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

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ClinicalTrials.gov Identifier: NCT01275066
Recruitment Status : Completed
First Posted : January 12, 2011
Results First Posted : April 15, 2014
Last Update Posted : July 7, 2014
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition MPS IV A
Interventions Drug: BMN 110 Weekly
Drug: Placebo
Drug: BMN 110 Every Other Week
Enrollment 177
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo BMN110 2.0 mg/kg/Qow BMN110 2.0 mg/kg/Week
Hide Arm/Group Description Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week. Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks. Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
Period Title: Overall Study
Started 60 59 58
Treated 59 59 58
Completed 59 59 57
Not Completed 1 0 1
Reason Not Completed
Withdrawal by Subject             0             0             1
Not Diagnosed w/ Disease             1             0             0
Arm/Group Title Placebo BMN110 2.0 mg/kg/Qow BMN110 2.0 mg/kg/Week Total
Hide Arm/Group Description Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week. Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks. Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week. Total of all reporting groups
Overall Number of Baseline Participants 59 59 58 176
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 59 participants 59 participants 58 participants 176 participants
15.0  (11.30) 15.3  (10.79) 13.1  (8.10) 14.5  (10.16)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 59 participants 59 participants 58 participants 176 participants
5 - 11 years 30 31 32 93
12 - 18 years 15 16 16 47
>= 19 years 14 12 10 36
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 59 participants 59 participants 58 participants 176 participants
Female
32
  54.2%
25
  42.4%
32
  55.2%
89
  50.6%
Male
27
  45.8%
34
  57.6%
26
  44.8%
87
  49.4%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 59 participants 59 participants 58 participants 176 participants
Hispanic or Latino
13
  22.0%
16
  27.1%
9
  15.5%
38
  21.6%
Not Hispanic or Latino
46
  78.0%
43
  72.9%
49
  84.5%
138
  78.4%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 59 participants 59 participants 58 participants 176 participants
American Indian or Alaska Native 0 0 0 0
Asian 11 15 14 40
Native Hawaiian or Other Pacific Islander 0 0 0 0
Black or African American 0 2 2 4
White 44 35 36 115
Other 4 7 6 17
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 59 participants 59 participants 58 participants 176 participants
Argentina 1 1 0 2
Brazil 6 10 5 21
Canada 4 5 5 14
Colombia 2 2 2 6
Denmark 0 0 1 1
France 7 5 8 20
Germany 4 5 1 10
Italy 4 4 2 10
Japan 0 4 2 6
Korea, South 3 1 3 7
Netherlands 1 2 3 6
Portugal 2 1 0 3
Qatar 1 0 1 2
Saudi Arabia 2 1 4 7
Taiwan 1 3 1 5
United Kingdom 9 4 10 23
United States 12 11 10 33
1.Primary Outcome
Title Change From Baseline in Endurance as Measured by the 6-minute Walk Test
Hide Description [Not Specified]
Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat (all patients receiving at least one dose of study drug). Two missing outcomes at Week 24 were imputed using method of multiple imputation.
Arm/Group Title Placebo BMN110 2.0 mg/kg/Qow BMN110 2.0 mg/kg/Week
Hide Arm/Group Description:
Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.
Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.
Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
Overall Number of Participants Analyzed 59 59 58
Mean (Standard Deviation)
Unit of Measure: meters
Baseline 211.9  (69.88) 205.7  (81.19) 203.9  (76.32)
Week 24 225.4  (83.22) 219.9  (87.60) 240.0  (86.61)
Change from Baseline to Week 24 13.5  (50.63) 14.2  (40.82) 36.0  (58.11)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, BMN110 2.0 mg/kg/Week
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0174
Comments Hochberg multiplicity adjustment - results considered statistically significant if both of p-values are p<0.05 or either of p-values is p<0.025.
Method ANCOVA
Comments ANCOVA of change from baseline with treatment, age group, baseline 6MWT category as covariates.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 22.5
Parameter Dispersion
Type: Standard Error of the mean
Value: 9.35
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, BMN110 2.0 mg/kg/Qow
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9542
Comments Hochberg multiplicity adjustment - results considered statistically significant if both of p-values are p<0.05 or either of p-values is p<0.025.
Method ANCOVA
Comments ANCOVA of change from baseline with treatment, age group, baseline 6MWT category as covariates.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.5
Parameter Dispersion
Type: Standard Error of the mean
Value: 9.29
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in Endurance as Measured by the 3-minute Stair Climb Test
Hide Description [Not Specified]
Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat (all patients receiving at least one dose of study drug). Two missing outcomes at Week 24 were imputed using method of multiple imputation.
Arm/Group Title Placebo BMN110 2.0 mg/kg/Qow BMN110 2.0 mg/kg/Week
Hide Arm/Group Description:
Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.
Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.
Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
Overall Number of Participants Analyzed 59 59 58
Mean (Standard Deviation)
Unit of Measure: stairs/minute
Baseline 30.0  (14.05) 27.1  (15.80) 29.6  (16.44)
Week 24 33.6  (18.36) 30.4  (17.77) 34.3  (18.70)
Change from Baseline to Week 24 3.6  (8.51) 3.2  (10.29) 4.7  (7.99)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, BMN110 2.0 mg/kg/Week
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4935
Comments Hochberg multiplicity adjustment - results considered statistically significant if both of p-values are p<0.05 or either of p-values is p<0.025.
Method ANCOVA
Comments ANCOVA of change from baseline with treatment, age group, baseline 6MWT category, and baseline 3MSCT as covariates.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.1
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.66
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, BMN110 2.0 mg/kg/Qow
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7783
Comments Hochberg multiplicity adjustment - results considered statistically significant if both of p-values are p<0.05 or either of p-values is p<0.025.
Method ANCOVA
Comments ANCOVA of change from baseline with treatment, age group, baseline 6MWT category, and baseline 3MSCT as covariates.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -0.5
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.66
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Percent Change From Baseline in Urine Keratan Sulfate Normalized for Urine Creatinine
Hide Description [Not Specified]
Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat (all patients receiving at least one dose of study drug). Nine missing outcomes at Week 24 were imputed using method of multiple imputation.
Arm/Group Title Placebo BMN110 2.0 mg/kg/Qow BMN110 2.0 mg/kg/Week
Hide Arm/Group Description:
Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.
Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.
Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
Overall Number of Participants Analyzed 59 59 58
Mean (Standard Deviation)
Unit of Measure: percent change
-3.6  (27.41) -35.3  (20.74) -43.7  (22.29)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, BMN110 2.0 mg/kg/Week
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Hochberg multiplicity adjustment - results considered statistically significant if both of p-values are p<0.05 or either of p-values is p<0.025.
Method ANCOVA
Comments ANCOVA of change from baseline with treatment, age group, baseline 6MWT category, and baseline uKS normalized for creatinine, as covariates.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -40.7
Parameter Dispersion
Type: Standard Error of the mean
Value: 4.20
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, BMN110 2.0 mg/kg/Qow
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Hochberg multiplicity adjustment - results considered statistically significant if both of p-values are p<0.05 or either of p-values is p<0.025.
Method ANCOVA
Comments ANCOVA of change from baseline with treatment, age group, baseline 6MWT category, and baseline uKS normalized for creatinine, as covariates.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -30.2
Parameter Dispersion
Type: Standard Error of the mean
Value: 4.19
Estimation Comments [Not Specified]
Time Frame Study Period, through 24 weeks
Adverse Event Reporting Description Treatment Emergent Events Only
 
Arm/Group Title Placebo BMN110 2.0 mg/kg/Qow BMN110 2.0 mg/kg/Week
Hide Arm/Group Description Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week. Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks. Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
All-Cause Mortality
Placebo BMN110 2.0 mg/kg/Qow BMN110 2.0 mg/kg/Week
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Hide Serious Adverse Events
Placebo BMN110 2.0 mg/kg/Qow BMN110 2.0 mg/kg/Week
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/59 (3.39%)      4/59 (6.78%)      9/58 (15.52%)    
Ear and labyrinth disorders       
Deafness  1  1/59 (1.69%)  1 0/59 (0.00%)  0 0/58 (0.00%)  0
Gastrointestinal disorders       
Vomiting  1  0/59 (0.00%)  0 0/59 (0.00%)  0 1/58 (1.72%)  1
General disorders       
Infusion site pain  1  0/59 (0.00%)  0 0/59 (0.00%)  0 1/58 (1.72%)  1
Immune system disorders       
Anaphylactic reaction  1  0/59 (0.00%)  0 1/59 (1.69%)  1 0/58 (0.00%)  0
Hypersensitivity  1  0/59 (0.00%)  0 0/59 (0.00%)  0 1/58 (1.72%)  1
Infections and infestations       
Dengue fever  1  0/59 (0.00%)  0 1/59 (1.69%)  1 0/58 (0.00%)  0
Lower respiratory tract infection  1  0/59 (0.00%)  0 0/59 (0.00%)  0 1/58 (1.72%)  1
Otitis media  1  0/59 (0.00%)  0 1/59 (1.69%)  1 1/58 (1.72%)  1
Pneumonia  1  0/59 (0.00%)  0 0/59 (0.00%)  0 2/58 (3.45%)  2
Viral upper respiratory tract infection  1  0/59 (0.00%)  0 0/59 (0.00%)  0 1/58 (1.72%)  1
Nervous system disorders       
Cervical cord compression  1  1/59 (1.69%)  1 0/59 (0.00%)  0 0/58 (0.00%)  0
Skin and subcutaneous tissue disorders       
Urticaria  1  0/59 (0.00%)  0 0/59 (0.00%)  0 1/58 (1.72%)  1
Surgical and medical procedures       
Suture removal  1  0/59 (0.00%)  0 1/59 (1.69%)  1 0/58 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo BMN110 2.0 mg/kg/Qow BMN110 2.0 mg/kg/Week
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   57/59 (96.61%)      59/59 (100.00%)      56/58 (96.55%)    
Cardiac disorders       
Mitral valve incompetence  1  4/59 (6.78%)  4 3/59 (5.08%)  3 3/58 (5.17%)  3
Pulmonary valve incompetence  1  2/59 (3.39%)  2 1/59 (1.69%)  1 3/58 (5.17%)  3
Tachycardia  1  6/59 (10.17%)  7 2/59 (3.39%)  6 3/58 (5.17%)  8
Tricuspid valve incompetence  1  3/59 (5.08%)  3 7/59 (11.86%)  7 4/58 (6.90%)  4
Ear and labyrinth disorders       
Ear pain  1  5/59 (8.47%)  6 8/59 (13.56%)  11 3/58 (5.17%)  3
Eye disorders       
Corneal opacity  1  1/59 (1.69%)  1 0/59 (0.00%)  0 5/58 (8.62%)  5
Gastrointestinal disorders       
Abdominal discomfort  1  1/59 (1.69%)  1 3/59 (5.08%)  4 2/58 (3.45%)  4
Abdominal pain  1  5/59 (8.47%)  5 8/59 (13.56%)  14 14/58 (24.14%)  23
Abdominal pain upper  1  5/59 (8.47%)  6 4/59 (6.78%)  4 9/58 (15.52%)  22
Diarrhoea  1  7/59 (11.86%)  8 12/59 (20.34%)  14 12/58 (20.69%)  14
Dyspepsia  1  4/59 (6.78%)  4 1/59 (1.69%)  1 1/58 (1.72%)  1
Flatulence  1  3/59 (5.08%)  4 1/59 (1.69%)  1 0/58 (0.00%)  0
Nausea  1  12/59 (20.34%)  13 14/59 (23.73%)  22 18/58 (31.03%)  37
Vomiting  1  21/59 (35.59%)  42 21/59 (35.59%)  44 25/58 (43.10%)  60
General disorders       
Chest discomfort  1  0/59 (0.00%)  0 1/59 (1.69%)  1 3/58 (5.17%)  3
Chills  1  1/59 (1.69%)  1 6/59 (10.17%)  7 6/58 (10.34%)  7
Device occlusion  1  1/59 (1.69%)  1 2/59 (3.39%)  2 3/58 (5.17%)  4
Fatigue  1  15/59 (25.42%)  24 8/59 (13.56%)  10 9/58 (15.52%)  17
Infusion site extravasation  1  2/59 (3.39%)  2 4/59 (6.78%)  4 2/58 (3.45%)  2
Infusion site pain  1  0/59 (0.00%)  0 4/59 (6.78%)  7 3/58 (5.17%)  3
Non-cardiac chest pain  1  0/59 (0.00%)  0 3/59 (5.08%)  3 1/58 (1.72%)  1
Oedema peripheral  1  2/59 (3.39%)  2 4/59 (6.78%)  6 1/58 (1.72%)  1
Puncture site pain  1  2/59 (3.39%)  2 3/59 (5.08%)  3 1/58 (1.72%)  1
Pyrexia  1  17/59 (28.81%)  29 22/59 (37.29%)  35 25/58 (43.10%)  47
Immune system disorders       
Hypersensitivity  1  1/59 (1.69%)  1 4/59 (6.78%)  7 2/58 (3.45%)  2
Infections and infestations       
Ear infection  1  1/59 (1.69%)  1 2/59 (3.39%)  2 5/58 (8.62%)  5
Gastroenteritis  1  4/59 (6.78%)  4 8/59 (13.56%)  10 7/58 (12.07%)  8
Influenza  1  3/59 (5.08%)  4 5/59 (8.47%)  5 2/58 (3.45%)  3
Nasopharyngitis  1  9/59 (15.25%)  12 12/59 (20.34%)  13 10/58 (17.24%)  11
Otitis media  1  4/59 (6.78%)  4 5/59 (8.47%)  5 8/58 (13.79%)  9
Pharyngitis  1  7/59 (11.86%)  7 3/59 (5.08%)  3 4/58 (6.90%)  4
Rhinitis  1  6/59 (10.17%)  8 4/59 (6.78%)  8 5/58 (8.62%)  8
Upper respiratory tract infection  1  9/59 (15.25%)  14 10/59 (16.95%)  13 10/58 (17.24%)  15
Viral infection  1  1/59 (1.69%)  1 6/59 (10.17%)  6 3/58 (5.17%)  3
Viral upper respiratory tract infection  1  3/59 (5.08%)  5 4/59 (6.78%)  5 2/58 (3.45%)  3
Injury, poisoning and procedural complications       
Fall  1  1/59 (1.69%)  1 4/59 (6.78%)  5 0/58 (0.00%)  0
Head injury  1  0/59 (0.00%)  0 3/59 (5.08%)  3 2/58 (3.45%)  2
Ligament sprain  1  3/59 (5.08%)  3 0/59 (0.00%)  0 0/58 (0.00%)  0
Investigations       
Blood pressure diastolic increased  1  2/59 (3.39%)  5 3/59 (5.08%)  4 2/58 (3.45%)  2
Blood pressure systolic increased  1  2/59 (3.39%)  5 3/59 (5.08%)  16 1/58 (1.72%)  1
Body temperature increased  1  2/59 (3.39%)  10 2/59 (3.39%)  2 4/58 (6.90%)  24
Oxygen saturation decreased  1  6/59 (10.17%)  19 7/59 (11.86%)  8 6/58 (10.34%)  10
Respiratory rate increased  1  3/59 (5.08%)  3 1/59 (1.69%)  1 1/58 (1.72%)  1
Musculoskeletal and connective tissue disorders       
Arthralgia  1  17/59 (28.81%)  27 9/59 (15.25%)  14 10/58 (17.24%)  14
Back pain  1  6/59 (10.17%)  7 10/59 (16.95%)  17 7/58 (12.07%)  10
Musculoskeletal pain  1  3/59 (5.08%)  4 2/59 (3.39%)  2 3/58 (5.17%)  4
Musculoskeletal stiffness  1  1/59 (1.69%)  1 3/59 (5.08%)  3 0/58 (0.00%)  0
Myalgia  1  0/59 (0.00%)  0 1/59 (1.69%)  1 3/58 (5.17%)  4
Neck pain  1  0/59 (0.00%)  0 3/59 (5.08%)  5 5/58 (8.62%)  6
Osteopenia  1  3/59 (5.08%)  3 3/59 (5.08%)  3 0/58 (0.00%)  0
Pain in extremity  1  9/59 (15.25%)  13 14/59 (23.73%)  24 9/58 (15.52%)  16
Nervous system disorders       
Dizziness  1  3/59 (5.08%)  3 4/59 (6.78%)  6 7/58 (12.07%)  10
Headache  1  21/59 (35.59%)  38 24/59 (40.68%)  52 24/58 (41.38%)  69
Hyperreflexia  1  3/59 (5.08%)  6 1/59 (1.69%)  3 0/58 (0.00%)  0
Paraesthesia  1  0/59 (0.00%)  0 0/59 (0.00%)  0 3/58 (5.17%)  6
Somnolence  1  0/59 (0.00%)  0 0/59 (0.00%)  0 3/58 (5.17%)  3
Psychiatric disorders       
Agitation  1  0/59 (0.00%)  0 2/59 (3.39%)  2 3/58 (5.17%)  3
Reproductive system and breast disorders       
Dysmenorrhoea  1  2/59 (3.39%)  2 3/59 (5.08%)  3 1/58 (1.72%)  1
Respiratory, thoracic and mediastinal disorders       
Asthma  1  0/59 (0.00%)  0 3/59 (5.08%)  3 1/58 (1.72%)  1
Cough  1  21/59 (35.59%)  28 17/59 (28.81%)  29 16/58 (27.59%)  20
Dyspnoea  1  3/59 (5.08%)  4 6/59 (10.17%)  8 7/58 (12.07%)  12
Epistaxis  1  3/59 (5.08%)  5 2/59 (3.39%)  2 3/58 (5.17%)  3
Nasal congestion  1  5/59 (8.47%)  8 5/59 (8.47%)  7 5/58 (8.62%)  7
Oropharyngeal pain  1  7/59 (11.86%)  8 9/59 (15.25%)  12 12/58 (20.69%)  14
Rhinorrhoea  1  4/59 (6.78%)  6 4/59 (6.78%)  9 5/58 (8.62%)  5
Throat irritation  1  0/59 (0.00%)  0 0/59 (0.00%)  0 3/58 (5.17%)  4
Tonsillar hypertrophy  1  3/59 (5.08%)  3 0/59 (0.00%)  0 0/58 (0.00%)  0
Skin and subcutaneous tissue disorders       
Eczema  1  3/59 (5.08%)  3 1/59 (1.69%)  1 1/58 (1.72%)  2
Petechiae  1  0/59 (0.00%)  0 3/59 (5.08%)  3 0/58 (0.00%)  0
Pruritus  1  2/59 (3.39%)  2 3/59 (5.08%)  5 4/58 (6.90%)  4
Rash  1  5/59 (8.47%)  6 6/59 (10.17%)  7 6/58 (10.34%)  9
Urticaria  1  0/59 (0.00%)  0 4/59 (6.78%)  5 3/58 (5.17%)  5
Vascular disorders       
Flushing  1  0/59 (0.00%)  0 1/59 (1.69%)  1 5/58 (8.62%)  7
Hot flush  1  1/59 (1.69%)  1 4/59 (6.78%)  6 3/58 (5.17%)  3
Hypertension  1  4/59 (6.78%)  13 4/59 (6.78%)  18 3/58 (5.17%)  6
Hypotension  1  1/59 (1.69%)  1 2/59 (3.39%)  3 3/58 (5.17%)  3
Poor venous access  1  4/59 (6.78%)  8 1/59 (1.69%)  1 3/58 (5.17%)  4
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: BioMarin Medical Information Services
Organization: BioMarin Pharmaceutical Inc.
Phone: (800) 983-4587
EMail: medinfo@bmrn.com
Layout table for additonal information
Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01275066    
Other Study ID Numbers: MOR-004
2010-020198-18 ( EudraCT Number )
10/H1306/87 ( Other Identifier: National Research Ethics Service )
18972/0213/001-0001 ( Other Identifier: Medicines and Healthcare Products Regulatory Agency )
2011_038#B201129 ( Other Identifier: Medical Ethics Review Committee (METC) )
145240 ( Other Identifier: Health Canada )
2011-01-09 ( Other Identifier: Committee for the Protection of Personnes )
20110012889 ( Other Identifier: Korean Food & Drug Administration (KFDA) )
0999935174 ( Other Identifier: Taiwan FDA )
First Submitted: January 10, 2011
First Posted: January 12, 2011
Results First Submitted: March 11, 2014
Results First Posted: April 15, 2014
Last Update Posted: July 7, 2014