Trial Outcomes & Findings for Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation (NCT NCT01697319)
NCT ID: NCT01697319
Last Updated: 2016-01-12
Results Overview
FDT assesses the ability to use the hand in daily tasks. The test involves turning 16 wooden pegs over as quickly as possible on a hardwood pegboard with one hand requiring a three-jaw chuck prehension pattern between the fingers and thumb within a two-minute time limit. Hand function is evaluated by how fast a patient can turn over pegs in the given time limit, i.e. speed (number of pegs/minute).
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
16 participants
Primary outcome timeframe
Up to 96 weeks
Results posted on
2016-01-12
Participant Flow
Participant milestones
| Measure |
BMN 110 at 2.0 mg/kg/Week
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.
|
|---|---|
|
Primary Phase
STARTED
|
16
|
|
Primary Phase
COMPLETED
|
15
|
|
Primary Phase
NOT COMPLETED
|
1
|
|
Extension Phase
STARTED
|
15
|
|
Extension Phase
COMPLETED
|
0
|
|
Extension Phase
NOT COMPLETED
|
15
|
Reasons for withdrawal
| Measure |
BMN 110 at 2.0 mg/kg/Week
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.
|
|---|---|
|
Primary Phase
Withdrawal by Subject
|
1
|
|
Extension Phase
Terminated by Sponsor
|
15
|
Baseline Characteristics
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
Baseline characteristics by cohort
| Measure |
BMN 110 at 2.0 mg/kg/Week
n=16 Participants
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.
|
|---|---|
|
Age, Continuous
|
20.7 years
STANDARD_DEVIATION 8.38 • n=5 Participants
|
|
Age, Customized
5 to 11 years
|
1 participants
n=5 Participants
|
|
Age, Customized
12 to 18 years
|
8 participants
n=5 Participants
|
|
Age, Customized
>=19 years
|
7 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
2 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
1 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
12 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
6 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
4 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 96 weeksPopulation: Modified ITT Population
FDT assesses the ability to use the hand in daily tasks. The test involves turning 16 wooden pegs over as quickly as possible on a hardwood pegboard with one hand requiring a three-jaw chuck prehension pattern between the fingers and thumb within a two-minute time limit. Hand function is evaluated by how fast a patient can turn over pegs in the given time limit, i.e. speed (number of pegs/minute).
Outcome measures
| Measure |
BMN 110 at 2.0 mg/kg/Week
n=15 Participants
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.
|
|---|---|
|
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
Non-Dominant Hand Wk 96 % Chg from Baseline
|
-2.0 % of change
Standard Deviation 2.83
|
|
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
Non-Dominant Hand Wk 72 % Chg from Baseline
|
49.3 % of change
Standard Deviation 126.26
|
|
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
Dominant Hand Wk 24 % Chg from Baseline
|
1.8 % of change
Standard Deviation 45.60
|
|
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
Dominant Hand Wk 48 % Chg from Baseline
|
23.1 % of change
Standard Deviation 53.88
|
|
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
Dominant Hand Wk 72 % Chg from Baseline
|
37.2 % of change
Standard Deviation 54.94
|
|
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
Dominant Hand Wk 96 % Chg from Baseline
|
22.5 % of change
Standard Deviation 42.21
|
|
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
Non-Dominant Hand Wk 24 % Chg from Baseline
|
13.8 % of change
Standard Deviation 32.54
|
|
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
Non-Dominant Hand Wk 48 % Chg from Baseline
|
6.0 % of change
Standard Deviation 35.78
|
PRIMARY outcome
Timeframe: Up to 96 weeksPopulation: Modified ITT Population
A grip-strength dynamometer and a pinch meter were used to measure grip strength and pinch strength. The results report change from baseline in strength for dominant and non-dominant hand in a forearm and wrist supported position.
Outcome measures
| Measure |
BMN 110 at 2.0 mg/kg/Week
n=15 Participants
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.
|
|---|---|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Grip Dominant Hand Wk 48 Chg from Baseline
|
-0.3 kg
Standard Deviation 0.90
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Grip Dominant Hand Wk 72 Chg from Baseline
|
-0.1 kg
Standard Deviation 0.33
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Pinch Non-Dominant Hand Wk 96 Chg from Baseline
|
-0.3 kg
Standard Deviation 0.36
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Grip Dominant Hand Wk 24 Chg from Baseline
|
0.1 kg
Standard Deviation 0.89
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Grip Dominant Hand Wk 96 Chg from Baseline
|
0.3 kg
Standard Deviation 0.23
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Grip Non-Dominant Hand Wk24 Chg from Baseline
|
-0.0 kg
Standard Deviation 0.70
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Grip Non-Dominant Hand Wk 48 Chg from Baseline
|
-0.1 kg
Standard Deviation 0.66
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Grip Non-Dominant Hand Wk 72 Chg from Baseline
|
0.1 kg
Standard Deviation 0.79
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Grip Non-Dominant Hand Wk 96 Chg from Baseline
|
0.4 kg
Standard Deviation 0.87
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Pinch Dominant Hand Wk 24 Chg from Baseline
|
-0.1 kg
Standard Deviation 0.32
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Pinch Dominant Hand Wk 48 Chg from Baseline
|
-0.0 kg
Standard Deviation 0.30
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Pinch Dominant Hand Wk 72 Chg from Baseline
|
-0.1 kg
Standard Deviation 0.23
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Pinch Dominant Hand Wk 96 Chg from Baseline
|
-0.6 kg
Standard Deviation NA
One observation only
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Pinch Non-Dominant Hand Wk 24 Chg from Baseline
|
-0.0 kg
Standard Deviation 0.18
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Pinch Non-Dominant Hand Wk 48 Chg from Baseline
|
0.0 kg
Standard Deviation 0.27
|
|
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Pinch Non-Dominant Hand Wk 72 Chg from Baseline
|
-0.1 kg
Standard Deviation 0.31
|
PRIMARY outcome
Timeframe: Up to 96 weeksPopulation: Modified ITT Population
The timed 25-Foot Walk Test (25FWT) is an assessment of mobility and performance of leg function. The patient was instructed to walk a marked 25-foot course as quickly as possible in a time limit of 3 minutes and immediately walk back the same distance when reaching one end.The patient is allowed to use any ambulation method to move. The outcome measures the speed (feet / min) of moving.
Outcome measures
| Measure |
BMN 110 at 2.0 mg/kg/Week
n=15 Participants
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.
|
|---|---|
|
Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT)
Wk 24 % Change from Baseline Two Attempts Combined
|
95.7 % of change
Standard Deviation 142.37
|
|
Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT)
Wk 48 % Change from Baseline Two Attempts Combined
|
53.5 % of change
Standard Deviation 102.74
|
|
Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT)
Wk 72 % Change from Baseline Two Attempts Combined
|
48.3 % of change
Standard Deviation 145.01
|
|
Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT)
Wk 96 % Change from Baseline Two Attempts Combined
|
NA % of change
Standard Deviation NA
Small sample size and zero denominators
|
SECONDARY outcome
Timeframe: Up to 96 weeksPopulation: Modified ITT Population
Urinary keratan sulfate and urinary creatinine were measured through quantitative analysis. uKS is normalized to creatinine.
Outcome measures
| Measure |
BMN 110 at 2.0 mg/kg/Week
n=15 Participants
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.
|
|---|---|
|
Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS)
Week 48 % Change from Baseline
|
-43.4 % of change
Standard Deviation 24.82
|
|
Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS)
Week 24 % Change from Baseline
|
-47.7 % of change
Standard Deviation 15.40
|
|
Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS)
Week 72 % Change from Baseline
|
-45.5 % of change
Standard Deviation 19.67
|
|
Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS)
Week 96 % Change from Baseline
|
-48.3 % of change
Standard Deviation 24.13
|
Adverse Events
BMN 110 at 2.0 mg/kg/Week
Serious events: 7 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BMN 110 at 2.0 mg/kg/Week
n=15 participants at risk
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.
|
|---|---|
|
Immune system disorders
Hypersensitivity
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Infections and infestations
Lower respiratory tract infection
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Otitis media
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Pneumonia bacterial
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Pneumonia mycoplasmal
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Bone disorder
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Nervous system disorders
Cervical cord compression
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Psychiatric disorders
Sleep disorder
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
6.7%
1/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Surgical and medical procedures
Central venous catheterisation
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Vascular disorders
Poor venous access
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
Other adverse events
| Measure |
BMN 110 at 2.0 mg/kg/Week
n=15 participants at risk
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for an initial treatment phase of 48 weeks and an extension treatment phase of up to an additional 96 weeks.
|
|---|---|
|
Cardiac disorders
Atrioventricular block first degree
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Cardiac disorders
Sinus tachycardia
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Cardiac disorders
Tachycardia
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Ear and labyrinth disorders
Ear discomfort
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Ear and labyrinth disorders
Ear disorder
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Ear and labyrinth disorders
Ear pain
|
13.3%
2/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Ear and labyrinth disorders
Otorrhoea
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Eye disorders
Corneal opacity
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Eye disorders
Dry eye
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Eye disorders
Eye disorder
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Eye disorders
Eye pain
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Eye disorders
Eye pruritus
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Eye disorders
Eyelid cyst
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Eye disorders
Eyelid pain
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Eye disorders
Lacrimation increased
|
6.7%
1/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Gastrointestinal disorders
Abdominal discomfort
|
13.3%
2/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Gastrointestinal disorders
Abdominal pain
|
20.0%
3/15 • Number of events 7 • Study Period
All Adverse Events
|
|
Gastrointestinal disorders
Abdominal pain upper
|
40.0%
6/15 • Number of events 12 • Study Period
All Adverse Events
|
|
Gastrointestinal disorders
Constipation
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Gastrointestinal disorders
Diarrhoea
|
46.7%
7/15 • Number of events 28 • Study Period
All Adverse Events
|
|
Gastrointestinal disorders
Gastritis
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Gastrointestinal disorders
Nausea
|
46.7%
7/15 • Number of events 19 • Study Period
All Adverse Events
|
|
Gastrointestinal disorders
Vomiting
|
40.0%
6/15 • Number of events 29 • Study Period
All Adverse Events
|
|
General disorders
Catheter site pain
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
General disorders
Chest pain
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
General disorders
Device occlusion
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
General disorders
Extravasation
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
General disorders
Face oedema
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
General disorders
Fatigue
|
26.7%
4/15 • Number of events 5 • Study Period
All Adverse Events
|
|
General disorders
Feeling cold
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
General disorders
Influenza like illness
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
General disorders
Infusion site extravasation
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
General disorders
Infusion site pain
|
6.7%
1/15 • Number of events 3 • Study Period
All Adverse Events
|
|
General disorders
Infusion site reaction
|
6.7%
1/15 • Number of events 5 • Study Period
All Adverse Events
|
|
General disorders
Injection site pain
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
General disorders
Localised oedema
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
General disorders
Malaise
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
General disorders
Medical device pain
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
General disorders
Nodule
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
General disorders
Non-cardiac chest pain
|
13.3%
2/15 • Number of events 5 • Study Period
All Adverse Events
|
|
General disorders
Oedema peripheral
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
General disorders
Pain
|
26.7%
4/15 • Number of events 5 • Study Period
All Adverse Events
|
|
General disorders
Pyrexia
|
60.0%
9/15 • Number of events 26 • Study Period
All Adverse Events
|
|
Hepatobiliary disorders
Cholelithiasis
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Immune system disorders
Allergy to arthropod bite
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Immune system disorders
Anaphylactic reaction
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Immune system disorders
Anaphylactoid reaction
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Immune system disorders
Hypersensitivity
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Immune system disorders
Reaction to colouring
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Immune system disorders
Seasonal allergy
|
13.3%
2/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Infections and infestations
Bronchitis
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Infections and infestations
Cystitis
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Infections and infestations
Ear infection
|
13.3%
2/15 • Number of events 5 • Study Period
All Adverse Events
|
|
Infections and infestations
Eye infection
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Gastroenteritis
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Infections and infestations
Hordeolum
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Influenza
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Lower respiratory tract infection
|
20.0%
3/15 • Number of events 4 • Study Period
All Adverse Events
|
|
Infections and infestations
Nasopharyngitis
|
60.0%
9/15 • Number of events 19 • Study Period
All Adverse Events
|
|
Infections and infestations
Otitis externa fungal
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Otitis media
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Infections and infestations
Periodontitis
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Pulpitis dental
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Respiratory tract infection
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Rhinitis
|
13.3%
2/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Infections and infestations
Sinusitis
|
20.0%
3/15 • Number of events 4 • Study Period
All Adverse Events
|
|
Infections and infestations
Skin candida
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Subcutaneous abscess
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Infections and infestations
Upper respiratory tract infection
|
46.7%
7/15 • Number of events 14 • Study Period
All Adverse Events
|
|
Infections and infestations
Urinary tract infection
|
6.7%
1/15 • Number of events 6 • Study Period
All Adverse Events
|
|
Infections and infestations
Viral infection
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Injury, poisoning and procedural complications
Chillblains
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Injury, poisoning and procedural complications
Fall
|
13.3%
2/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Injury, poisoning and procedural complications
Heat exhaustion
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Injury, poisoning and procedural complications
Limb injury
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Injury, poisoning and procedural complications
Tooth fracture
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Investigations
Alanine aminotransferase increased
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Investigations
Blood alkaline phosphatase increased
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Investigations
Blood bilirubin increased
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Investigations
Blood glucose increased
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Investigations
Body temperature increased
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Investigations
Electrocardiogram PR prolongation
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Investigations
QRS axis abnormal
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Investigations
Weight increased
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Metabolism and nutrition disorders
Decreased appetite
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
33.3%
5/15 • Number of events 28 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
33.3%
5/15 • Number of events 16 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
13.3%
2/15 • Number of events 5 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
26.7%
4/15 • Number of events 8 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
33.3%
5/15 • Number of events 8 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Pubic pain
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Sensation of heaviness
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Tenosynovitis
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Nervous system disorders
Burning sensation
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Nervous system disorders
Clonus
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Nervous system disorders
Dizziness
|
26.7%
4/15 • Number of events 10 • Study Period
All Adverse Events
|
|
Nervous system disorders
Headache
|
73.3%
11/15 • Number of events 42 • Study Period
All Adverse Events
|
|
Nervous system disorders
Hypoaesthesia
|
6.7%
1/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Nervous system disorders
Lethargy
|
13.3%
2/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Nervous system disorders
Paraesthesia
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Nervous system disorders
Somnolence
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Psychiatric disorders
Anxiety
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Psychiatric disorders
Depressed mood
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Psychiatric disorders
Depression
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Psychiatric disorders
Restlessness
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Renal and urinary disorders
Dysuria
|
6.7%
1/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Renal and urinary disorders
Nephrolithiasis
|
6.7%
1/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
40.0%
6/15 • Number of events 15 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
13.3%
2/15 • Number of events 4 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
33.3%
5/15 • Number of events 10 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
26.7%
4/15 • Number of events 14 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
6.7%
1/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Restrictive pulmonary disease
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
20.0%
3/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
13.3%
2/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Acne
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Erythema
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Pigmentation disorder
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
6.7%
1/15 • Number of events 6 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Pruritus generalised
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Rash
|
20.0%
3/15 • Number of events 3 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Rash follicular
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Surgical and medical procedures
Tooth extraction
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Vascular disorders
Haematoma
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Vascular disorders
Hypotension
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Vascular disorders
Pallor
|
6.7%
1/15 • Number of events 1 • Study Period
All Adverse Events
|
|
Vascular disorders
Poor venous access
|
13.3%
2/15 • Number of events 2 • Study Period
All Adverse Events
|
Additional Information
BioMarin Medical Information Services
BioMarin Pharmaceutical Inc.
Phone: 800-983-4587
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60