Trial Outcomes & Findings for Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI (NCT NCT00067470)
NCT ID: NCT00067470
Last Updated: 2009-12-24
Results Overview
Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
39 participants
Primary outcome timeframe
baseline and 24 weeks
Results posted on
2009-12-24
Participant Flow
Study Locations:Oakland, CA, USA Mainz, Germany Manchester, England Porto Alegre, Brazil Lyon, France Porto, Portugal Study Duration: 24 weeks First enrollment: 21 July 2003 Last Dose Given: 1 April 2004 Last Assessment: 8 April 2004
Participant milestones
| Measure |
rhASB
Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
|
Placebo
Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB
|
|---|---|---|
|
Overall Study
STARTED
|
19
|
20
|
|
Overall Study
COMPLETED
|
19
|
19
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
rhASB
Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
|
Placebo
Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
Baseline characteristics by cohort
| Measure |
rhASB
n=19 Participants
Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
|
Placebo
n=20 Participants
Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB
|
Total
n=39 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
13.7 years
STANDARD_DEVIATION 6.47 • n=5 Participants
|
10.7 years
STANDARD_DEVIATION 4.35 • n=7 Participants
|
12.2 years
STANDARD_DEVIATION 5.62 • n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White, non-Hispanic
|
15 participants
n=5 Participants
|
9 participants
n=7 Participants
|
24 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
1 participants
n=5 Participants
|
3 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black
|
1 participants
n=5 Participants
|
2 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Indigenous
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 participants
n=5 Participants
|
4 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=5 Participants
|
4 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
4 participants
n=5 Participants
|
4 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
3 participants
n=5 Participants
|
3 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Brazil
|
4 participants
n=5 Participants
|
4 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Region of Enrollment
France
|
2 participants
n=5 Participants
|
3 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Region of Enrollment
Portugal
|
4 participants
n=5 Participants
|
2 participants
n=7 Participants
|
6 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: baseline and 24 weeksPopulation: The efficacy analysis included all subjects except one from the placebo group who withdrew from the study prior to the week 24 assessment.
Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
Outcome measures
| Measure |
rhASB
n=19 Participants
Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
|
Placebo
n=19 Participants
Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB
|
|---|---|---|
|
Change From Baseline in 12-minute Walk Test at 24 Weeks
|
109 meters
Standard Deviation 154
|
26 meters
Standard Deviation 122
|
SECONDARY outcome
Timeframe: baseline and 24 weeksPopulation: The efficacy analysis included all subjects except one from the placbo group who withdrew from the study prior to the week 24 assessment.
Number of stairs climbed per minute in 3 minutes at 24 weeks minus number of stairs climbed per minute in 3 minutes at baseline
Outcome measures
| Measure |
rhASB
n=19 Participants
Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
|
Placebo
n=19 Participants
Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB
|
|---|---|---|
|
Change From Baseline in 3-minute Stair Climb at 24 Weeks
|
7.4 stairs/min
Standard Deviation 9.9
|
2.7 stairs/min
Standard Deviation 6.9
|
SECONDARY outcome
Timeframe: baseline and 24 weeksPopulation: Baseline uGAG level was measured for 19 rhASB-treated subjects and 20 placebo-treated subjects. However, uGAG levels at 24 weeks were measured for 19 rhASB-treated subjects and 19 placebo-treated subjects, because 1 placebo-treated subject withdrew from the study before week 24.
Glycosaminoglycan (GAG) level measured in urine
Outcome measures
| Measure |
rhASB
n=19 Participants
Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
|
Placebo
n=19 Participants
Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB
|
|---|---|---|
|
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
24-week Urinary GAG Level
|
85 micrograms per mg creatinine
Standard Deviation 35
|
317 micrograms per mg creatinine
Standard Deviation 80
|
|
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
Baseline Urinary GAG Level
|
346 micrograms per mg creatinine
Standard Deviation 128
|
330 micrograms per mg creatinine
Standard Deviation 114
|
Adverse Events
rhASB
Serious events: 3 serious events
Other events: 19 other events
Deaths: 0 deaths
Placebo
Serious events: 4 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
rhASB
n=19 participants at risk
Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
|
Placebo
n=20 participants at risk
Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal Strangulated Hernia
|
0.00%
0/19
|
10.0%
2/20 • Number of events 2
|
|
Infections and infestations
Apnoea
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Gastrointestinal disorders
Cardiac failure congestive
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Convulsion
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Nervous system disorders
Corneal lesion
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Headache
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Ileus
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Nervous system disorders
Intracranial pressure increased
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Cardiac disorders
Obstructive airways disorder
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Pneumonia
|
0.00%
0/19
|
15.0%
3/20 • Number of events 3
|
|
Eye disorders
Viral infection
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
Other adverse events
| Measure |
rhASB
n=19 participants at risk
Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
|
Placebo
n=20 participants at risk
Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
5.3%
1/19 • Number of events 1
|
10.0%
2/20 • Number of events 2
|
|
Gastrointestinal disorders
Abdominal pain
|
42.1%
8/19 • Number of events 8
|
30.0%
6/20 • Number of events 6
|
|
Gastrointestinal disorders
Abdominal pain lower
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Gastrointestinal disorders
Abdominal pain upper
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
Gastrointestinal disorders
Abdominal strangulated hernia
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Abscess oral
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Skin and subcutaneous tissue disorders
Acne
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Infections and infestations
Acute tonsillitis
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Adenoidal hypertrophy
|
10.5%
2/19 • Number of events 2
|
5.0%
1/20 • Number of events 1
|
|
Psychiatric disorders
Adjustment disorder
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Psychiatric disorders
Agitation
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
15.8%
3/19 • Number of events 3
|
15.0%
3/20 • Number of events 3
|
|
Blood and lymphatic system disorders
Anaemia
|
5.3%
1/19 • Number of events 1
|
15.0%
3/20 • Number of events 3
|
|
General disorders
Anasarca
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Psychiatric disorders
Anxiety
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
General disorders
Application site erythema
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Nervous system disorders
Areflexia
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
42.1%
8/19 • Number of events 8
|
35.0%
7/20 • Number of events 7
|
|
General disorders
Asthenia
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
21.1%
4/19 • Number of events 4
|
20.0%
4/20 • Number of events 4
|
|
Renal and urinary disorders
Bladder pain
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Injury, poisoning and procedural complications
Blister
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Investigations
Blood phosphorus increased
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Investigations
Blood pressure increased
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
General disorders
Bloody discharge
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial obstruction
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Bronchitis
|
0.00%
0/19
|
10.0%
2/20 • Number of events 2
|
|
Infections and infestations
Bronchopneumonia
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Cardiac disorders
Cardiac disorder
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Cardiac disorders
Cardiac failure
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Cardiac disorders
Cardiac valve disease
|
0.00%
0/19
|
10.0%
2/20 • Number of events 2
|
|
Cardiac disorders
Cardiomegaly
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Catarrh
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Ear and labyrinth disorders
Cerumen impaction
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Nervous system disorders
Cervical myelopathy
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
General disorders
Chest pain
|
15.8%
3/19 • Number of events 3
|
5.0%
1/20 • Number of events 1
|
|
Nervous system disorders
Clonus
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Cardiac disorders
Conduction disorder
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Psychiatric disorders
Confusional state
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Eye disorders
Conjunctivitis
|
21.1%
4/19 • Number of events 4
|
0.00%
0/20
|
|
Gastrointestinal disorders
Constipation
|
5.3%
1/19 • Number of events 1
|
10.0%
2/20 • Number of events 2
|
|
Nervous system disorders
Convulsion
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Eye disorders
Corneal opacity
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
26.3%
5/19 • Number of events 5
|
30.0%
6/20 • Number of events 6
|
|
Infections and infestations
Dental caries
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Psychiatric disorders
Depression
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhea
|
15.8%
3/19 • Number of events 3
|
30.0%
6/20 • Number of events 6
|
|
Nervous system disorders
Dizziness
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Gastrointestinal disorders
Dysphagia
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
21.1%
4/19 • Number of events 4
|
10.0%
2/20 • Number of events 2
|
|
Renal and urinary disorders
Dysuria
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Infections and infestations
Ear infection
|
10.5%
2/19 • Number of events 2
|
10.0%
2/20 • Number of events 2
|
|
Ear and labyrinth disorders
Ear pain
|
42.1%
8/19 • Number of events 8
|
20.0%
4/20 • Number of events 4
|
|
Skin and subcutaneous tissue disorders
Eczema
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Nervous system disorders
Epilepsy
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Exanthem
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Eye disorders
Eye discharge
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Eye disorders
Eye irritation
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Eye disorders
Eye pain
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Eye disorders
Eye pruritus
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Eye disorders
Eyelid oedema
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Face oedema
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
General disorders
Fatigue
|
10.5%
2/19 • Number of events 2
|
15.0%
3/20 • Number of events 3
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Folliculitis
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Fungal skin infection
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Furuncle
|
5.3%
1/19 • Number of events 1
|
10.0%
2/20 • Number of events 2
|
|
General disorders
Gait abnormal
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Gastroenteritis
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
Gastrointestinal disorders
Gingivitis
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Eye disorders
Glaucoma
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
General disorders
Granuloma
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Vascular disorders
Haematoma
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Nervous system disorders
Headache
|
42.1%
8/19 • Number of events 8
|
60.0%
12/20 • Number of events 12
|
|
Ear and labyrinth disorders
Hearing impaired
|
10.5%
2/19 • Number of events 2
|
5.0%
1/20 • Number of events 1
|
|
Hepatobiliary disorders
Hepatomegaly
|
0.00%
0/19
|
15.0%
3/20 • Number of events 3
|
|
Hepatobiliary disorders
Hepatosplenomegaly
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
General disorders
Hernia pain
|
10.5%
2/19 • Number of events 2
|
15.0%
3/20 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Musculoskeletal and connective tissue disorders
Hip deformity
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Hordeolum
|
5.3%
1/19 • Number of events 1
|
10.0%
2/20 • Number of events 2
|
|
Reproductive system and breast disorders
Hydrocele
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Vascular disorders
Hyperaemia
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Vascular disorders
Hypertension
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
General disorders
Hyperthermia
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Ear and labyrinth disorders
Hypoacusis
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Vascular disorders
Hypotension
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Metabolism and nutrition disorders
Hypovolaemia
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Infection
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
General disorders
Influenza like illness
|
15.8%
3/19 • Number of events 3
|
15.0%
3/20 • Number of events 3
|
|
General disorders
Infusion site pain
|
5.3%
1/19 • Number of events 1
|
10.0%
2/20 • Number of events 2
|
|
Gastrointestinal disorders
Intestinal spasm
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Investigations
Intraocular pressure increased
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Joint Stiffness
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Eye disorders
Keratoconjunctivitis sicca
|
0.00%
0/19
|
10.0%
2/20 • Number of events 2
|
|
Eye disorders
Lacrimation increased
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
General disorders
Localised oedema
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Nervous system disorders
Loss of consciousness
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Infections and infestations
Lower respiratory tract infection
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
General disorders
Malaise
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
General disorders
Mass
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Mastoiditis
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Nervous system disorders
Migraine
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Cardiac disorders
Mitral valve stenosis
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Muscle cramp
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.5%
2/19 • Number of events 2
|
5.0%
1/20 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Nail discolouration
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
Infections and infestations
Nasopharyngitis
|
5.3%
1/19 • Number of events 1
|
25.0%
5/20 • Number of events 5
|
|
Gastrointestinal disorders
Nausea
|
21.1%
4/19 • Number of events 4
|
25.0%
5/20 • Number of events 5
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
10.5%
2/19 • Number of events 2
|
15.0%
3/20 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Obstructive airways disorder
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
General disorders
Oedema
|
0.00%
0/19
|
10.0%
2/20 • Number of events 2
|
|
General disorders
Oedema peripheral
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Oral Candidiasis
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Otitis externa
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Infections and infestations
Otitis media
|
26.3%
5/19 • Number of events 5
|
20.0%
4/20 • Number of events 4
|
|
Infections and infestations
Otitis media acute
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Ear and labyrinth disorders
Otorrhoea
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
Investigations
Oxygen saturation decreased
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
General disorders
Pain
|
26.3%
5/19 • Number of events 5
|
5.0%
1/20 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.5%
2/19 • Number of events 2
|
40.0%
8/20 • Number of events 8
|
|
Nervous system disorders
Paraesthesia
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Nervous system disorders
Paresis
|
5.3%
1/19 • Number of events 1
|
10.0%
2/20 • Number of events 2
|
|
Vascular disorders
Peripheral coldness
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Vascular disorders
Petechiae
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Infections and infestations
Pharyngitis
|
15.8%
3/19 • Number of events 3
|
5.0%
1/20 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Infections and infestations
Pneumonia
|
0.00%
0/19
|
20.0%
4/20 • Number of events 4
|
|
Vascular disorders
Poor venous access
|
5.3%
1/19 • Number of events 1
|
15.0%
3/20 • Number of events 3
|
|
Injury, poisoning and procedural complications
Post procedural pain
|
5.3%
1/19 • Number of events 1
|
10.0%
2/20 • Number of events 2
|
|
Renal and urinary disorders
Proteinuria
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
15.8%
3/19 • Number of events 3
|
15.0%
3/20 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
General disorders
Pyrexia
|
42.1%
8/19 • Number of events 8
|
40.0%
8/20 • Number of events 8
|
|
Skin and subcutaneous tissue disorders
Rash
|
15.8%
3/19 • Number of events 3
|
10.0%
2/20 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Infections and infestations
Respiratory tract infection viral
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Restrictive pulmonary disease
|
10.5%
2/19 • Number of events 2
|
15.0%
3/20 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/19
|
20.0%
4/20 • Number of events 4
|
|
General disorders
Rigors
|
21.1%
4/19 • Number of events 4
|
0.00%
0/20
|
|
Infections and infestations
Sinusitis
|
0.00%
0/19
|
20.0%
4/20 • Number of events 4
|
|
Skin and subcutaneous tissue disorders
Skin reaction
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Nervous system disorders
Sleep apnoea syndrome
|
5.3%
1/19 • Number of events 1
|
10.0%
2/20 • Number of events 2
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Nervous system disorders
Somnolence
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Nervous system disorders
Spinal cord compression
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Blood and lymphatic system disorders
Splenomegaly
|
0.00%
0/19
|
10.0%
2/20 • Number of events 2
|
|
Investigations
Sputum abnormal
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
General disorders
Suprapubic pain
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Cardiac disorders
Tachycardia
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
General disorders
Thirst
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Psychiatric disorders
Tic
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Gastrointestinal disorders
Tongue oedema
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar hypertrophy
|
10.5%
2/19 • Number of events 2
|
5.0%
1/20 • Number of events 1
|
|
Gastrointestinal disorders
Tooth Disorder
|
0.00%
0/19
|
10.0%
2/20 • Number of events 2
|
|
Gastrointestinal disorders
Tooth discoloration
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Infections and infestations
Tracheitis
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Ear and labyrinth disorders
Tympanic membrane hyperaemia
|
5.3%
1/19 • Number of events 1
|
5.0%
1/20 • Number of events 1
|
|
Gastrointestinal disorders
Umbilical hernia
|
10.5%
2/19 • Number of events 2
|
0.00%
0/20
|
|
Infections and infestations
Upper respiratory tract infection
|
21.1%
4/19 • Number of events 4
|
35.0%
7/20 • Number of events 7
|
|
Renal and urinary disorders
Urinary incontinence
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Investigations
Urine output decreased
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
5.3%
1/19 • Number of events 1
|
0.00%
0/20
|
|
Infections and infestations
Vaginitis
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Cardiac disorders
Ventricular hypertrophy
|
0.00%
0/19
|
5.0%
1/20 • Number of events 1
|
|
Eye disorders
Visual acuity reduced
|
5.3%
1/19 • Number of events 1
|
10.0%
2/20 • Number of events 2
|
|
Gastrointestinal disorders
Vomiting
|
31.6%
6/19 • Number of events 6
|
35.0%
7/20 • Number of events 7
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
10.5%
2/19 • Number of events 2
|
5.0%
1/20 • Number of events 1
|
Additional Information
Medical Information Services
BioMarin Pharmaceutical Inc.
Phone: 800-983-4587
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee The first publication of the results shall be made by Sponsor in a joint publication. If such a multi-center publication is not submitted within 12 months after conclusion of the study, the PI may publish the results from their site individually, subject however to comply with the other terms of the agreement.
- Publication restrictions are in place
Restriction type: OTHER