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A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01242111
Recruitment Status : Terminated
First Posted : November 16, 2010
Results First Posted : September 30, 2015
Last Update Posted : September 30, 2015
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions MPS IV A
Mucopolysaccharidosis IVA
Morquio A Syndrome
Intervention Drug: BMN 110
Enrollment 20

Recruitment Details  
Pre-assignment Details  
Arm/Group Title BMN 110
Hide Arm/Group Description BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Period Title: Overall Study
Started 20
Completed 17
Not Completed 3
Reason Not Completed
Adverse Event             1
Withdrawal by Subject             1
Did not consent to MOR-100 follow-up             1
Arm/Group Title BMN 110
Hide Arm/Group Description BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Overall Number of Baseline Participants 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants
8.4  (2.90)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 20 participants
>=4 to < 8 years 10
>=8 to <10 years 5
>=10 to <=18 years 5
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
1.Primary Outcome
Title Safety Evaluation
Hide Description

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.

Time Frame Entire Study Period, up to 240 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The primary outcome measure data is presented in more detail under the Adverse Events section.
Arm/Group Title BMN 110
Hide Arm/Group Description:
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
Serious Adverse Events 19
Other (Not Including Serious) Adverse Events 20
2.Secondary Outcome
Title Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Hide 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 and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial
Show Outcome Measure DataHide Outcome Measure Data
Hide 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
Hide Arm/Group Description:
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: meters
MOR-002 Week 12 Change from Baseline (n=19) -20.7  (85.95)
MOR-002 Week 24 Change from Baseline (n=17) 16.3  (71.74)
MOR-002 Week 36 Change from Baseline (n=17) 13.8  (63.25)
MOR-002 Week 48 Change from Baseline (n=17) -4.8  (64.70)
MOR-002 Week 72 Change from Baseline (n=17) 4.0  (87.24)
MOR-100 Week 0 Change from Baseline (n=16) 15.7  (89.00)
MOR-100 Week 24 Change from Baseline (n=16) 24.5  (101.23)
MOR-100 Week 48 Change from Baseline (n=16) 6.8  (98.66)
MOR-100 Week 72 Change from Baseline (n=17) -49.8  (132.63)
MOR-100 Week 96 Change from Baseline (n=16) 11.2  (85.24)
MOR-100 Week 120 Change from Baseline (n=16) 4.2  (94.09)
MOR-100 Week 144 Change from Baseline (n=13) 3.1  (106.82)
MOR-100 Week 192 Change from Baseline (n=9) -37.1  (103.96)
3.Secondary Outcome
Title Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Hide Description Change from baseline in the 3-minute Stair Climb Test (3MSCT). 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 and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial
Show Outcome Measure DataHide Outcome Measure Data
Hide 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
Hide Arm/Group Description:
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: steps/min
MOR-002 Week 12 Change from Baseline (n=19) 0.3  (14.07)
MOR-002 Week 24 Change from Baseline (n=17) 6.1  (8.66)
MOR-002 Week 36 Change from Baseline (n=17) 7.8  (13.69)
MOR-002 Week 48 Change from Baseline (n=17) 9.7  (14.42)
MOR-002 Week 72 Change from Baseline (n=17) 9.7  (13.91)
MOR-100 Week 0 Change from Baseline (n=16) 12.7  (13.96)
MOR-100 Week 24 Change from Baseline (n=16) 13.4  (17.07)
MOR-100 Week 48 Change from Baseline (n=16) 6.6  (16.87)
MOR-100 Week 72 Change from Baseline (n=16) -1.4  (21.11)
MOR-100 Week 96 Change from Baseline (n=16) 9.9  (18.84)
MOR-100 Week 120 Change from Baseline (n=16) 6.2  (14.41)
MOR-100 Week 144 Change from Baseline (n=12) 5.4  (11.93)
MOR-100 Week 192 Change from Baseline (n=7) -0.2  (10.34)
4.Secondary Outcome
Title Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Hide 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 and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 168 weeks during the MOR-100 extension trial
Show Outcome Measure DataHide Outcome Measure Data
Hide 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
Hide Arm/Group Description:
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: percentage change
MOR-002 Wk 12 Percent Change from Baseline (n=19) -23.2  (19.04)
MOR-002 Wk 24 Percent Change from Baseline (n=18) -27.9  (17.92)
MOR-002 Wk 36 Percent Change from Baseline (n=18) -40.6  (20.16)
MOR-002 Wk 72 Percent Change from Baseline (n=17) -32.2  (17.10)
MOR-100 Wk 0 Percent Change from Baseline (n=17) -30.0  (19.23)
MOR-100 Wk 24 Percent Change from Baseline (n=17) -43.6  (19.56)
MOR-100 Wk 48 Percent Change from Baseline (n=16) -41.9  (19.29)
MOR-100 Wk 72 Percent Change from Baseline (n=12) -36.4  (36.70)
MOR-100 Wk 96 Percent Change from Baseline (n=17) -49.7  (19.93)
MOR-100 Wk 120 Percent Change from Baseline (n=16) -49.3  (22.28)
MOR-100 Wk 144 Percent Change from Baseline (n=16) -56.6  (19.54)
MOR-100 Wk 168 Percent Change from Baseline (n=15) -58.9  (16.02)
5.Secondary Outcome
Title Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Hide Description Percent Change from baseline in Maximum Voluntary Ventilation.
Time Frame Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial
Show Outcome Measure DataHide Outcome Measure Data
Hide 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
Hide Arm/Group Description:
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: percentage change
MOR-002 Wk 12 Percent Change from Baseline (n=14) 9.9  (21.29)
MOR-002 Wk 24 Percent Change from Baseline (n=13) 11.0  (21.48)
MOR-002 Wk 36 Percent Change from Baseline (n=14) 10.5  (17.43)
MOR-002 Wk 72 Percent Change from Baseline (n=14) 18.4  (20.77)
MOR-100 Wk 0 Percent Change from Baseline (n=13) 11.1  (16.44)
MOR-100 Wk 24 Percent Change from Baseline (n=13) 9.8  (22.25)
MOR-100 Wk 48 Percent Change from Baseline (n=12) 3.5  (17.78)
MOR-100 Wk 72 Percent Change from Baseline (n=13) 10.1  (27.83)
MOR-100 Wk 96 Percent Change from Baseline (n=12) -4.9  (35.53)
MOR-100 Wk 120 Percent Change from Baseline (n=13) -3.3  (28.35)
MOR-100 Wk 144 Percent Change from Baseline (n=11) -3.5  (34.61)
MOR-100 Wk 192 Percent Change from Baseline (n=6) 28.2  (38.78)
6.Secondary Outcome
Title Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Hide Description Percent Change from baseline in Forced Vital Capacity.
Time Frame Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial
Show Outcome Measure DataHide Outcome Measure Data
Hide 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
Hide Arm/Group Description:
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: percentage change
MOR-002 Wk 12 Percent Change from Baseline (n=18) 3.4  (10.85)
MOR-002 Wk 24 Percent Change from Baseline (n=16) 0.2  (16.60)
MOR-002 Wk 36 Percent Change from Baseline (n=16) 10.7  (20.81)
MOR-002 Wk 72 Percent Change from Baseline (n=16) 12.5  (14.88)
MOR-100 Wk 0 Percent Change from Baseline (n=15) 11.8  (14.97)
MOR-100 Wk 24 Percent Change from Baseline (n=14) 15.3  (16.31)
MOR-100 Wk 48 Percent Change from Baseline (n=13) 15.8  (16.56)
MOR-100 Wk 72 Percent Change from Baseline (n=15) 16.1  (21.96)
MOR-100 Wk 96 Percent Change from Baseline (n=14) 14.8  (17.36)
MOR-100 Wk 120 Percent Change from Baseline (n=16) 22.8  (21.14)
MOR-100 Wk 144 Percent Change from Baseline (n=12) 17.5  (24.32)
MOR-100 Wk 192 Percent Change from Baseline (n=7) 18.6  (30.98)
Time Frame Study Period, up to 240 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title BMN 110
Hide Arm/Group Description BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
All-Cause Mortality
BMN 110
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
BMN 110
Affected / at Risk (%) # Events
Total   19/20 (95.00%)    
Cardiac disorders   
Supraventricular tachycardia  1  1/20 (5.00%)  1
Tachycardia  1  1/20 (5.00%)  1
General disorders   
Gait disturbance  1  1/20 (5.00%)  1
Infusion site inflammation  1  1/20 (5.00%)  1
Infusion site reaction  1  2/20 (10.00%)  3
Malaise  1  1/20 (5.00%)  1
Pyrexia  1  2/20 (10.00%)  2
Immune system disorders   
Type I hypersensitivity  1  1/20 (5.00%)  1
Infections and infestations   
Abdominal abscess  1  1/20 (5.00%)  1
Abscess limb  1  1/20 (5.00%)  1
Catheter site infection  1  1/20 (5.00%)  1
Device related infection  1  2/20 (10.00%)  2
Infusion site infection  1  1/20 (5.00%)  1
Lobar pneumonia  1  1/20 (5.00%)  1
Lower respiratory tract infection  1  2/20 (10.00%)  5
Otitis media  1  3/20 (15.00%)  3
Pneumonia  1  1/20 (5.00%)  1
Pneumonia mycoplasmal  1  1/20 (5.00%)  1
Respiratory syncytial virus infection  1  1/20 (5.00%)  1
Viral infection  1  2/20 (10.00%)  2
Injury, poisoning and procedural complications   
Infusion related reaction  1  1/20 (5.00%)  3
Procedural pain  1  1/20 (5.00%)  1
Road traffic accident  1  1/20 (5.00%)  1
Skull fracture  1  1/20 (5.00%)  1
Musculoskeletal and connective tissue disorders   
Knee deformity  1  6/20 (30.00%)  7
Pain in extremity  1  1/20 (5.00%)  1
Nervous system disorders   
Cervical cord compression  1  2/20 (10.00%)  2
Psychiatric disorders   
Panic attack  1  1/20 (5.00%)  1
Respiratory, thoracic and mediastinal disorders   
Pulmonary oedema  1  1/20 (5.00%)  1
Sleep apnoea syndrome  1  1/20 (5.00%)  2
Skin and subcutaneous tissue disorders   
Rash  1  1/20 (5.00%)  3
Rash maculo-papular  1  1/20 (5.00%)  1
Surgical and medical procedures   
Abscess drainage  1  1/20 (5.00%)  2
Bone operation  1  1/20 (5.00%)  1
Central venous catheterisation  1  2/20 (10.00%)  2
Knee operation  1  2/20 (10.00%)  2
Medical device implantation  1  2/20 (10.00%)  2
Medical device removal  1  2/20 (10.00%)  2
Orthopaedic procedure  1  3/20 (15.00%)  3
Vascular disorders   
Flushing  1  1/20 (5.00%)  2
Hypertension  1  1/20 (5.00%)  1
Jugular vein distension  1  1/20 (5.00%)  1
Poor venous access  1  8/20 (40.00%)  9
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 16.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
BMN 110
Affected / at Risk (%) # Events
Total   20/20 (100.00%)    
Blood and lymphatic system disorders   
Eosinophilia  1  1/20 (5.00%)  1
Lymphadenopathy  1  4/20 (20.00%)  5
Cardiac disorders   
Aortic valve incompetence  1  3/20 (15.00%)  3
Diastolic dysfunction  1  1/20 (5.00%)  1
Mitral valve disease  1  2/20 (10.00%)  2
Mitral valve incompetence  1  3/20 (15.00%)  3
Pulmonary valve incompetence  1  3/20 (15.00%)  3
Tachycardia  1  3/20 (15.00%)  6
Tricuspid valve incompetence  1  3/20 (15.00%)  3
Congenital, familial and genetic disorders   
Congenital heart valve disorder  1  1/20 (5.00%)  1
Ear and labyrinth disorders   
Cerumen impaction  1  3/20 (15.00%)  3
Deafness  1  1/20 (5.00%)  1
Deafness neurosensory  1  1/20 (5.00%)  2
Ear canal erythema  1  1/20 (5.00%)  1
Ear discomfort  1  1/20 (5.00%)  1
Ear disorder  1  1/20 (5.00%)  1
Ear pain  1  12/20 (60.00%)  41
Ear pruritus  1  1/20 (5.00%)  1
Hyperacusis  1  1/20 (5.00%)  2
Hypoacusis  1  2/20 (10.00%)  4
Middle ear disorder  1  1/20 (5.00%)  1
Motion sickness  1  4/20 (20.00%)  9
Otorrhoea  1  2/20 (10.00%)  2
Tinnitus  1  1/20 (5.00%)  1
Tympanic membrane disorder  1  3/20 (15.00%)  3
Tympanic membrane hyperaemia  1  2/20 (10.00%)  2
Tympanic membrane perforation  1  1/20 (5.00%)  1
Endocrine disorders   
Autoimmune thyroiditis  1  1/20 (5.00%)  2
Eye disorders   
Conjunctivitis  1  3/20 (15.00%)  4
Dry eye  1  1/20 (5.00%)  1
Eye discharge  1  1/20 (5.00%)  1
Eye pain  1  1/20 (5.00%)  1
Eye pruritus  1  1/20 (5.00%)  1
Eye swelling  1  2/20 (10.00%)  3
Eyelid cyst  1  1/20 (5.00%)  1
Ocular hyperaemia  1  1/20 (5.00%)  1
Gastrointestinal disorders   
Abdominal discomfort  1  2/20 (10.00%)  2
Abdominal pain  1  12/20 (60.00%)  27
Abdominal pain upper  1  11/20 (55.00%)  33
Constipation  1  5/20 (25.00%)  5
Dental caries  1  3/20 (15.00%)  5
Dental plaque  1  1/20 (5.00%)  1
Diarrhoea  1  12/20 (60.00%)  28
Dyspepsia  1  1/20 (5.00%)  1
Faecal incontinence  1  1/20 (5.00%)  2
Gastrooesophageal reflux disease  1  2/20 (10.00%)  3
Gingival hyperpigmentation  1  1/20 (5.00%)  1
Glossodynia  1  1/20 (5.00%)  1
Haematemesis  1  1/20 (5.00%)  1
Lip disorder  1  1/20 (5.00%)  1
Lip pain  1  2/20 (10.00%)  2
Mouth ulceration  1  1/20 (5.00%)  1
Nausea  1  5/20 (25.00%)  8
Retching  1  2/20 (10.00%)  3
Tongue discolouration  1  1/20 (5.00%)  1
Tooth deposit  1  1/20 (5.00%)  1
Tooth disorder  1  1/20 (5.00%)  1
Toothache  1  5/20 (25.00%)  8
Vomiting  1  16/20 (80.00%)  92
General disorders   
Application site reaction  1  1/20 (5.00%)  1
Application site vesicles  1  1/20 (5.00%)  1
Catheter site erythema  1  3/20 (15.00%)  9
Catheter site extravasation  1  7/20 (35.00%)  12
Catheter site inflammation  1  1/20 (5.00%)  1
Catheter site oedema  1  1/20 (5.00%)  1
Catheter site pain  1  4/20 (20.00%)  5
Catheter site rash  1  1/20 (5.00%)  1
Catheter site related reaction  1  2/20 (10.00%)  3
Catheter site scab  1  1/20 (5.00%)  1
Catheter site swelling  1  2/20 (10.00%)  6
Chest pain  1  1/20 (5.00%)  2
Chills  1  1/20 (5.00%)  1
Device connection issue  1  1/20 (5.00%)  1
Device dislocation  1  1/20 (5.00%)  2
Device expulsion  1  1/20 (5.00%)  2
Device leakage  1  1/20 (5.00%)  1
Device malfunction  1  1/20 (5.00%)  1
Device occlusion  1  3/20 (15.00%)  3
Extravasation  1  2/20 (10.00%)  3
Fatigue  1  7/20 (35.00%)  11
Feeling hot  1  2/20 (10.00%)  2
Gait disturbance  1  3/20 (15.00%)  4
Influenza like illness  1  3/20 (15.00%)  3
Infusion site erythema  1  1/20 (5.00%)  1
Infusion site extravasation  1  1/20 (5.00%)  2
Infusion site pain  1  1/20 (5.00%)  1
Infusion site reaction  1  1/20 (5.00%)  3
Injection site reaction  1  1/20 (5.00%)  1
Local swelling  1  2/20 (10.00%)  3
Malaise  1  4/20 (20.00%)  8
Medical device complication  1  2/20 (10.00%)  4
Medical device site reaction  1  1/20 (5.00%)  1
Pain  1  6/20 (30.00%)  12
Pyrexia  1  18/20 (90.00%)  103
Vaccination site pain  1  2/20 (10.00%)  2
Hepatobiliary disorders   
Hepatomegaly  1  1/20 (5.00%)  2
Immune system disorders   
Drug hypersensitivity  1  1/20 (5.00%)  1
Seasonal allergy  1  5/20 (25.00%)  8
Infections and infestations   
Abdominal abscess  1  1/20 (5.00%)  1
Catheter site infection  1  1/20 (5.00%)  1
Ear infection  1  6/20 (30.00%)  15
Eye infection  1  2/20 (10.00%)  2
Fungal infection  1  1/20 (5.00%)  1
Fungal skin infection  1  2/20 (10.00%)  2
Furuncle  1  3/20 (15.00%)  4
Gastroenteritis viral  1  1/20 (5.00%)  1
Gingivitis  1  2/20 (10.00%)  2
Helminthic infection  1  1/20 (5.00%)  1
Herpes simplex  1  1/20 (5.00%)  2
Herpes zoster  1  2/20 (10.00%)  2
Impetigo  1  1/20 (5.00%)  3
Infection  1  2/20 (10.00%)  2
Influenza  1  1/20 (5.00%)  1
Lice infestation  1  1/20 (5.00%)  2
Localised infection  1  2/20 (10.00%)  2
Lower respiratory tract infection  1  5/20 (25.00%)  13
Molluscum contagiosum  1  1/20 (5.00%)  2
Nail infection  1  1/20 (5.00%)  1
Nasopharyngitis  1  17/20 (85.00%)  53
Oral candidiasis  1  1/20 (5.00%)  1
Otitis externa  1  1/20 (5.00%)  1
Otitis media  1  3/20 (15.00%)  3
Paronychia  1  1/20 (5.00%)  1
Pharyngitis  1  1/20 (5.00%)  1
Postoperative wound infection  1  1/20 (5.00%)  1
Rash pustular  1  1/20 (5.00%)  1
Rhinitis  1  5/20 (25.00%)  8
Skin infection  1  3/20 (15.00%)  3
Subcutaneous abscess  1  1/20 (5.00%)  1
Tinea pedis  1  4/20 (20.00%)  4
Tonsillitis  1  3/20 (15.00%)  5
Upper respiratory tract infection  1  5/20 (25.00%)  5
Urinary tract infection  1  1/20 (5.00%)  2
Varicella  1  1/20 (5.00%)  1
Viral infection  1  1/20 (5.00%)  1
Viral upper respiratory tract infection  1  1/20 (5.00%)  1
Wound infection  1  1/20 (5.00%)  1
Injury, poisoning and procedural complications   
Arthropod bite  1  4/20 (20.00%)  5
Arthropod sting  1  1/20 (5.00%)  1
Contusion  1  6/20 (30.00%)  8
Ear injury  1  1/20 (5.00%)  1
Excoriation  1  4/20 (20.00%)  4
Eye contusion  1  3/20 (15.00%)  3
Face injury  1  1/20 (5.00%)  1
Fall  1  8/20 (40.00%)  20
Head injury  1  8/20 (40.00%)  14
Infusion related reaction  1  2/20 (10.00%)  2
Injury  1  2/20 (10.00%)  2
Laceration  1  2/20 (10.00%)  2
Ligament sprain  1  1/20 (5.00%)  1
Limb injury  1  3/20 (15.00%)  4
Lip injury  1  1/20 (5.00%)  1
Neck injury  1  1/20 (5.00%)  1
Occupational exposure to radiation  1  1/20 (5.00%)  1
Periorbital contusion  1  1/20 (5.00%)  1
Post procedural complication  1  1/20 (5.00%)  1
Procedural headache  1  1/20 (5.00%)  1
Procedural pain  1  7/20 (35.00%)  7
Procedural vomiting  1  1/20 (5.00%)  1
Scar  1  3/20 (15.00%)  3
Scratch  1  1/20 (5.00%)  1
Skeletal injury  1  1/20 (5.00%)  1
Sunburn  1  1/20 (5.00%)  1
Thermal burn  1  1/20 (5.00%)  1
Upper limb fracture  1  1/20 (5.00%)  1
Wound complication  1  1/20 (5.00%)  1
Investigations   
Alanine aminotransferase increased  1  1/20 (5.00%)  1
Aspartate aminotransferase increased  1  1/20 (5.00%)  1
Aspiration joint  1  1/20 (5.00%)  1
Blood cholesterol increased  1  2/20 (10.00%)  2
Blood immunoglobulin E increased  1  6/20 (30.00%)  6
Blood immunoglobulin G decreased  1  1/20 (5.00%)  1
Blood lactate dehydrogenase increased  1  1/20 (5.00%)  1
Blood pressure systolic increased  1  1/20 (5.00%)  1
Blood sodium increased  1  1/20 (5.00%)  1
Body temperature increased  1  4/20 (20.00%)  6
Breath sounds abnormal  1  1/20 (5.00%)  1
Cardiac murmur  1  2/20 (10.00%)  3
Computerised tomogram  1  1/20 (5.00%)  1
Electrocardiogram T wave amplitude decreased  1  1/20 (5.00%)  2
Eosinophil count increased  1  2/20 (10.00%)  3
Eosinophil percentage increased  1  1/20 (5.00%)  1
Liver palpable subcostal  1  1/20 (5.00%)  1
Nuclear magnetic resonance imaging  1  6/20 (30.00%)  8
Nuclear magnetic resonance imaging spinal  1  1/20 (5.00%)  1
Oxygen saturation decreased  1  1/20 (5.00%)  3
Protein total abnormal  1  1/20 (5.00%)  1
Protein urine  1  1/20 (5.00%)  2
Respiratory rate increased  1  3/20 (15.00%)  3
Ultrasound scan abnormal  1  1/20 (5.00%)  1
Weight increased  1  2/20 (10.00%)  4
Metabolism and nutrition disorders   
Appetite disorder  1  2/20 (10.00%)  2
Decreased appetite  1  2/20 (10.00%)  3
Dehydration  1  1/20 (5.00%)  1
Hypercholesterolaemia  1  1/20 (5.00%)  2
Vitamin D deficiency  1  1/20 (5.00%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia  1  12/20 (60.00%)  58
Atlantoaxial instability  1  1/20 (5.00%)  1
Axillary mass  1  1/20 (5.00%)  1
Back pain  1  9/20 (45.00%)  17
Bursitis  1  1/20 (5.00%)  1
Joint instability  1  1/20 (5.00%)  1
Joint stiffness  1  1/20 (5.00%)  2
Joint swelling  1  2/20 (10.00%)  2
Knee deformity  1  2/20 (10.00%)  2
Mobility decreased  1  5/20 (25.00%)  6
Muscle spasms  1  1/20 (5.00%)  3
Muscular weakness  1  2/20 (10.00%)  3
Musculoskeletal chest pain  1  1/20 (5.00%)  1
Musculoskeletal pain  1  2/20 (10.00%)  3
Myalgia  1  1/20 (5.00%)  1
Neck pain  1  8/20 (40.00%)  12
Pain in extremity  1  17/20 (85.00%)  76
Pain in jaw  1  1/20 (5.00%)  1
Nervous system disorders   
Ageusia  1  1/20 (5.00%)  1
Areflexia  1  1/20 (5.00%)  1
Cervical cord compression  1  1/20 (5.00%)  1
Clonus  1  3/20 (15.00%)  4
Dizziness  1  4/20 (20.00%)  7
Headache  1  16/20 (80.00%)  100
Lethargy  1  5/20 (25.00%)  5
Migraine  1  1/20 (5.00%)  1
Neuralgia  1  1/20 (5.00%)  1
Paraesthesia  1  1/20 (5.00%)  1
Reflexes abnormal  1  1/20 (5.00%)  1
Spinal cord compression  1  1/20 (5.00%)  2
Psychiatric disorders   
Anxiety  1  1/20 (5.00%)  1
Insomnia  1  2/20 (10.00%)  3
Renal and urinary disorders   
Dysuria  1  1/20 (5.00%)  1
Enuresis  1  1/20 (5.00%)  3
Incontinence  1  1/20 (5.00%)  3
Polyuria  1  1/20 (5.00%)  1
Urinary incontinence  1  1/20 (5.00%)  1
Reproductive system and breast disorders   
Balanitis  1  2/20 (10.00%)  2
Dysmenorrhoea  1  2/20 (10.00%)  3
Genital ulceration  1  1/20 (5.00%)  1
Menorrhagia  1  1/20 (5.00%)  1
Penile pain  1  1/20 (5.00%)  1
Perineal ulceration  1  1/20 (5.00%)  1
Scrotal erythema  1  1/20 (5.00%)  1
Vulvovaginal pruritus  1  1/20 (5.00%)  1
Respiratory, thoracic and mediastinal disorders   
Asthma  1  2/20 (10.00%)  3
Cough  1  18/20 (90.00%)  81
Dry throat  1  1/20 (5.00%)  1
Dysphonia  1  1/20 (5.00%)  2
Dyspnoea  1  4/20 (20.00%)  5
Dyspnoea exertional  1  1/20 (5.00%)  1
Epistaxis  1  4/20 (20.00%)  12
Nasal congestion  1  2/20 (10.00%)  3
Nasal obstruction  1  5/20 (25.00%)  6
Oropharyngeal pain  1  15/20 (75.00%)  31
Pharyngeal disorder  1  1/20 (5.00%)  1
Pharyngeal erythema  1  1/20 (5.00%)  1
Pharyngeal oedema  1  1/20 (5.00%)  1
Productive cough  1  2/20 (10.00%)  3
Rales  1  1/20 (5.00%)  1
Respiratory failure  1  1/20 (5.00%)  1
Rhinitis allergic  1  1/20 (5.00%)  1
Rhinorrhoea  1  3/20 (15.00%)  6
Sleep apnoea syndrome  1  1/20 (5.00%)  1
Tachypnoea  1  1/20 (5.00%)  2
Wheezing  1  4/20 (20.00%)  6
Skin and subcutaneous tissue disorders   
Acne  1  2/20 (10.00%)  2
Dermatitis  1  1/20 (5.00%)  1
Dermatitis contact  1  1/20 (5.00%)  1
Dermatitis diaper  1  1/20 (5.00%)  1
Dry skin  1  5/20 (25.00%)  6
Eczema  1  2/20 (10.00%)  3
Eczema asteatotic  1  1/20 (5.00%)  1
Erythema  1  4/20 (20.00%)  43
Lichen planus  1  1/20 (5.00%)  1
Miliaria  1  1/20 (5.00%)  1
Onychomadesis  1  1/20 (5.00%)  1
Pain of skin  1  1/20 (5.00%)  1
Papule  1  2/20 (10.00%)  2
Petechiae  1  1/20 (5.00%)  1
Pigmentation disorder  1  6/20 (30.00%)  14
Pruritus  1  5/20 (25.00%)  11
Psoriasis  1  1/20 (5.00%)  3
Rash  1  11/20 (55.00%)  34
Rash erythematous  1  1/20 (5.00%)  1
Rash generalised  1  1/20 (5.00%)  1
Rash maculo-papular  1  5/20 (25.00%)  35
Skin depigmentation  1  1/20 (5.00%)  1
Skin discolouration  1  1/20 (5.00%)  1
Skin disorder  1  4/20 (20.00%)  5
Skin exfoliation  1  1/20 (5.00%)  1
Skin mass  1  2/20 (10.00%)  4
Urticaria  1  2/20 (10.00%)  53
Surgical and medical procedures   
Catheter removal  1  1/20 (5.00%)  2
Cautery to nose  1  1/20 (5.00%)  1
Central venous catheterisation  1  1/20 (5.00%)  1
Ear tube insertion  1  1/20 (5.00%)  1
Medical device removal  1  4/20 (20.00%)  4
Thrombectomy  1  1/20 (5.00%)  1
Vascular disorders   
Flushing  1  4/20 (20.00%)  8
Hot flush  1  2/20 (10.00%)  2
Hypertension  1  3/20 (15.00%)  6
Hypotension  1  2/20 (10.00%)  3
Poor venous access  1  3/20 (15.00%)  6
Thrombosis  1  2/20 (10.00%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 16.1
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 it shall make reference to the relevant multi-centre publication. PI agrees to submit material to Sponsor for review at least 60 days prior to publication and incorporate all reasonable comments. During review, 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
Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01242111     History of Changes
Other Study ID Numbers: MOR-100
First Submitted: October 28, 2010
First Posted: November 16, 2010
Results First Submitted: July 10, 2015
Results First Posted: September 30, 2015
Last Update Posted: September 30, 2015