A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

• ClinicalTrials.gov Identifier: NCT00884949
• Recruitment Status: Completed
• First Posted: April 21, 2009
• Results First Posted: June 30, 2014
• Last Update Posted: June 30, 2014
• Sponsor: BioMarin Pharmaceutical
• Information provided by (Responsible Party): BioMarin Pharmaceutical

• Study Type: Interventional
• Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: MPS IV A
• Intervention: Drug: BMN 110
• Enrollment: 20

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	BMN 110
Arm/Group Description	Dose-Escalation Period: Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen: Weeks 1-12: 0.1 mg/kg/week; Weeks 13-24: 1.0 mg/kg/week; Weeks 25-36: 2.0 mg/kg/week Continuation Period: Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.
Period Title: Weeks 1-12: 0.1 mg/kg/Week
Started	20
Completed	18
Not Completed	2
Reason Not Completed
Adverse Event	1
Withdrawal by Subject	1
Period Title: Weeks 13-24: 1.0 mg/kg/Week
Started	18
Completed	18
Not Completed	0
Period Title: Weeks 25-36: 2.0 mg/kg/Week
Started	18
Completed	18
Not Completed	0
Period Title: Continuation Period
Started	18
Completed	18
Not Completed	0

Baseline Characteristics

Arm/Group Title		BMN 110
Arm/Group Description		Dose-Escalation Period:
Overall Number of Baseline Participants		20
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	20 participants
		8.0 (2.89)
Age, Customized; Measure Type: Number; Unit of measure: Participants	Number Analyzed	20 participants
>=4 to <8 years		10
>=8 to <10 years		6
>=10 to <=18 years		4
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	20 participants
	Female	8 40.0%
	Male	12 60.0%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	20 participants
	Hispanic or Latino	0 0.0%
	Not Hispanic or Latino	20 100.0%
	Unknown or Not Reported	0 0.0%
Race/Ethnicity, Customized; Measure Type: Number; Unit of measure: Participants	Number Analyzed	20 participants
American Indian or Alaska Native		0
Asian		9
Native Hawaiian or Other Pacific Islander		0
Black or African American		0
White		9
Other		2
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United Kingdom	Number Analyzed	20 participants
		20

Outcome Measures

1. Primary Outcome

Title	Subject Incidence of Treatment Emergent AEs
Description	The primary objective of the study was to evaluate the safety of weekly infusions of BMN 110 administered in escalating doses to subjects with MPS IVA. The safety variable incidence of TEAE is summarized.
Time Frame	Entire Study, through week 84

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	0.1 mg/kg/Week	1.0 mg/kg/Week	2.0 mg/kg/Week	Continuation Period	Entire Study
Arm/Group Description:	Dose-Escalation Period: Weeks 1-12: 0.1 mg/kg/week Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 0.1 mg/kg/week	Dose-Escalation Period: Weeks 13-24: 1.0 mg/kg/week Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 1.0 mg/kg/week	Dose-Escalation Period: Weeks 25-36: 2.0 mg/kg/week Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 2.0 mg/kg/week	Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.	Entire Study period includes both Dose-Escalation Period and Continuation Period.
Overall Number of Participants Analyzed	20	18	18	18	20
Measure Type: Number; Unit of Measure: participants
Any AEs	18	18	17	17	20
Any Study Drug-Related AEs	12	10	7	8	14
Any SAEs	6	2	8	6	14
Any Study Drug-Related SAEs	2	1	2	1	4
Any AEs During Infusion	15	13	10	15	17
Any SAEs During Infusion	5	0	1	1	6
Any AEs Causing Study Discontinuation	1	0	0	0	1
Any Study Drug-Related AE Causing Study Discont.	1	0	0	0	1
AEs Causing Permanent Study Drug Discont.	1	0	0	0	1
Drug-Related AE Causing Permanent StudyDrug Discon	1	0	0	0	1
Any SAEs Causing Study Discontinuation	1	0	0	0	1
Any SAEs Causing Permanent Study Drug Discont.	1	0	0	0	1
Study Drug-Related SAE Causing Study Discont.	1	0	0	0	1
StudyDrug-Related SAE Causing Permanent DrugDiscon	1	0	0	0	1
Death	0	0	0	0	0

2. Secondary Outcome

Title	Change From Baseline in 6MWT
Description	Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes.
Time Frame	Baseline to Weeks 12, 24, 36, 48, 72

Outcome Measure Data

Analysis Population Description

Intent-to-Treat population (all subjects who enrolled in the study). Two patients were either physically (score was designated as 0 m) or developmentally (score was set to missing) unable to perform the 6MWT. The analysis was based on observed cases.

Arm/Group Title	BMN 110
Arm/Group Description:	Dose-Escalation Period: Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen: Weeks 1-12: 0.1 mg/kg/week; Weeks 13-24: 1.0 mg/kg/week; Weeks 25-36: 2.0 mg/kg/week Continuation Period: Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.
Overall Number of Participants Analyzed	20
Mean (Standard Deviation); Unit of Measure: meters
Week 12 Change from Baseline (n=19)	-20.7 (85.95)
Week 24 Change from Baseline (n=17)	16.3 (71.74)
Week 36 Change from Baseline (n=17)	13.8 (63.25)
Week 48 Change from Baseline (n=17)	-4.8 (64.70)
Week 72 Change from Baseline (n=17)	4.0 (87.24)

3. Secondary Outcome

Title	Change From Baseline in 3MSCT
Description	Change from baseline in the 3-minute Stair Climb Test. Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute.
Time Frame	Baseline to Weeks 12, 24, 36, 48, 72

Outcome Measure Data

Analysis Population Description

Intent-to-Treat population (all subjects who enrolled in the study). One patient was developmentally unable to perform the 3MSCT and the test scores were set to missing. The analysis was based on observed cases.

Arm/Group Title	BMN 110
Arm/Group Description:	Dose-Escalation Period: Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen: Weeks 1-12: 0.1 mg/kg/week; Weeks 13-24: 1.0 mg/kg/week; Weeks 25-36: 2.0 mg/kg/week Continuation Period: Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.
Overall Number of Participants Analyzed	20
Mean (Standard Deviation); Unit of Measure: steps/min
Week 12 Change from Baseline (n=19)	0.3 (14.07)
Week 24 Change from Baseline (n=17)	6.1 (8.66)
Week 36 Change from Baseline (n=17)	7.8 (13.69)
Week 48 Change from Baseline (n=17)	9.7 (14.42)
Week 72 Change from Baseline (n=17)	9.7 (13.91)

4. Secondary Outcome

Title	Percent Change From Baseline in uKS
Description	Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value *100%
Time Frame	Baseline to Weeks 12, 24, 36, 72

Outcome Measure Data

Analysis Population Description

Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases.

Arm/Group Title	BMN 110
Arm/Group Description:	Dose-Escalation Period: Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen: Weeks 1-12: 0.1 mg/kg/week; Weeks 13-24: 1.0 mg/kg/week; Weeks 25-36: 2.0 mg/kg/week Continuation Period: Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.
Overall Number of Participants Analyzed	20
Mean (Standard Deviation); Unit of Measure: percentage of uKS
Week 12 Percent Change from Baseline (n=19)	-23.2 (19.04)
Week 24 Percent Change from Baseline (n=18)	-27.9 (17.92)
Week 36 Percent Change from Baseline (n=18)	-40.6 (20.16)
Week 72 Percent Change from Baseline (n=17)	-32.2 (17.10)

5. Secondary Outcome

Title	Percent Change From Baseline in MVV
Description	Percent Change from baseline in Maximum Voluntary Ventilation.
Time Frame	Baseline to Weeks 12, 24, 36, 72

Outcome Measure Data

Analysis Population Description

Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases.

Arm/Group Title	BMN 110
Arm/Group Description:	Dose-Escalation Period: Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen: Weeks 1-12: 0.1 mg/kg/week; Weeks 13-24: 1.0 mg/kg/week; Weeks 25-36: 2.0 mg/kg/week Continuation Period: Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.
Overall Number of Participants Analyzed	20
Mean (Standard Deviation); Unit of Measure: percentage of MVV
Week 12 Percent Change from Baseline (n=14)	9.9 (21.29)
Week 24 Percent Change from Baseline (n=13)	11.0 (21.48)
Week 36 Percent Change from Baseline (n=14)	10.5 (17.43)
Week 72 Percent Change from Baseline (n=14)	18.4 (20.77)

6. Secondary Outcome

Title	Percent Change From Baseline in FVC
Description	Percent Change from baseline in Forced Vital Capacity.
Time Frame	Baseline to Weeks 12, 24, 36, 72

Outcome Measure Data

Analysis Population Description

Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases.

Arm/Group Title	BMN 110
Arm/Group Description:	Dose-Escalation Period: Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen: Weeks 1-12: 0.1 mg/kg/week; Weeks 13-24: 1.0 mg/kg/week; Weeks 25-36: 2.0 mg/kg/week Continuation Period: Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.
Overall Number of Participants Analyzed	20
Mean (Standard Deviation); Unit of Measure: percentage of FVC
Week 12 Percent Change from Baseline (n=18)	3.4 (10.85)
Week 24 Percent Change from Baseline (n=16)	0.2 (16.60)
Week 36 Percent Change from Baseline (n=16)	10.7 (20.82)
Week 72 Percent Change from Baseline (n=16)	12.5 (14.88)

Adverse Events

Time Frame	Study Period, through 84 weeks
Adverse Event Reporting Description	All Adverse Events
Arm/Group Title	0.1 mg/kg/Week		1.0 mg/kg/Week		2.0 mg/kg/Week		Continuation Period		Entire Study
Arm/Group Description	Dose-Escalation Period: Weeks 1-12: 0.1 mg/kg/week Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 0.1 mg/kg/week		Dose-Escalation Period: Weeks 13-24: 1.0 mg/kg/week Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 1.0 mg/kg/week		Dose-Escalation Period: Weeks 25-36: 2.0 mg/kg/week Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 2.0 mg/kg/week		Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.		Entire Study period includes both Dose-Escalation Period and Continuation Period.
All-Cause Mortality
	0.1 mg/kg/Week		1.0 mg/kg/Week		2.0 mg/kg/Week		Continuation Period		Entire Study
	Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)
Total	--/--		--/--		--/--		--/--		--/--
Serious Adverse Events
	0.1 mg/kg/Week		1.0 mg/kg/Week		2.0 mg/kg/Week		Continuation Period		Entire Study
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	6/20 (30.00%)		2/18 (11.11%)		8/18 (44.44%)		6/18 (33.33%)		14/20 (70.00%)
General disorders
Gait disturbance † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Infusion related reaction † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	3	1/20 (5.00%)	4
Immune system disorders
Type I hypersensitivity † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Infections and infestations
Abdominal abscess † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Abscess limb † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Catheter site infection † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Implant site infection † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Lower respiratory tract infection † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	1	2/20 (10.00%)	2
Otitis media † 1	2/20 (10.00%)	2	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/20 (10.00%)	2
Pneumonia † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Injury, poisoning and procedural complications
Road traffic accident † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Musculoskeletal and connective tissue disorders
Knee deformity † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	2/18 (11.11%)	2	2/18 (11.11%)	2	5/20 (25.00%)	5
Pain in extremity † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Skin and subcutaneous tissue disorders
Drug eruption † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Rash generalised † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Rash maculo-papular † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Surgical and medical procedures
Abscess drainage † 1	1/20 (5.00%)	1	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	2
Catheterisation venous † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	3/18 (16.67%)	3	0/18 (0.00%)	0	3/20 (15.00%)	3
Vascular disorders
Poor venous access † 1	3/20 (15.00%)	3	0/18 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	1	4/20 (20.00%)	5
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDRA 12.0
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	0.1 mg/kg/Week		1.0 mg/kg/Week		2.0 mg/kg/Week		Continuation Period		Entire Study
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	18/20 (90.00%)		18/18 (100.00%)		16/18 (88.89%)		17/18 (94.44%)		20/20 (100.00%)
Blood and lymphatic system disorders
Lymphadenopathy † 1	0/20 (0.00%)	0	2/18 (11.11%)	2	0/18 (0.00%)	0	0/18 (0.00%)	0	2/20 (10.00%)	2
Cardiac disorders
Mitral valve disease † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	0/18 (0.00%)	0	2/20 (10.00%)	2
Tachycardia † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Ear and labyrinth disorders
Cerumen impaction † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Deafness neurosensory † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Ear canal erythema † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Ear disorder † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Ear pain † 1	1/20 (5.00%)	1	1/18 (5.56%)	2	1/18 (5.56%)	3	5/18 (27.78%)	10	5/20 (25.00%)	16
External ear disorder † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Hyperacusis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Hypoacusis † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	2/20 (10.00%)	2
Inner ear disorder † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Motion sickness † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Otorrhoea † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Tinnitus † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Endocrine disorders
Autoimmune thyroiditis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Eye disorders
Conjunctivitis † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	2/20 (10.00%)	2
Eye discharge † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Eyelid cyst † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Gastrointestinal disorders
Abdominal pain † 1	3/20 (15.00%)	3	1/18 (5.56%)	2	1/18 (5.56%)	3	1/18 (5.56%)	2	4/20 (20.00%)	10
Abdominal pain upper † 1	4/20 (20.00%)	8	1/18 (5.56%)	2	3/18 (16.67%)	6	4/18 (22.22%)	4	8/20 (40.00%)	20
Constipation † 1	1/20 (5.00%)	1	1/18 (5.56%)	1	0/18 (0.00%)	0	1/18 (5.56%)	1	3/20 (15.00%)	3
Dental caries † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	2/20 (10.00%)	2
Diarrhoea † 1	0/20 (0.00%)	0	2/18 (11.11%)	2	3/18 (16.67%)	4	1/18 (5.56%)	2	6/20 (30.00%)	8
Faecal incontinence † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Gastrooesophageal reflux disease † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Glossodynia † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Nausea † 1	1/20 (5.00%)	1	1/18 (5.56%)	1	0/18 (0.00%)	0	2/18 (11.11%)	3	3/20 (15.00%)	5
Retching † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	2
Salivary gland enlargement † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Toothache † 1	1/20 (5.00%)	1	2/18 (11.11%)	2	1/18 (5.56%)	1	1/18 (5.56%)	1	4/20 (20.00%)	5
Vomiting † 1	2/20 (10.00%)	3	7/18 (38.89%)	7	4/18 (22.22%)	6	12/18 (66.67%)	26	13/20 (65.00%)	42
General disorders
Application site vesicles † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Catheter site erythema † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	2
Catheter site pain † 1	3/20 (15.00%)	3	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	3/20 (15.00%)	4
Chills † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Extravasation † 1	1/20 (5.00%)	2	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	2
Feeling hot † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	1	2/20 (10.00%)	2
Gait disturbance † 1	2/20 (10.00%)	2	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/20 (10.00%)	2
Implant site erythema † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	4	1/20 (5.00%)	4
Implant site extravasation † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Implant site rash † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Influenza like illness † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Infusion site erythema † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	1	1/20 (5.00%)	2
Infusion site inflammation † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Infusion site oedema † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Injection site pain † 1	0/20 (0.00%)	0	2/18 (11.11%)	2	0/18 (0.00%)	0	0/18 (0.00%)	0	2/20 (10.00%)	2
Injection site reaction † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Malaise † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	2	1/20 (5.00%)	3
Pyrexia † 1	6/20 (30.00%)	6	9/18 (50.00%)	12	5/18 (27.78%)	8	10/18 (55.56%)	20	14/20 (70.00%)	46
Hepatobiliary disorders
Hepatomegaly † 1	1/20 (5.00%)	1	1/18 (5.56%)	1	0/18 (0.00%)	0	1/18 (5.56%)	1	2/20 (10.00%)	3
Immune system disorders
Drug hypersensitivity † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Hypersensitivity † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Infections and infestations
Abdominal abscess † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Catheter site infection † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Ear infection † 1	1/20 (5.00%)	1	1/18 (5.56%)	1	0/18 (0.00%)	0	3/18 (16.67%)	4	5/20 (25.00%)	6
Eye infection † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/18 (5.56%)	1	2/20 (10.00%)	2
Gastroenteritis viral † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Helminthic infection † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Impetigo † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	3	1/20 (5.00%)	3
Infection † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Lice infestation † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	1	1/20 (5.00%)	2
Localised infection † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Lower respiratory tract infection † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	2/20 (10.00%)	2
Nail infection † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Nasopharyngitis † 1	2/20 (10.00%)	3	3/18 (16.67%)	3	1/18 (5.56%)	1	4/18 (22.22%)	6	8/20 (40.00%)	13
Otitis externa † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Pharyngitis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	2/20 (10.00%)	2
Rhinitis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	2/20 (10.00%)	2
Skin infection † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Subcutaneous abscess † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Tinea pedis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Tonsillitis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Varicella † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Injury, poisoning and procedural complications
Arthropod bite † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Arthropod sting † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Contusion † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	1	2/18 (11.11%)	2	2/20 (10.00%)	4
Device migration † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	2/20 (10.00%)	3
Excoriation † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Eye injury † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Face injury † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	2	1/20 (5.00%)	2
Fall † 1	2/20 (10.00%)	3	0/18 (0.00%)	0	2/18 (11.11%)	2	1/18 (5.56%)	2	5/20 (25.00%)	7
Head injury † 1	2/20 (10.00%)	3	1/18 (5.56%)	2	1/18 (5.56%)	1	1/18 (5.56%)	1	4/20 (20.00%)	7
Injury † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	2/20 (10.00%)	2
Joint injury † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Medical device complication † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	2/20 (10.00%)	2
Mouth injury † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Procedural pain † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	2/20 (10.00%)	2
Procedural vomiting † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Thermal burn † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Investigations
Alanine aminotransferase increased † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Aspartate aminotransferase increased † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Blood immunoglobulin E increased † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Blood immunoglobulin G decreased † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Blood lactate dehydrogenase increased † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Blood sodium increased † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Cardiac murmur † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Computerised tomogram † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Echocardiogram abnormal † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Electrocardiogram T wave amplitude decreased † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Eosinophil count increased † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	2/20 (10.00%)	2
Nuclear magnetic resonance imaging † 1	1/20 (5.00%)	1	2/18 (11.11%)	2	0/18 (0.00%)	0	4/18 (22.22%)	4	6/20 (30.00%)	7
Oxygen saturation decreased † 1	0/20 (0.00%)	0	1/18 (5.56%)	3	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	3
Protein total abnormal † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Respiratory rate increased † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Weight increased † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Metabolism and nutrition disorders
Dehydration † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Hypercholesterolaemia † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Musculoskeletal and connective tissue disorders
Arthralgia † 1	3/20 (15.00%)	3	3/18 (16.67%)	4	0/18 (0.00%)	0	4/18 (22.22%)	4	8/20 (40.00%)	11
Back pain † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	3/18 (16.67%)	4	4/20 (20.00%)	5
Bursitis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Mobility decreased † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	1	3/20 (15.00%)	3
Muscular weakness † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Neck pain † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Pain in extremity † 1	4/20 (20.00%)	6	3/18 (16.67%)	3	1/18 (5.56%)	1	4/18 (22.22%)	5	10/20 (50.00%)	15
Pain in jaw † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Nervous system disorders
Ageusia † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Clonus † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Dizziness † 1	2/20 (10.00%)	3	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/20 (10.00%)	3
Headache † 1	4/20 (20.00%)	8	2/18 (11.11%)	3	1/18 (5.56%)	8	6/18 (33.33%)	16	9/20 (45.00%)	35
Lethargy † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Spinal cord compression † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Psychiatric disorders
Insomnia † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Renal and urinary disorders
Enuresis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Incontinence † 1	0/20 (0.00%)	0	1/18 (5.56%)	3	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	3
Urinary incontinence † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	2/20 (10.00%)	2
Reproductive system and breast disorders
Balanitis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Penile pain † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Respiratory, thoracic and mediastinal disorders
Cough † 1	1/20 (5.00%)	1	5/18 (27.78%)	7	3/18 (16.67%)	3	10/18 (55.56%)	18	13/20 (65.00%)	29
Dry throat † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Dyspnoea † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Epistaxis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Nasal congestion † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	1/18 (5.56%)	2	1/18 (5.56%)	1	3/20 (15.00%)	4
Oropharyngeal pain † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	1/18 (5.56%)	1	4/18 (22.22%)	4	6/20 (30.00%)	6
Pharyngeal oedema † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Productive cough † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Rales † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Wheezing † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Skin and subcutaneous tissue disorders
Dermatitis diaper † 1	1/20 (5.00%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Dry skin † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/18 (5.56%)	1	2/20 (10.00%)	2
Erythema † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	2/18 (11.11%)	2	2/20 (10.00%)	2
Petechiae † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Pruritus † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Psoriasis † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Rash † 1	1/20 (5.00%)	1	1/18 (5.56%)	1	0/18 (0.00%)	0	3/18 (16.67%)	4	4/20 (20.00%)	6
Rash generalised † 1	0/20 (0.00%)	0	1/18 (5.56%)	2	0/18 (0.00%)	0	1/18 (5.56%)	1	2/20 (10.00%)	3
Rash maculo-papular † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	3	1/20 (5.00%)	3
Rash papular † 1	0/20 (0.00%)	0	1/18 (5.56%)	3	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	3
Rash pruritic † 1	1/20 (5.00%)	1	1/18 (5.56%)	1	0/18 (0.00%)	0	1/18 (5.56%)	2	3/20 (15.00%)	4
Skin disorder † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	2	1/20 (5.00%)	2
Skin lesion † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Skin ulcer † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Surgical and medical procedures
Cautery to nose † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	1/20 (5.00%)	1
Induction of anaesthesia † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Vascular disorders
Flushing † 1	0/20 (0.00%)	0	2/18 (11.11%)	2	1/18 (5.56%)	1	1/18 (5.56%)	1	3/20 (15.00%)	4
Hot flush † 1	0/20 (0.00%)	0	0/18 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	1/20 (5.00%)	1
Hypotension † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
Poor venous access † 1	0/20 (0.00%)	0	1/18 (5.56%)	1	0/18 (0.00%)	0	0/18 (0.00%)	0	1/20 (5.00%)	1
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDRA 12.0

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

PI cannot publish any data prior to multi-centre publication. If publication related to sub-sets of data shall make reference to the relevant multi-centre publication. PI agrees to submit data to Sponsor for review and comment 60 days prior to publication. During review period, Sponsor may request that publication be delayed for up to 6 months from date of first submission to Sponsor in order for Sponsor to take steps to protect its proprietary information.

Results Point of Contact

• Name/Title: BioMarin Medical Information Services
• Organization: BioMarin Pharmaceutical Inc.
• Phone: 800-983-4587
• EMail: medinfo@bmrn.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Hendriksz C, Santra S, Jones SA, Geberhiwot T, Jesaitis L, Long B, Qi Y, Hawley SM, Decker C. Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment. Mol Genet Metab. 2018 Apr;123(4):479-487. doi: 10.1016/j.ymgme.2018.02.011. Epub 2018 Feb 19.

• Responsible Party: BioMarin Pharmaceutical
• ClinicalTrials.gov Identifier: NCT00884949
• Other Study ID Numbers: MOR-002
• First Submitted: April 10, 2009
• First Posted: April 21, 2009
• Results First Submitted: March 13, 2014
• Results First Posted: June 30, 2014
• Last Update Posted: June 30, 2014