Trial Outcomes & Findings for Study of rhASB in Patients With Mucopolysaccharidosis VI (NCT NCT00104234)
NCT ID: NCT00104234
Last Updated: 2010-02-02
Results Overview
Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
39 participants
Primary outcome timeframe
Baseline of ASB-03-05 through week 96 of ASB-03-06
Results posted on
2010-02-02
Participant Flow
Global study sites were hospitals and medical clinics. First Enrollment: 05FEB04 Last Dose: 28SEP06
Participant milestones
| Measure |
rhASB/rhASB
Patients received rhASB in the double-blind study(ASB-03-05, NCT00067470)and continued to receive rhASB in the extension study(ASB-03-06).
|
Placebo/rhASB
Patients received placebo in the double-blind study (ASB-03-05, NCT00067470)then received rhASB in the extension study (ASB-03-06).
|
|---|---|---|
|
Overall Study
STARTED
|
19
|
20
|
|
Overall Study
COMPLETED
|
19
|
19
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of rhASB in Patients With Mucopolysaccharidosis VI
Baseline characteristics by cohort
| Measure |
rhASB/rhASB
n=19 Participants
Patients received rhASB in the double-blind study(ASB-03-05, NCT00067470)and continued to receive rhASB in the extension study(ASB-03-06).
|
Placebo/rhASB
n=20 Participants
Patients received placebo in the double-blind study (ASB-03-05, NCT00067470)then received rhASB in the extension study (ASB-03-06).
|
Total
n=39 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
Age
|
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 of ASB-03-05 through week 96 of ASB-03-06Population: The efficacy analysis included all subjects who continued in the extension study (ASB-03-06) except the 1 subject from the rhASB/rhASB group and 1 subject from the placebo/rhASB group who missed the Week 96 measurement.
Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.
Outcome measures
| Measure |
rhASB/rhASB
n=18 Participants
Patients received rhASB in the double-blind study(ASB-03-05, NCT00067470)and continued to receive rhASB in the extension study(ASB-03-06).
|
Placebo/rhASB
n=18 Participants
Patients received placebo in the double-blind study (ASB-03-05, NCT00067470)then received rhASB in the extension study (ASB-03-06).
|
|---|---|---|
|
12-Minute Walk Test
|
187 meters
Standard Deviation 187
|
118 meters
Standard Deviation 127
|
SECONDARY outcome
Timeframe: Baseline ASB-03-05 through week 96 of ASB-03-06.Population: The efficacy analysis included all subjects who continued in the extension study (ASB-03-06) except 1 subject from the placebo/rhASB group who missed the Week 96 measurement.
Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group.
Outcome measures
| Measure |
rhASB/rhASB
n=19 Participants
Patients received rhASB in the double-blind study(ASB-03-05, NCT00067470)and continued to receive rhASB in the extension study(ASB-03-06).
|
Placebo/rhASB
n=18 Participants
Patients received placebo in the double-blind study (ASB-03-05, NCT00067470)then received rhASB in the extension study (ASB-03-06).
|
|---|---|---|
|
3-Minute Stair Climb
|
13.1 stairs/min
Standard Deviation 16.1
|
11.1 stairs/min
Standard Deviation 10.0
|
SECONDARY outcome
Timeframe: 72 weeksPopulation: rhASB/rhASB and placebo/rhASB groups were combined for the analysis, representing 72 weeks of rhASB treatment in each group.
Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24.
Outcome measures
| Measure |
rhASB/rhASB
n=37 Participants
Patients received rhASB in the double-blind study(ASB-03-05, NCT00067470)and continued to receive rhASB in the extension study(ASB-03-06).
|
Placebo/rhASB
Patients received placebo in the double-blind study (ASB-03-05, NCT00067470)then received rhASB in the extension study (ASB-03-06).
|
|---|---|---|
|
Change in Urinary Glycosaminoglycans (GAG) Level
|
-231 microgram/mg creatinine
Standard Deviation 91
|
—
|
Adverse Events
All Participants
Serious events: 14 serious events
Other events: 38 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Participants
n=38 participants at risk
rhASB/rhASB and placebo/rhASB groups were combined for analysis.
|
|---|---|
|
Cardiac disorders
Cardiac failure
|
5.3%
2/38 • Number of events 2
|
|
Nervous system disorders
Cervical cord compression
|
2.6%
1/38 • Number of events 1
|
|
Investigations
Body increased temperature
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Broncopneumopathy
|
13.2%
5/38 • Number of events 5
|
|
Nervous system disorders
Cervical myelopathy
|
5.3%
2/38 • Number of events 2
|
|
Nervous system disorders
Convulsion
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Corneal Abscess
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.6%
1/38 • Number of events 1
|
|
General disorders
Cyst
|
2.6%
1/38 • Number of events 1
|
|
Nervous system disorders
Dizziness
|
2.6%
1/38 • Number of events 1
|
|
Vascular disorders
Haemorrhage
|
5.3%
2/38 • Number of events 2
|
|
Congenital, familial and genetic disorders
Hip Dysplasia
|
2.6%
1/38 • Number of events 1
|
|
Surgical and medical procedures
Hip surgery
|
2.6%
1/38 • Number of events 1
|
|
Gastrointestinal disorders
Inguinal hernia
|
2.6%
1/38 • Number of events 1
|
|
Gastrointestinal disorders
Inguinal hernia obstructive
|
2.6%
1/38 • Number of events 1
|
|
Nervous system disorders
Intracranial pressure increased
|
2.6%
1/38 • Number of events 1
|
|
Investigations
Intraocular pressure increased
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Mastoiditis
|
2.6%
1/38 • Number of events 1
|
|
Cardiac disorders
Mitral valve stenosis
|
2.6%
1/38 • Number of events 1
|
|
Surgical and medical procedures
Osteotomy
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Pneumonia
|
5.3%
2/38 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
5.3%
2/38 • Number of events 2
|
|
Nervous system disorders
Spinal cord compression
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Staphylococcal sepsis
|
2.6%
1/38 • Number of events 1
|
|
Nervous system disorders
Tension headache
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Tracheal stenosis
|
2.6%
1/38 • Number of events 1
|
|
Ear and labyrinth disorders
Vertigo
|
2.6%
1/38 • Number of events 1
|
Other adverse events
| Measure |
All Participants
n=38 participants at risk
rhASB/rhASB and placebo/rhASB groups were combined for analysis.
|
|---|---|
|
Injury, poisoning and procedural complications
Excoriation
|
5.3%
2/38 • Number of events 2
|
|
Injury, poisoning and procedural complications
Fall
|
5.3%
2/38 • Number of events 2
|
|
Injury, poisoning and procedural complications
Joint injury
|
5.3%
2/38 • Number of events 2
|
|
Vascular disorders
Hypotension
|
10.5%
4/38 • Number of events 4
|
|
Vascular disorders
Poor venous access
|
10.5%
4/38 • Number of events 4
|
|
Vascular disorders
Haematoma
|
5.3%
2/38 • Number of events 2
|
|
Cardiac disorders
Cardiac valve disease
|
13.2%
5/38 • Number of events 5
|
|
Vascular disorders
Hyperaemia
|
5.3%
2/38 • Number of events 2
|
|
Vascular disorders
Hypertension
|
5.3%
2/38 • Number of events 2
|
|
Cardiac disorders
Atrial hypertrophy
|
5.3%
2/38 • Number of events 2
|
|
Cardiac disorders
Cardiac failure
|
5.3%
2/38 • Number of events 2
|
|
Cardiac disorders
Cyanosis
|
5.3%
2/38 • Number of events 2
|
|
Cardiac disorders
Dilatation atrial
|
5.3%
2/38 • Number of events 2
|
|
Investigations
Neutrophil count increased
|
7.9%
3/38 • Number of events 3
|
|
Investigations
QRS axis abnormal
|
7.9%
3/38 • Number of events 3
|
|
Investigations
Blood alkaline phosphatase increased
|
5.3%
2/38 • Number of events 2
|
|
Investigations
Cardiac murmur
|
5.3%
2/38 • Number of events 2
|
|
Investigations
Oxygen saturation decreased
|
5.3%
2/38 • Number of events 2
|
|
Blood and lymphatic system disorders
Anaemia
|
7.9%
3/38 • Number of events 3
|
|
Infections and infestations
Ear infection
|
44.7%
17/38 • Number of events 17
|
|
Infections and infestations
Nasopharyngitis
|
42.1%
16/38 • Number of events 16
|
|
Infections and infestations
Upper respiratory tract infection
|
39.5%
15/38 • Number of events 15
|
|
Infections and infestations
Gastroenteritis
|
31.6%
12/38 • Number of events 12
|
|
Infections and infestations
Bronchitis
|
23.7%
9/38 • Number of events 9
|
|
Infections and infestations
Otitis externa
|
21.1%
8/38 • Number of events 8
|
|
Infections and infestations
Influenza
|
18.4%
7/38 • Number of events 7
|
|
Infections and infestations
Otitis media acute
|
18.4%
7/38 • Number of events 7
|
|
Infections and infestations
Sinusitis
|
15.8%
6/38 • Number of events 6
|
|
Infections and infestations
Hordeolum
|
13.2%
5/38 • Number of events 5
|
|
Infections and infestations
Pharyngitis
|
13.2%
5/38 • Number of events 5
|
|
Infections and infestations
Laryngitis
|
10.5%
4/38 • Number of events 4
|
|
Infections and infestations
Pneumonia
|
10.5%
4/38 • Number of events 4
|
|
Infections and infestations
Dental Caries
|
7.9%
3/38 • Number of events 3
|
|
Infections and infestations
Respiratory tract infection
|
7.9%
3/38 • Number of events 3
|
|
Infections and infestations
Tinea pedis
|
7.9%
3/38 • Number of events 3
|
|
Infections and infestations
Otitis media
|
15.8%
6/38 • Number of events 6
|
|
Infections and infestations
Varicella
|
7.9%
3/38 • Number of events 3
|
|
Infections and infestations
Bronchopneumonia
|
5.3%
2/38 • Number of events 2
|
|
Infections and infestations
Folliculitis
|
5.3%
2/38 • Number of events 2
|
|
Infections and infestations
Fungal infection
|
5.3%
2/38 • Number of events 2
|
|
Infections and infestations
Furuncle
|
5.3%
2/38 • Number of events 2
|
|
Infections and infestations
Lower respiratory tract infection
|
5.3%
2/38 • Number of events 2
|
|
Infections and infestations
Subcutaneous abscess
|
5.3%
2/38 • Number of events 2
|
|
Infections and infestations
Tonsillitis
|
5.3%
2/38 • Number of events 2
|
|
Infections and infestations
Tooth abscess
|
5.3%
2/38 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
73.7%
28/38 • Number of events 28
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
34.2%
13/38 • Number of events 13
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
21.1%
8/38 • Number of events 8
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
18.4%
7/38 • Number of events 7
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
15.8%
6/38 • Number of events 6
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
15.8%
6/38 • Number of events 6
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertention
|
13.2%
5/38 • Number of events 5
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis
|
10.5%
4/38 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
10.5%
4/38 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
7.9%
3/38 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Restrictive pulmonary disease
|
7.9%
3/38 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Apnoea
|
5.3%
2/38 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Chest pain
|
5.3%
2/38 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.3%
2/38 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Increased bronchial secretion
|
5.3%
2/38 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal swelling
|
5.3%
2/38 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
5.3%
2/38 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
5.3%
2/38 • Number of events 2
|
|
Gastrointestinal disorders
Diarrhoea
|
55.3%
21/38 • Number of events 21
|
|
Gastrointestinal disorders
Vomiting
|
47.4%
18/38 • Number of events 18
|
|
Gastrointestinal disorders
Abdominal pain
|
39.5%
15/38 • Number of events 15
|
|
Gastrointestinal disorders
Nausea
|
28.9%
11/38 • Number of events 11
|
|
Gastrointestinal disorders
Abdominal pain upper
|
18.4%
7/38 • Number of events 7
|
|
Gastrointestinal disorders
Constipation
|
15.8%
6/38 • Number of events 6
|
|
Gastrointestinal disorders
Toothache
|
10.5%
4/38 • Number of events 4
|
|
Gastrointestinal disorders
Dyspepsia
|
7.9%
3/38 • Number of events 3
|
|
Gastrointestinal disorders
Abdominal distension
|
5.3%
2/38 • Number of events 2
|
|
Gastrointestinal disorders
Inguinal hernia
|
5.3%
2/38 • Number of events 2
|
|
Gastrointestinal disorders
Mouth ulceration
|
5.3%
2/38 • Number of events 2
|
|
Gastrointestinal disorders
Stomatitis
|
5.3%
2/38 • Number of events 2
|
|
Nervous system disorders
Headache
|
71.1%
27/38 • Number of events 27
|
|
Nervous system disorders
Dizziness
|
13.2%
5/38 • Number of events 5
|
|
Nervous system disorders
Paraesthesia
|
10.5%
4/38 • Number of events 4
|
|
Nervous system disorders
Hyperreflexia
|
7.9%
3/38 • Number of events 3
|
|
Nervous system disorders
Hypoaesthenia
|
7.9%
3/38 • Number of events 3
|
|
Nervous system disorders
Tremor
|
7.9%
3/38 • Number of events 3
|
|
Nervous system disorders
Cervical myelopathy
|
5.3%
2/38 • Number of events 2
|
|
Nervous system disorders
Migrane
|
5.3%
2/38 • Number of events 2
|
|
Nervous system disorders
Somnolence
|
5.3%
2/38 • Number of events 2
|
|
General disorders
Pyrexia
|
63.2%
24/38 • Number of events 24
|
|
General disorders
Fatigue
|
21.1%
8/38 • Number of events 8
|
|
General disorders
Influenza like illness
|
21.1%
8/38 • Number of events 8
|
|
General disorders
Chest pain
|
18.4%
7/38 • Number of events 7
|
|
General disorders
Pain
|
10.5%
4/38 • Number of events 4
|
|
General disorders
Hernia pain
|
7.9%
3/38 • Number of events 3
|
|
General disorders
Infusion site pain
|
7.9%
3/38 • Number of events 3
|
|
General disorders
Oedema peripheral
|
7.9%
3/38 • Number of events 3
|
|
General disorders
Rigors
|
7.9%
3/38 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Athralgia
|
50.0%
19/38 • Number of events 19
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
47.4%
18/38 • Number of events 18
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
39.5%
15/38 • Number of events 15
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
10.5%
4/38 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Joint stiffness
|
10.5%
4/38 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.5%
4/38 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Hip deformity
|
7.9%
3/38 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Groin pain
|
5.3%
2/38 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Intervertibral disc disorder
|
5.3%
2/38 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Joint contracture
|
5.3%
2/38 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Kyphoscoliosis
|
5.3%
2/38 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
5.3%
2/38 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
21.1%
8/38 • Number of events 8
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
18.4%
7/38 • Number of events 7
|
|
Skin and subcutaneous tissue disorders
Rash
|
13.2%
5/38 • Number of events 5
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
10.5%
4/38 • Number of events 4
|
|
Skin and subcutaneous tissue disorders
Erythema
|
7.9%
3/38 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
Prurigo
|
7.9%
3/38 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
Acne
|
5.3%
2/38 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Eczema
|
5.3%
2/38 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Exanthem
|
5.3%
2/38 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Face oedema
|
5.3%
2/38 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Pityriasis rosea
|
5.3%
2/38 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
5.3%
2/38 • Number of events 2
|
|
Eye disorders
Conjunctivitis
|
18.4%
7/38 • Number of events 7
|
|
Eye disorders
Eye irritation
|
10.5%
4/38 • Number of events 4
|
|
Eye disorders
Eye pain
|
10.5%
4/38 • Number of events 4
|
|
Eye disorders
Eye redness
|
10.5%
4/38 • Number of events 4
|
|
Eye disorders
Visual acuity reduced
|
10.5%
4/38 • Number of events 4
|
|
Eye disorders
Eyelid oedema
|
5.3%
2/38 • Number of events 2
|
|
Eye disorders
Glaucoma
|
5.3%
2/38 • Number of events 2
|
|
Eye disorders
Keratoconjunctivitis sicca
|
5.3%
2/38 • Number of events 2
|
|
Ear and labyrinth disorders
Ear pain
|
34.2%
13/38 • Number of events 13
|
|
Ear and labyrinth disorders
Hypoacusis
|
18.4%
7/38 • Number of events 7
|
|
Ear and labyrinth disorders
Otorrhoea
|
18.4%
7/38 • Number of events 7
|
|
Ear and labyrinth disorders
Cerumen impaction
|
7.9%
3/38 • Number of events 3
|
|
Injury, poisoning and procedural complications
Post procedural pain
|
26.3%
10/38 • Number of events 10
|
|
Injury, poisoning and procedural complications
Head injury
|
10.5%
4/38 • Number of events 4
|
|
Injury, poisoning and procedural complications
Procedural site reaction
|
7.9%
3/38 • Number of events 3
|
|
Blood and lymphatic system disorders
Leukopenia
|
5.3%
2/38 • Number of events 2
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
7.9%
3/38 • Number of events 3
|
|
Metabolism and nutrition disorders
Iron deficiency
|
5.3%
2/38 • Number of events 2
|
|
Psychiatric disorders
Abnormal behavior
|
5.3%
2/38 • Number of events 2
|
|
Psychiatric disorders
Anxiety
|
5.3%
2/38 • Number of events 2
|
|
Renal and urinary disorders
Dysuria
|
7.9%
3/38 • Number of events 3
|
|
Hepatobiliary disorders
Hepatomegaly
|
5.3%
2/38 • Number of events 2
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin Papilloma
|
5.3%
2/38 • Number of events 2
|
|
Surgical and medical procedures
Tooth extraction
|
5.3%
2/38 • Number of events 2
|
Additional Information
Medical Information Services
BioMarin Pharmaceutical Inc.
Phone: 8009834587
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 multicenter 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