Safety and Exercise Study of Two Doses of BMN 110 for Mor... | NCT01609062

Label	Type	Description	Intervention Names
BMN 110 Weekly at 2.0 mg/kg/week	Experimental		Drug: BMN 110
BMN 110 Weekly at 4.0 mg/kg/week	Experimental		Drug: BMN 110

Name	Type	Description	Arm Group Labels	Other Names
BMN 110	Drug	Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for up to 192 weeks.	BMN 110 Weekly at 2.0 mg/kg/week

Measure	Description	Time Frame
Safety Evaluation	The primary objective of the study is to evaluate the safety of weekly infusions of BMN 110; the safety variables included Adverse Events (AEs). The primary outcome measure data is presented in more detail under the Adverse Events section.	Entire Study Period, up to 192 weeks or ETV (early termination visit)

Measure	Description	Time Frame
6-minute Walk Test (6MWT)	Change from baseline to Week 12, 24, and 52 as measured in distance walked (meters) in 6MWT.	Baseline, Week 12, 24, and 52
3-minute Stair Climb Test (3MSCT)	Change from baseline to Week 12, 24, and 52 as measured in speed (stairs/min) in 3MSCT.

Name	Affiliation	Role
Adam Shaywitz, MD PhD	BioMarin Pharmaceutical	Study Director

PubMed Identifier	Type	Citation	Retractions
29159458	Derived	Berger KI, Burton BK, Lewis GD, Tarnopolsky M, Harmatz PR, Mitchell JJ, Muschol N, Jones SA, Sutton VR, Pastores GM, Lau H, Sparkes R, Shaywitz AJ. Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alfa. JIMD Rep. 2018;42:9-17. doi: 10.1007/8904_2017_70. Epub 2017 Nov 21.

ID	Title	Description
FG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
FG001	BMN 110 Weekly at 4.0 mg/kg/Week

ID	Title	Description
BG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BG002	Total	Total of all reporting groups

Units	Counts
Participants	BG00015 BG00110 BG00225

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Safety Evaluation

The primary objective of the study is to evaluate the safety of weekly infusions of BMN 110; the safety variables included Adverse Events (AEs).

The primary outcome measure data is presented in more detail under the Adverse Events section.

Posted

Number

participants

Entire Study Period, up to 192 weeks or ETV (early termination visit)

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Units	Counts
Participants	OG00015 OG00110 OG00225

Title

Denominators

Categories

Serious Adverse Events

Title	Measurements
	OG0003 OG0012 OG0025

Other (Not Including Serious) Adverse Events

Secondary

6-minute Walk Test (6MWT)

Change from baseline to Week 12, 24, and 52 as measured in distance walked (meters) in 6MWT.

Modified Intent-to-Treat Analysis Set

Posted

Mean

Standard Deviation

meters

Baseline, Week 12, 24, and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Units	Counts
Participants

Secondary

3-minute Stair Climb Test (3MSCT)

Change from baseline to Week 12, 24, and 52 as measured in speed (stairs/min) in 3MSCT.

Modified Intent-to-Treat Analysis Set

Posted

Mean

Standard Deviation

stairs/min

Baseline, Week 12, 24, and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Units	Counts
Participants

Secondary

Respiratory Function Test (MVV and FVC)

Respiratory Function was assessed by spirometry in accordance with American Thoracic Society standards.

Percent change from baseline to Week 12, 24, and 52 as measured by Maximum Voluntary Ventilation (MVV, L/min) Percent change from baseline to Week 12, 24, and 52 as measured by Forced Vital Capacity (FVC, L)

Modified Intent-to-treat Analysis Set

Posted

Mean

Standard Deviation

Percent Change

Baseline, Week 12, 24, and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Units	Counts

Secondary

Normalized Urine Keratan Sulfate (uKS)

Urinary KS was measured by a quantitative method and normalized using the sample urinary creatinine measurement.

Percent change from baseline to Week 12, 24, and 52 in normalized urine keratan sulfate (ug/mg).

Modified Intent-to-treat Analysis Set

Posted

Mean

Standard Deviation

Percent Change

Baseline, Week 12, 24, and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Units	Counts
Participants

Secondary

Cardiopulmonary Exercise Testing (CPET) - Duration of Exercise

Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing.

Change from baseline to Week 25 and 52 as measured by the CPET Duration of Exercise (min)

A subset of mITT subjects who were scheduled to perform CPET

Posted

Mean

Standard Deviation

Percent Change

Baseline, Week 25 and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Secondary

Cardiopulmonary Exercise Testing (CPET) - Peak Workload

Percent change from baseline to Week 25 and 52 as measured by the CPET Peak workload (watt)

A subset of mITT subjects who were scheduled to perform CPET

Posted

Mean

Standard Deviation

Percent Change

Baseline, Week 25 and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Secondary

Cardiopulmonary Exercise Testing (CPET) - O2 Pulse

Percent change from baseline to Week 25 and 52 as measured by the CPET O2 pulse (ml/beat)

A subset of mITT subjects who were scheduled to perform CPET

Posted

Mean

Standard Deviation

Percent Change

Baseline, Week 25 and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Secondary

Cardiopulmonary Exercise Testing (CPET) - Aerobic Efficiency

Percent change from baseline to Week 25 and 52 as measured by the CPET Aerobic Efficiency (ml/watt).

Note that decline in Aerobic Efficiency translate into an improvement

A subset of mITT subjects who were scheduled to perform CPET

Posted

Mean

Standard Deviation

Percent Change

Baseline, Week 25 and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Secondary

Muscle Strength Testing (MST) - Knee Extension Test

Change from baseline to Week 25 and 52 as measured by the peak force in MST knee extension test (newton meters).

Modified Intent-to-treat Analysis Set

Posted

Mean

Standard Deviation

Percent Change

Baseline, Week 25 and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Units	Counts
Participants

Secondary

Muscle Strength Testing (MST) - Knee Flexion Test

Percent change from baseline to Week 25 and 52 as measured by the peak force in MST knee flexion test (newton meters).

Modified Intent-to-treat Analysis Set

Posted

Mean

Standard Deviation

Percent Change

Baseline, Week 25 and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Units	Counts
Participants

Secondary

Muscle Strength Testing (MST) - Elbow Flexion Test

Percent change from baseline to Week 25 and 52 as measured by the peak force in MST elbow flexion test (newton meters).

Modified Intent-to-treat Analysis Set

Posted

Mean

Standard Deviation

Percent Change

Baseline, Week 25 and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG002	All Subjects	Both groups combined

Units	Counts
Participants

Secondary

Adolescent Pediatric Pain Tool (APPT) - Pain Intensity

The APPT is a validated, multidimensional tool to evaluate pain in children, adolescents, and young adults. The complete APPT is measured in three parts - Part 1 of the APPT scale determines the subject's pain locations using a body template. Part 2 of the APPT scale determines the intensity of the pain using a 10 cm visual analog scale (VAS) with the lowest point of the scale (0) labeled No Pain and the highest point on the scale (10) labeled Worst Possible Pain. Intermediate regions of the sale were labeled with 3 intermediate descriptors (Little Pain, Medium Pain, and Large Pain). Part 3 of the APPT scale characterizes the pain by tracking the number and percentage of words selected by subjects to describe their pain from a total of 57 choices. Part 2 corresponds most closely to other typically used pain scales (based on VAS) and for this reason the results from Part 2 are presented here.

Change from baseline to Week 12, 24, and 52 in pain intensity.

Modified Intent-to-treat

Posted

Mean

Standard Deviation

units on a scale

Baseline, Week 12, 24, and 52

ID	Title	Description
OG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
OG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Lymph node pain	Blood and lymphatic system disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Bradycardia	Cardiac disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Sinus bradycardia	Cardiac disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Tachycardia	Cardiac disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0012 events1 affected10 at risk
Pectus carinatum	Congenital, familial and genetic disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Ear discomfort	Ear and labyrinth disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Ear pain	Ear and labyrinth disorders	MedDRA 16.1	Systematic Assessment	EG0003 events2 affected15 at risk EG0012 events2 affected10 at risk
Ear pruritus	Ear and labyrinth disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Motion sickness	Ear and labyrinth disorders	MedDRA 16.1	Systematic Assessment	EG0003 events2 affected15 at risk EG0010 events0 affected10 at risk
Otorrhoea	Ear and labyrinth disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Tinnitus	Ear and labyrinth disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Eye pruritus	Eye disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Photophobia	Eye disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Vision blurred	Eye disorders	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0010 events0 affected10 at risk
Vitreous floaters	Eye disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Abdominal discomfort	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0003 events1 affected15 at risk EG0010 events0 affected10 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG00018 events6 affected15 at risk EG00123 events3 affected10 at risk
Abdominal pain lower	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0008 events4 affected15 at risk EG00111 events4 affected10 at risk
Aerophagia	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Anal pruritus	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Aphthous stomatitis	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Constipation	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG00021 events6 affected15 at risk EG00110 events4 affected10 at risk
Dyspepsia	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0016 events1 affected10 at risk
Gastrooesophageal reflux disease	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Haematochezia	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Lip oedema	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Nausea	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG00033 events10 affected15 at risk EG0016 events5 affected10 at risk
Oral pain	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Retching	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Swollen tongue	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Toothache	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Vomiting	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG00034 events11 affected15 at risk EG00110 events5 affected10 at risk
Catheter site pain	General disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Chest pain	General disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Chills	General disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Cyst	General disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Device malfunction	General disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Extravasation	General disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Face oedema	General disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Fatigue	General disorders	MedDRA 16.1	Systematic Assessment	EG00012 events8 affected15 at risk EG0015 events2 affected10 at risk
Influenza like illness	General disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Infusion site bruising	General disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Infusion site erythema	General disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Infusion site extravasation	General disorders	MedDRA 16.1	Systematic Assessment	EG0006 events3 affected15 at risk EG0014 events2 affected10 at risk
Infusion site swelling	General disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Injection site pain	General disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Injection site pruritus	General disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0012 events1 affected10 at risk
Injection site reaction	General disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Irritability	General disorders	MedDRA 16.1	Systematic Assessment	EG0003 events2 affected15 at risk EG0010 events0 affected10 at risk
Local swelling	General disorders	MedDRA 16.1	Systematic Assessment	EG0003 events2 affected15 at risk EG0011 events1 affected10 at risk
Malaise	General disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0011 events1 affected10 at risk
Medical device pain	General disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Oedema peripheral	General disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Pain	General disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0011 events1 affected10 at risk
Pyrexia	General disorders	MedDRA 16.1	Systematic Assessment	EG00024 events9 affected15 at risk EG0019 events3 affected10 at risk
Seasonal allergy	Immune system disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0012 events2 affected10 at risk
Ear infection	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0003 events2 affected15 at risk EG0010 events0 affected10 at risk
Enterobiasis	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0003 events1 affected15 at risk EG0010 events0 affected10 at risk
Fungal infection	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Gastroenteritis	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0003 events2 affected15 at risk EG0015 events3 affected10 at risk
Gastroenteritis viral	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Gastrointestinal infection	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Influenza	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0004 events4 affected15 at risk EG0011 events1 affected10 at risk
Nasopharyngitis	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG00010 events6 affected15 at risk EG00116 events7 affected10 at risk
Oral herpes	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Otitis media	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0011 events1 affected10 at risk
Pharyngitis	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Pharyngitis streptococcal	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0005 events2 affected15 at risk EG0010 events0 affected10 at risk
Pneumonia	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Rhinitis	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0012 events2 affected10 at risk
Sinusitis	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG00013 events5 affected15 at risk EG0017 events5 affected10 at risk
Urinary tract infection	Infections and infestations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Excoriation	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0011 events1 affected10 at risk
Fall	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0013 events2 affected10 at risk
Hand fracture	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Head injury	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Inadequate analgesia	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Infusion related reaction	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Injection related reaction	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0010 events0 affected10 at risk
Joint injury	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Laceration	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Ligament sprain	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Limb crushing injury	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Limb injury	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Muscle strain	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Procedural pain	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0008 events5 affected15 at risk EG0012 events2 affected10 at risk
Wound complication	Injury, poisoning and procedural complications	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Blood glucose decreased	Investigations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Haemoglobin increased	Investigations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Urine leukocyte esterase positive	Investigations	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Urine output decreased	Investigations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Weight decreased	Investigations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
White blood cells urine positive	Investigations	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Decreased appetite	Metabolism and nutrition disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0011 events1 affected10 at risk
Hyperglycaemia	Metabolism and nutrition disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Hypophosphataemia	Metabolism and nutrition disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Increased appetite	Metabolism and nutrition disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG00028 events6 affected15 at risk EG00111 events5 affected10 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG00013 events6 affected15 at risk EG0011 events1 affected10 at risk
Elbow deformity	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Flank pain	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Joint swelling	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0011 events1 affected10 at risk
Knee deformity	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0011 events1 affected10 at risk
Kyphoscoliosis	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Muscle spasms	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Musculoskeletal chest pain	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0004 events3 affected15 at risk EG0010 events0 affected10 at risk
Musculoskeletal stiffness	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0005 events2 affected15 at risk EG0010 events0 affected10 at risk
Osteoarthritis	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Osteochondrosis	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Osteopenia	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG00022 events8 affected15 at risk EG0015 events4 affected10 at risk
Coordination abnormal	Nervous system disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Dizziness	Nervous system disorders	MedDRA 16.1	Systematic Assessment	EG0008 events6 affected15 at risk EG0013 events2 affected10 at risk
Dysgeusia	Nervous system disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Headache	Nervous system disorders	MedDRA 16.1	Systematic Assessment	EG00089 events13 affected15 at risk EG00144 events7 affected10 at risk
Lethargy	Nervous system disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Neuralgia	Nervous system disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Poor quality sleep	Nervous system disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Somnolence	Nervous system disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Syncope	Nervous system disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Anxiety	Psychiatric disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0013 events1 affected10 at risk
Depression	Psychiatric disorders	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0010 events0 affected10 at risk
Insomnia	Psychiatric disorders	MedDRA 16.1	Systematic Assessment	EG0003 events3 affected15 at risk EG0010 events0 affected10 at risk
Phobia	Psychiatric disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Dysuria	Renal and urinary disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Enuresis	Renal and urinary disorders	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0010 events0 affected10 at risk
Pollakiuria	Renal and urinary disorders	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0010 events0 affected10 at risk
Asthma	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0011 events1 affected10 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0009 events6 affected15 at risk EG0018 events4 affected10 at risk
Dysphonia	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0004 events4 affected15 at risk EG0010 events0 affected10 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0005 events3 affected15 at risk EG0011 events1 affected10 at risk
Hypoventilation	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0009 events7 affected15 at risk EG0012 events2 affected10 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG00011 events7 affected15 at risk EG0016 events4 affected10 at risk
Productive cough	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Respiratory distress	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0011 events1 affected10 at risk
Throat irritation	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0012 events2 affected10 at risk
Wheezing	Respiratory, thoracic and mediastinal disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Acne	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0014 events2 affected10 at risk
Decubitus ulcer	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Dermatitis allergic	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0011 events1 affected10 at risk
Dermatitis contact	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Dry skin	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0012 events1 affected10 at risk
Ecchymosis	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Eczema	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0002 events2 affected15 at risk EG0010 events0 affected10 at risk
Erythema	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0009 events4 affected15 at risk EG0012 events2 affected10 at risk
Nail disorder	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0006 events3 affected15 at risk EG0011 events1 affected10 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0007 events6 affected15 at risk EG0013 events1 affected10 at risk
Rash erythematous	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Rash generalised	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Rash pruritic	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0013 events2 affected10 at risk
Swelling face	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA 16.1	Systematic Assessment	EG00011 events3 affected15 at risk EG0010 events0 affected10 at risk
Catheterisation venous	Surgical and medical procedures	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Central venous catheterisation	Surgical and medical procedures	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0011 events1 affected10 at risk
Medical device removal	Surgical and medical procedures	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0012 events2 affected10 at risk
Tooth extraction	Surgical and medical procedures	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0010 events0 affected10 at risk
Flushing	Vascular disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Hot flush	Vascular disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Hypotension	Vascular disorders	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0010 events0 affected10 at risk
Pallor	Vascular disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Peripheral coldness	Vascular disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk

Facility	Status	City	State	ZIP	Country	Contacts
		Oakland	California		United States

ID	Term
D009085	Mucopolysaccharidosis IV
D016464	Lysosomal Storage Diseases

ID	Term
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D017520	Mucinoses
D003240	Connective Tissue Diseases
D017437	Skin and Connective Tissue Diseases
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

ID	Term
C102295	GALNS protein, human
D002810	Chondroitinases and Chondroitin Lyases
C491158	Galns protein, mouse
D013429	Sulfatases
D056947	Enzyme Replacement Therapy

ID	Term
D004950	Esterases
D006867	Hydrolases
D004798	Enzymes
D045762	Enzymes and Coenzymes
D011133	Polysaccharide-Lyases
D019757	Carbon-Oxygen Lyases
D008190	Lyases
D057487	Enzyme Therapy
D004358	Drug Therapy
D013812	Therapeutics

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	BMN 110 Weekly at 2.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.			3	15	15	15
EG001	BMN 110 Weekly at 4.0 mg/kg/Week	BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.			2	10	10	10

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Inflammatory bowel disease	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Umbilical hernia	Gastrointestinal disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Knee deformity	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk
Kyphoscoliosis	Musculoskeletal and connective tissue disorders	MedDRA 16.1	Systematic Assessment	EG0002 events1 affected15 at risk EG0010 events0 affected10 at risk
Enuresis	Renal and urinary disorders	MedDRA 16.1	Systematic Assessment	EG0001 events1 affected15 at risk EG0010 events0 affected10 at risk
Medical device removal	Surgical and medical procedures	MedDRA 16.1	Systematic Assessment	EG0000 events0 affected15 at risk EG0011 events1 affected10 at risk