased
|
12.5%
2/16
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.
|
|
Eye disorders
Photophobia
|
6.2%
1/16
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.
|
|
Eye disorders
Vision blurred
|
12.5%
2/16
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
|
43.8%
7/16
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 upper
|
25.0%
4/16
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
Chapped lips
|
6.2%
1/16
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
|
18.8%
3/16
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
|
31.2%
5/16
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
Dyspepsia
|
18.8%
3/16
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
Nausea
|
56.2%
9/16
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
Toothache
|
12.5%
2/16
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
|
56.2%
9/16
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
Asthenia
|
12.5%
2/16
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
Catheter site pain
|
6.2%
1/16
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
Chest pain
|
12.5%
2/16
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
|
18.8%
3/16
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
|
6.2%
1/16
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 cold
|
12.5%
2/16
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
|
12.5%
2/16
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
Hyperthermia
|
6.2%
1/16
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
Injection site pain
|
6.2%
1/16
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
|
6.2%
1/16
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
|
6.2%
1/16
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
|
6.2%
1/16
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
|
43.8%
7/16
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.
|
|
Immune system disorders
Seasonal allergy
|
6.2%
1/16
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
Gastroenteritis
|
6.2%
1/16
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
Herpes simplex
|
6.2%
1/16
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
Hordeolum
|
6.2%
1/16
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
Infection
|
6.2%
1/16
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
|
25.0%
4/16
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
Pharyngitis
|
25.0%
4/16
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
Tonsillitis
|
6.2%
1/16
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
Viral infection
|
6.2%
1/16
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
Wound infection
|
6.2%
1/16
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
Concussion
|
6.2%
1/16
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
Contusion
|
6.2%
1/16
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
Fall
|
12.5%
2/16
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
Head injury
|
6.2%
1/16
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 injury
|
6.2%
1/16
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
|
12.5%
2/16
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
Post-traumatic pain
|
6.2%
1/16
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
Procedural complication
|
6.2%
1/16
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
Procedural pain
|
6.2%
1/16
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
Testicular injury
|
6.2%
1/16
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
Tibia fracture
|
6.2%
1/16
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
Wound
|
6.2%
1/16
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
Albumin urine present
|
6.2%
1/16
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 bilirubin increased
|
6.2%
1/16
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 pressure increased
|
6.2%
1/16
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
Body temperature increased
|
18.8%
3/16
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
Creatinine renal clearance decreased
|
6.2%
1/16
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
Heart rate increased
|
6.2%
1/16
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
Anorexia
|
12.5%
2/16
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
Arthralgia
|
25.0%
4/16
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
Back pain
|
12.5%
2/16
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
Chest wall pain
|
6.2%
1/16
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
Flank pain
|
6.2%
1/16
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
Groin pain
|
6.2%
1/16
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
|
6.2%
1/16
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
Myalgia
|
6.2%
1/16
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
|
25.0%
4/16
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
Shoulder pain
|
18.8%
3/16
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
Cognitive disorder
|
6.2%
1/16
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
Coordination abnormal
|
6.2%
1/16
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
|
25.0%
4/16
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
Dyspraxia
|
6.2%
1/16
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
|
62.5%
10/16
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
Hyperreflexia
|
6.2%
1/16
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
Lethargy
|
12.5%
2/16
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
Paraesthesia
|
37.5%
6/16
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
Tremor
|
6.2%
1/16
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.
|
|
Psychiatric disorders
Insomnia
|
6.2%
1/16
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.
|
|
Renal and urinary disorders
Microalbuminuria
|
6.2%
1/16
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
|
18.8%
3/16
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
|
12.5%
2/16
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
|
25.0%
4/16
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
Rhinitis allergic
|
12.5%
2/16
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
Rhinorrhoea
|
25.0%
4/16
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
Sneezing
|
6.2%
1/16
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
Throat irritation
|
6.2%
1/16
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
Angiokeratoma
|
18.8%
3/16
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
Hyperhidrosis
|
6.2%
1/16
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
Pruritus
|
6.2%
1/16
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
Rash
|
6.2%
1/16
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
Rash erythematous
|
6.2%
1/16
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
Rash generalised
|
6.2%
1/16
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
Urticaria localised
|
6.2%
1/16
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.
|
|
Vascular disorders
Hypotension
|
6.2%
1/16
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.
|
|
Vascular disorders
Pallor
|
6.2%
1/16
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.
|
|
Vascular disorders
Poor peripheral circulation
|
6.2%
1/16
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.
|
|
Vascular disorders
Vasoconstriction
|
6.2%
1/16
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
Genzyme Medical Information
Genzyme Corporation
Phone: 800-745-4447
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