Trial Outcomes & Findings for Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) (NCT NCT01515956)
NCT ID: NCT01515956
Last Updated: 2017-08-10
Results Overview
Number of Participants Experiencing Adverse Events
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
52 weeks
Results posted on
2017-08-10
Participant Flow
Participant milestones
| Measure |
BMN110 2.0 mg/kg/Week
Weekly intravenous infusions of BMN 110 at a dose of 2.0 mg/kg for 52 consecutive weeks. Each infusion will be administered over a period of approximately 4 hours.
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Baseline characteristics by cohort
| Measure |
BMN110 2.0 mg/kg/Week
n=15 Participants
2.0 mg/kg/week
|
|---|---|
|
Age, Continuous
|
3.1 years
STANDARD_DEVIATION 1.34 • n=5 Participants
|
|
Age, Customized
0 to <3 years
|
7 participants
n=5 Participants
|
|
Age, Customized
>=3 to <5 years
|
8 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
4 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
10 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
2 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
6 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=5 Participants
|
|
Urinary Keratan Sulfate Measures
|
35.9 ug/mg
STANDARD_DEVIATION 12.32 • n=5 Participants
|
PRIMARY outcome
Timeframe: 52 weeksPopulation: Safety Population
Number of Participants Experiencing Adverse Events
Outcome measures
| Measure |
BMN110 2.0 mg/kg/Week
n=15 Participants
BMN110 2.0 mg/kg/week
|
|---|---|
|
To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion
|
15 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 52Population: Efficacy Analysis Set - All enrolled patients with at least 1 post treatment efficacy measurement will be included in efficacy analyses.
Percent Change from Baseline to Week 52 for Urinary Keratan Sulfate measures.
Outcome measures
| Measure |
BMN110 2.0 mg/kg/Week
n=11 Participants
BMN110 2.0 mg/kg/week
|
|---|---|
|
Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures
|
-44.3 percentage change
Standard Deviation 21.15
|
SECONDARY outcome
Timeframe: Baseline to Week 52Population: Efficacy analysis set - All enrolled patients with at least 1 post treatment efficacy measurement will be included in efficacy analyses.
Changes in growth over time will be assessed using anthropometric measurements and radiographs of lower extremities. Z-scores are the normalized scores derived from the reference population mean and standard deviation (A positive change from baseline indicates that the population has moved closer to the reference population and represents a positive outcome).
Outcome measures
| Measure |
BMN110 2.0 mg/kg/Week
n=15 Participants
BMN110 2.0 mg/kg/week
|
|---|---|
|
Change From Baseline in Normalized Growth Rate Z-Scores
Baseline
|
-0.6 z-score
Standard Deviation 0.64
|
|
Change From Baseline in Normalized Growth Rate Z-Scores
Change from Baseline to Week 52
|
0.2 z-score
Standard Deviation 1.04
|
Adverse Events
BMN110 2.0 mg/kg/Week
Serious events: 8 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BMN110 2.0 mg/kg/Week
n=15 participants at risk
BMN110 2.0 mg/kg/week
|
|---|---|
|
Congenital, familial and genetic disorders
Developmental hip dysplasia
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Medical device complication
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Pyrexia
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Immune system disorders
Hypersensitivity
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Device related infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Lower respiratory tract infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Sepsis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Skin infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Joint instability
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Cerebrospinal fluid leakage
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Cervical cord compression
|
26.7%
4/15 • Number of events 4 • 52 weeks
|
|
Nervous system disorders
Spinal cord oedema
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar hypertrophy
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Vascular disorders
Poor venous access
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
Other adverse events
| Measure |
BMN110 2.0 mg/kg/Week
n=15 participants at risk
BMN110 2.0 mg/kg/week
|
|---|---|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Cardiac disorders
Tachycardia
|
26.7%
4/15 • Number of events 9 • 52 weeks
|
|
Congenital, familial and genetic disorders
Developmental hip dysplasia
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Ear and labyrinth disorders
Aural polyp
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Ear and labyrinth disorders
Cerumen impaction
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Ear and labyrinth disorders
Conductive deafness
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Ear and labyrinth disorders
Deafness
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Ear and labyrinth disorders
Deafness bilateral
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Ear and labyrinth disorders
Ear haemorrhage
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Ear and labyrinth disorders
Ear pain
|
20.0%
3/15 • Number of events 17 • 52 weeks
|
|
Ear and labyrinth disorders
Hearing impaired
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Ear and labyrinth disorders
Middle ear effusion
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Ear and labyrinth disorders
Motion sickness
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Ear and labyrinth disorders
Otorrhoea
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Eye disorders
Amblyopia
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Eye disorders
Astigmatism
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Eye disorders
Conjunctivitis
|
26.7%
4/15 • Number of events 5 • 52 weeks
|
|
Eye disorders
Conjunctivitis allergic
|
6.7%
1/15 • Number of events 3 • 52 weeks
|
|
Eye disorders
Corneal opacity
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Eye disorders
Eye discharge
|
6.7%
1/15 • Number of events 3 • 52 weeks
|
|
Eye disorders
Eye pain
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Eye disorders
Eye swelling
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Eye disorders
Keratitis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Eye disorders
Trichiasis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Abdominal distension
|
13.3%
2/15 • Number of events 4 • 52 weeks
|
|
Gastrointestinal disorders
Abdominal pain
|
53.3%
8/15 • Number of events 14 • 52 weeks
|
|
Gastrointestinal disorders
Abdominal pain upper
|
33.3%
5/15 • Number of events 14 • 52 weeks
|
|
Gastrointestinal disorders
Colitis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Constipation
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Gastrointestinal disorders
Dental caries
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
73.3%
11/15 • Number of events 24 • 52 weeks
|
|
Gastrointestinal disorders
Dyspepsia
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Flatulence
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Gastrointestinal disorders
Gastritis
|
13.3%
2/15 • Number of events 3 • 52 weeks
|
|
Gastrointestinal disorders
Gingival pain
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Gastrointestinal disorders
Mouth ulceration
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Gastrointestinal disorders
Nausea
|
26.7%
4/15 • Number of events 5 • 52 weeks
|
|
Gastrointestinal disorders
Oral discomfort
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Oral disorder
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Gastrointestinal disorders
Retching
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Gastrointestinal disorders
Toothache
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Gastrointestinal disorders
Vomiting
|
86.7%
13/15 • Number of events 59 • 52 weeks
|
|
General disorders
Catheter site extravasation
|
26.7%
4/15 • Number of events 6 • 52 weeks
|
|
General disorders
Catheter site pain
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Catheter site related reaction
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
General disorders
Chest pain
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Chills
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Crepitations
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Device difficult to use
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Fatigue
|
26.7%
4/15 • Number of events 6 • 52 weeks
|
|
General disorders
Gait disturbance
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Implant site haemorrhage
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Infusion site extravasation
|
13.3%
2/15 • Number of events 3 • 52 weeks
|
|
General disorders
Injection site hypersensitivity
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Local swelling
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Malaise
|
20.0%
3/15 • Number of events 4 • 52 weeks
|
|
General disorders
Mass
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Medical device complication
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Medical device pain
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Medical device site reaction
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
General disorders
Pain
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
General disorders
Pyrexia
|
100.0%
15/15 • Number of events 143 • 52 weeks
|
|
Hepatobiliary disorders
Hepatic function abnormal
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Hepatobiliary disorders
Portal vein thrombosis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Acute sinusitis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Acute tonsillitis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Anal fungal infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Bronchitis
|
20.0%
3/15 • Number of events 7 • 52 weeks
|
|
Infections and infestations
Conjunctivitis infective
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Diarrhoea infectious
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Ear infection
|
20.0%
3/15 • Number of events 4 • 52 weeks
|
|
Infections and infestations
Exanthema subitum
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Infections and infestations
Fungal skin infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Gastroenteritis
|
13.3%
2/15 • Number of events 3 • 52 weeks
|
|
Infections and infestations
Gastroenteritis viral
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Infections and infestations
Gingival infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Herpes simplex
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Infections and infestations
Hordeolum
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Implant site infection
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Infections and infestations
Influenza
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Infections and infestations
Lower respiratory tract infection
|
13.3%
2/15 • Number of events 4 • 52 weeks
|
|
Infections and infestations
Molluscum contagiosum
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Infections and infestations
Nasopharyngitis
|
60.0%
9/15 • Number of events 25 • 52 weeks
|
|
Infections and infestations
Oral candidiasis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Otitis externa
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Infections and infestations
Otitis media
|
33.3%
5/15 • Number of events 6 • 52 weeks
|
|
Infections and infestations
Otitis media acute
|
20.0%
3/15 • Number of events 6 • 52 weeks
|
|
Infections and infestations
Otitis media chronic
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Penile infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Pharyngitis
|
13.3%
2/15 • Number of events 3 • 52 weeks
|
|
Infections and infestations
Postoperative wound infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Rash pustular
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Rhinitis
|
60.0%
9/15 • Number of events 22 • 52 weeks
|
|
Infections and infestations
Skin infection
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Infections and infestations
Tonsillitis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Tooth infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
53.3%
8/15 • Number of events 52 • 52 weeks
|
|
Infections and infestations
Urinary tract infection
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Infections and infestations
Viral infection
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Infections and infestations
Viral rash
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Viral upper respiratory tract infection
|
33.3%
5/15 • Number of events 8 • 52 weeks
|
|
Injury, poisoning and procedural complications
Contusion
|
33.3%
5/15 • Number of events 5 • 52 weeks
|
|
Injury, poisoning and procedural complications
Eye contusion
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Injury, poisoning and procedural complications
Foot fracture
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Head injury
|
13.3%
2/15 • Number of events 3 • 52 weeks
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
13.3%
2/15 • Number of events 3 • 52 weeks
|
|
Injury, poisoning and procedural complications
Injury corneal
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Laceration
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Injury, poisoning and procedural complications
Limb crushing injury
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Lip injury
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Postoperative fever
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Procedural pain
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Injury, poisoning and procedural complications
Procedural vomiting
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Scar
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Spinal cord injury
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Thermal burn
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Investigations
Aspartate aminotransferase increased
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Investigations
Blood bicarbonate decreased
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Investigations
Body temperature increased
|
20.0%
3/15 • Number of events 5 • 52 weeks
|
|
Investigations
Breath sounds abnormal
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Investigations
Heart rate increased
|
20.0%
3/15 • Number of events 11 • 52 weeks
|
|
Investigations
Oxygen saturation decreased
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Investigations
Respiratory rate increased
|
13.3%
2/15 • Number of events 10 • 52 weeks
|
|
Investigations
Venous pressure decreased
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Metabolism and nutrition disorders
Decreased appetite
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
46.7%
7/15 • Number of events 15 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Limb discomfort
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
33.3%
5/15 • Number of events 15 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Spinal disorder
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Cerebral haematoma
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Dizziness
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Headache
|
33.3%
5/15 • Number of events 13 • 52 weeks
|
|
Nervous system disorders
Hypoaesthesia
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Psychiatric disorders
Agitation
|
13.3%
2/15 • Number of events 3 • 52 weeks
|
|
Renal and urinary disorders
Dysuria
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Renal and urinary disorders
Haematuria
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Renal and urinary disorders
Pollakiuria
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Renal and urinary disorders
Vesicoureteric reflux
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Reproductive system and breast disorders
Balanoposthitis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Reproductive system and breast disorders
Oedema genital
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Adenoidal hypertrophy
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Allergic respiratory disease
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
73.3%
11/15 • Number of events 39 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Increased upper airway secretion
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Laryngospasm
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
40.0%
6/15 • Number of events 15 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
26.7%
4/15 • Number of events 23 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
53.3%
8/15 • Number of events 20 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
20.0%
3/15 • Number of events 5 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar hypertrophy
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Blister
|
13.3%
2/15 • Number of events 3 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Cold sweat
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
13.3%
2/15 • Number of events 3 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Eczema
|
26.7%
4/15 • Number of events 5 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Erythema
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
26.7%
4/15 • Number of events 7 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
40.0%
6/15 • Number of events 9 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Skin reaction
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
26.7%
4/15 • Number of events 36 • 52 weeks
|
|
Surgical and medical procedures
Catheter placement
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Surgical and medical procedures
Central venous catheter removal
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Surgical and medical procedures
Central venous catheterisation
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Surgical and medical procedures
Dental care
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Surgical and medical procedures
Ear tube insertion
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Surgical and medical procedures
Ear tube removal
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Surgical and medical procedures
Infusion
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Surgical and medical procedures
Joint fluid drainage
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Surgical and medical procedures
Orchidopexy
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Vascular disorders
Flushing
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Vascular disorders
Hot flush
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Vascular disorders
Hyperaemia
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Vascular disorders
Hypotension
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Vascular disorders
Pallor
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Vascular disorders
Poor venous access
|
26.7%
4/15 • Number of events 6 • 52 weeks
|
Additional Information
Peter Slasor/Sr Director, Biostatistics, Global Clinical Sciences
BioMarin Pharmaceutical Inc.
Phone: 415-506-6765
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place