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Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

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ClinicalTrials.gov Identifier: NCT01230801
Recruitment Status : Completed
First Posted : October 29, 2010
Results First Posted : June 11, 2018
Last Update Posted : June 11, 2018
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
Condition Pompe Disease
Intervention Biological: BMN 701
Enrollment 22
Recruitment Details  
Pre-assignment Details  
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Hide Arm/Group Description BMN 701 5 mg/kg IV BMN 701 10 mg/kg IV BMN 701 20 mg/kg IV
Period Title: Overall Study
Started 3 3 16
Completed 3 3 15
Not Completed 0 0 1
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Hide Arm/Group Description BMN 701 5 mg/kg IV BMN 701 10 mg/kg IV BMN 701 20 mg/kg IV Total of all reporting groups
Overall Number of Baseline Participants 3 3 16 22
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 16 participants 22 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
3
 100.0%
3
 100.0%
16
 100.0%
22
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants 3 participants 16 participants 22 participants
51.7  (6.81) 42.3  (12.90) 50.1  (5.37) 49.3  (7.00)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 16 participants 22 participants
Female
0
   0.0%
2
  66.7%
6
  37.5%
8
  36.4%
Male
3
 100.0%
1
  33.3%
10
  62.5%
14
  63.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 16 participants 22 participants
Hispanic or Latino
0
   0.0%
1
  33.3%
0
   0.0%
1
   4.5%
Not Hispanic or Latino
3
 100.0%
2
  66.7%
16
 100.0%
21
  95.5%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 16 participants 22 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
3
 100.0%
3
 100.0%
15
  93.8%
21
  95.5%
Other
0
   0.0%
0
   0.0%
1
   6.3%
1
   4.5%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 16 participants 22 participants
Australia
0
   0.0%
0
   0.0%
4
  25.0%
4
  18.2%
France
0
   0.0%
0
   0.0%
2
  12.5%
2
   9.1%
Germany
0
   0.0%
0
   0.0%
1
   6.3%
1
   4.5%
United Kingdom
0
   0.0%
0
   0.0%
6
  37.5%
6
  27.3%
United States
3
 100.0%
3
 100.0%
3
  18.8%
9
  40.9%
1.Primary Outcome
Title Number of Participants With Adverse Events
Hide Description Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Hide Arm/Group Description:
BMN 701 5 mg/kg IV
BMN 701 10 mg/kg IV
BMN 701 20 mg/kg IV
Overall Number of Participants Analyzed 3 3 16
Measure Type: Count of Participants
Unit of Measure: Participants
3
 100.0%
3
 100.0%
15
  93.8%
2.Secondary Outcome
Title Change From Baseline in Six Minutes Walk Test
Hide Description Change from Baseline in Six Minutes Walk Test. The 6MWT measured the maximum distance the subject could walk on a flat, hard surface in a period of 6 minutes
Time Frame Baseline up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Hide Arm/Group Description:
BMN 701 5 mg/kg IV
BMN 701 10 mg/kg IV
BMN 701 20 mg/kg IV
Total Results
Overall Number of Participants Analyzed 3 3 16 22
Mean (Standard Deviation)
Unit of Measure: meter
Baseline Number Analyzed 3 participants 3 participants 16 participants 22 participants
334  (227.12) 360  (51.4) 354.5  (156.94) 352.5  (151.05)
Change from Baseline - Week 6 Number Analyzed 2 participants 3 participants 16 participants 21 participants
-2.5  (23.33) -30.3  (56.50) 19.8  (35.11) 10.5  (40.08)
Change from Baseline - Week 12 Number Analyzed 3 participants 3 participants 15 participants 21 participants
31.2  (78.81) -13.7  (8.04) 11.1  (42.67) 10.4  (45.32)
Change from Baseline - Week 18 Number Analyzed 3 participants 3 participants 15 participants 21 participants
43.3  (89.44) -79.0  (104.61) 16.4  (55.09) 6.6  (73.43)
Change from Baseline - Week 24 Number Analyzed 3 participants 3 participants 16 participants 22 participants
36.0  (76.02) -42.7  (12.57) 22.3  (54.23) 15.3  (56.97)
3.Other Pre-specified Outcome
Title Change From Baseline in Percent Predicted Upright Forced Vital Capacity
Hide Description Change from Baseline in Percent Predicted Upright Forced Vital Capacity. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
Time Frame Baseline up to 24 week
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Hide Arm/Group Description:
BMN 701 5 mg/kg IV
BMN 701 10 mg/kg IV
BMN 701 20 mg/kg IV
Total Results
Overall Number of Participants Analyzed 3 3 16 22
Mean (Standard Deviation)
Unit of Measure: percentage of Predicted Upright FVC
Baseline Number Analyzed 3 participants 3 participants 16 participants 22 participants
69.3  (19.73) 67.3  (26.58) 58.1  (18.42) 60.9  (19.21)
Change from Baseline - Week 6 Number Analyzed 2 participants 3 participants 16 participants 21 participants
4.0  (4.24) -2.0  (2.65) 0.4  (3.3) 0.4  (3.46)
Change from Baseline - Week 12 Number Analyzed 3 participants 3 participants 16 participants 22 participants
-2.6  (9.5) -3.7  (2.89) 1.6  (4.35) 0.3  (5.25)
Change from Baseline - Week 18 Number Analyzed 3 participants 3 participants 15 participants 21 participants
1.5  (4.09) 0.3  (3.06) 1.2  (4.49) 1.1  (4.1)
Change from Baseline - Week 24 Number Analyzed 3 participants 3 participants 16 participants 22 participants
1.0  (2.65) -1.7  (3.06) 1.2  (3.89) 0.8  (3.65)
4.Other Pre-specified Outcome
Title Change From Baseline in Percent Predicted Supine Forced Vital Capacity
Hide Description Change from Baseline in Percent Predicted Supine Forced Vital Capacity. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
Time Frame Baseline up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Hide Arm/Group Description:
BMN 701 5 mg/kg IV
BMN 701 10 mg/kg IV
BMN 701 20 mg/kg IV
Total Results
Overall Number of Participants Analyzed 3 3 16 22
Mean (Standard Deviation)
Unit of Measure: percentage of Predicted Supine FVC
Baseline Number Analyzed 3 participants 3 participants 13 participants 19 participants
36.7  (17.95) 47.7  (32.58) 46.3  (21.93) 45  (22.1)
Change from Baseline - Week 6 Number Analyzed 2 participants 3 participants 13 participants 18 participants
2.1  (1.2) -1.3  (4.04) -0.8  (4.86) -0.6  (4.44)
Change from Baseline - Week 12 Number Analyzed 3 participants 3 participants 12 participants 18 participants
0.3  (4.22) -4.7  (2.89) 3.3  (3.87) 1.5  (4.7)
Change from Baseline - Week 18 Number Analyzed 3 participants 3 participants 12 participants 18 participants
1.3  (1.53) -1.3  (6.66) 2.3  (3.14) 1.5  (3.7)
Change from Baseline - Week 24 Number Analyzed 3 participants 3 participants 13 participants 19 participants
2.0  (3.0) -3.3  (5.51) 1.1  (4.42) 0.5  (4.53)
5.Other Pre-specified Outcome
Title Change From Baseline in Percent Predicted Upright Maximum Expiratory Pressure
Hide Description Change from Baseline in Percent Predicted Upright Maximum Expiratory Pressure. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
Time Frame Baseline up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point. No patient had baseline assessment in group of BMN 701 5 mg/kg.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Hide Arm/Group Description:
BMN 701 5 mg/kg IV
BMN 701 10 mg/kg IV
BMN 701 20 mg/kg IV
Total IV
Overall Number of Participants Analyzed 0 3 16 22
Mean (Standard Deviation)
Unit of Measure: percentage of Predicted MEP
Baseline Number Analyzed 0 participants 3 participants 16 participants 19 participants
31.1  (6.64) 36.3  (15.46) 35.5  (14.42)
Change from Baseline - Week 6 Number Analyzed 0 participants 3 participants 16 participants 19 participants
6.8  (4.06) 3.2  (5.38) 3.8  (5.27)
Change from Baseline - Week 12 Number Analyzed 0 participants 3 participants 16 participants 19 participants
1.4  (4.53) 2.2  (8.68) 2.0  (8.07)
Change from Baseline - Week 18 Number Analyzed 0 participants 3 participants 16 participants 19 participants
1.2  (2.06) 6.9  (7.21) 6.0  (6.94)
Change from Baseline - Week 24 Number Analyzed 0 participants 3 participants 16 participants 19 participants
2.5  (10.4) 5.2  (8.25) 4.8  (8.36)
6.Other Pre-specified Outcome
Title Change From Baseline in Percent Predicted Upright Maximum Inspiratory Pressure
Hide Description Change from Baseline in Percent Predicted Upright Maximum Inspiratory Pressure. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
Time Frame Baseline up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point. No patient had baseline assessment in group of BMN 701 5 mg/kg.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Hide Arm/Group Description:
BMN 701 5 mg/kg IV
BMN 701 10 mg/kg IV
BMN 701 20 mg/kg IV
Total Results
Overall Number of Participants Analyzed 0 3 16 22
Mean (Standard Deviation)
Unit of Measure: percentage of Predicted MIP
Baseline Number Analyzed 0 participants 3 participants 16 participants 19 participants
39.5  (21.87) 40.5  (25.01) 40.3  (23.97)
Change from baseline at week 6 Number Analyzed 0 participants 3 participants 16 participants 19 participants
1.7  (1.46) 5.3  (9.8) 4.7  (9.06)
Change from baseline at week 12 Number Analyzed 0 participants 3 participants 16 participants 19 participants
3.4  (6.74) 11.4  (10.92) 10.1  (10.65)
Change from baseline at week 18 Number Analyzed 0 participants 3 participants 15 participants 18 participants
2.9  (11.77) 14.5  (14.48) 12.6  (14.45)
Change from baseline at week 24 Number Analyzed 0 participants 3 participants 16 participants 19 participants
0.7  (6.69) 11.1  (8.31) 9.5  (8.81)
7.Other Pre-specified Outcome
Title Change From Baseline in Upright Maximum Ventilatory Volume
Hide Description Change from Baseline in Upright Maximum Ventilatory Volume. Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
Time Frame Baseline up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. As some patients did not attend or all tests were not completed at each visit, some time points had different numbers of patients analyzed. The total number of patients analyzed for each arm is listed as the maximum patients analyzed at any given time point. No patient had baseline assessment in group of BMN 701 5 mg/kg.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Hide Arm/Group Description:
BMN 701 5 mg/kg IV
BMN 701 10 mg/kg IV
BMN 701 20 mg/kg IV
Total Results
Overall Number of Participants Analyzed 0 3 16 22
Mean (Standard Deviation)
Unit of Measure: L/min
Baseline Number Analyzed 0 participants 3 participants 16 participants 19 participants
76  (41.04) 67.6  (25.9) 68.9  (27.5)
Change from Baseline - Week 6 Number Analyzed 0 participants 3 participants 16 participants 19 participants
-1.0  (0.35) 1.5  (11.1) 1.1  (10.18)
Change from Baseline - Week 12 Number Analyzed 0 participants 3 participants 16 participants 19 participants
-1.1  (2.73) 3.8  (11.64) 3.0  (10.82)
Change from Baseline - Week 18 Number Analyzed 0 participants 3 participants 15 participants 18 participants
-3.9  (2.86) 4.7  (10.89) 3.3  (10.46)
Change from Baseline - Week 24 Number Analyzed 0 participants 3 participants 15 participants 18 participants
-0.7  (9.1) 2.3  (10.71) 1.8  (10.28)
Time Frame 32 weeks (27 days of screening and baseline measurements + 24 weeks of treatment + 20 days of follow-up)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Hide Arm/Group Description BMN 701 5 mg/kg IV BMN 701 10 mg/kg IV BMN 701 20 mg/kg IV Total Results
All-Cause Mortality
BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)      0/3 (0.00%)      0/16 (0.00%)      0/22 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/3 (0.00%)      0/3 (0.00%)      4/16 (25.00%)      4/22 (18.18%)    
Immune system disorders         
Hypersensitivity  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Metabolism and nutrition disorders         
Hypoglycaemia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Psychiatric disorders         
Aggression  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Vascular disorders         
Hypertensive crisis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
1
Term from vocabulary, MedDRA 16.1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/3 (100.00%)      3/3 (100.00%)      15/16 (93.75%)      21/22 (95.45%)    
Cardiac disorders         
Bradycardia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Bundle branch block right  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Palpitations  1  0/3 (0.00%)  0 1/3 (33.33%)  1 3/16 (18.75%)  4 4/22 (18.18%)  5
Sinus tachycardia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Tachycardia  1  0/3 (0.00%)  0 1/3 (33.33%)  2 4/16 (25.00%)  16 5/22 (22.73%)  18
Eye disorders         
Diplopia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Vision blurred  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Gastrointestinal disorders         
Abdominal discomfort  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  2 2/22 (9.09%)  2
Abdominal distension  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Abdominal pain  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  2 1/22 (4.55%)  2
Abdominal pain upper  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  2 2/22 (9.09%)  2
Abdominal tenderness  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Constipation  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  2 2/22 (9.09%)  2
Diarrhoea  1  1/3 (33.33%)  1 0/3 (0.00%)  0 4/16 (25.00%)  5 5/22 (22.73%)  6
Dry mouth  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Dyspepsia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Dysphagia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Epigastric discomfort  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Gastritis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Gastrooesophageal reflux disease  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Haemorrhoids  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Nausea  1  0/3 (0.00%)  0 0/3 (0.00%)  0 8/16 (50.00%)  15 8/22 (36.36%)  15
Rectal haemorrhage  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Retching  1  1/3 (33.33%)  1 0/3 (0.00%)  0 0/16 (0.00%)  0 1/22 (4.55%)  1
Tongue discolouration  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Vomiting  1  1/3 (33.33%)  1 1/3 (33.33%)  1 5/16 (31.25%)  9 7/22 (31.82%)  11
General disorders         
Application site erythema  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Asthenia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Chest discomfort  1  1/3 (33.33%)  6 1/3 (33.33%)  2 5/16 (31.25%)  6 7/22 (31.82%)  14
Chills  1  0/3 (0.00%)  0 1/3 (33.33%)  1 3/16 (18.75%)  3 4/22 (18.18%)  4
Fatigue  1  1/3 (33.33%)  1 1/3 (33.33%)  1 0/16 (0.00%)  0 2/22 (9.09%)  2
Feeling cold  1  0/3 (0.00%)  0 1/3 (33.33%)  1 1/16 (6.25%)  1 2/22 (9.09%)  2
Feeling hot  1  0/3 (0.00%)  0 0/3 (0.00%)  0 3/16 (18.75%)  3 3/22 (13.64%)  3
Infusion site discomfort  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Local swelling  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  2 1/22 (4.55%)  2
Malaise  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  3 2/22 (9.09%)  3
Non-cardiac chest pain  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Oedema peripheral  1  0/3 (0.00%)  0 0/3 (0.00%)  0 3/16 (18.75%)  3 3/22 (13.64%)  3
Pain  1  0/3 (0.00%)  0 1/3 (33.33%)  1 1/16 (6.25%)  1 2/22 (9.09%)  2
Pyrexia  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Tenderness  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Thirst  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Vaccination site inflammation  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Vessel puncture site bruise  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Infections and infestations         
Candida infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Laryngitis  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Localised infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Lower respiratory tract infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  2 2/22 (9.09%)  2
Nasopharyngitis  1  1/3 (33.33%)  2 0/3 (0.00%)  0 2/16 (12.50%)  3 3/22 (13.64%)  5
Sinusitis  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Tooth abscess  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Upper respiratory tract infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  2 2/22 (9.09%)  2
Urinary tract infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Injury, poisoning and procedural complications         
Animal bite  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Arthropod bite  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Contusion  1  1/3 (33.33%)  1 1/3 (33.33%)  1 3/16 (18.75%)  4 5/22 (22.73%)  6
Fall  1  1/3 (33.33%)  2 3/3 (100.00%)  5 3/16 (18.75%)  4 7/22 (31.82%)  11
Infusion related reaction  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  2 2/22 (9.09%)  2
Laceration  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Post procedural haemorrhage  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Procedural pain  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Investigations         
Blood glucose decreased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Body temperature increased  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Cardiac murmur  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Complement factor decreased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Respiratory rate increased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  5 1/22 (4.55%)  5
Metabolism and nutrition disorders         
Dehydration  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Hyperglycaemia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Hyperphagia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Hypoglycaemia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 14/16 (87.50%)  41 14/22 (63.64%)  41
Lactic acidosis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Musculoskeletal and connective tissue disorders         
Arthralgia  1  0/3 (0.00%)  0 1/3 (33.33%)  1 1/16 (6.25%)  1 2/22 (9.09%)  2
Back pain  1  2/3 (66.67%)  4 0/3 (0.00%)  0 2/16 (12.50%)  3 4/22 (18.18%)  7
Joint swelling  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Muscle spasms  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Neck pain  1  1/3 (33.33%)  2 0/3 (0.00%)  0 1/16 (6.25%)  1 2/22 (9.09%)  3
Pain in extremity  1  0/3 (0.00%)  0 0/3 (0.00%)  0 3/16 (18.75%)  4 3/22 (13.64%)  4
Nervous system disorders         
Disturbance in attention  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Dizziness  1  0/3 (0.00%)  0 1/3 (33.33%)  1 6/16 (37.50%)  9 7/22 (31.82%)  10
Headache  1  1/3 (33.33%)  2 2/3 (66.67%)  8 6/16 (37.50%)  27 9/22 (40.91%)  37
Hypoaesthesia  1  1/3 (33.33%)  1 0/3 (0.00%)  0 0/16 (0.00%)  0 1/22 (4.55%)  1
Lethargy  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Paraesthesia  1  1/3 (33.33%)  1 0/3 (0.00%)  0 1/16 (6.25%)  1 2/22 (9.09%)  2
Tremor  1  0/3 (0.00%)  0 0/3 (0.00%)  0 3/16 (18.75%)  4 3/22 (13.64%)  4
Psychiatric disorders         
Anxiety  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Confusional state  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Depression  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Depressive symptom  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Nervousness  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  2 2/22 (9.09%)  2
Renal and urinary disorders         
Haematuria  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Reproductive system and breast disorders         
Dysmenorrhoea  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Vaginal haemorrhage  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Respiratory, thoracic and mediastinal disorders         
Cough  1  1/3 (33.33%)  1 1/3 (33.33%)  1 2/16 (12.50%)  2 4/22 (18.18%)  4
Dyspnoea  1  0/3 (0.00%)  0 1/3 (33.33%)  2 5/16 (31.25%)  10 6/22 (27.27%)  12
Dyspnoea exertional  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Hypopnoea  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Laryngeal oedema  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Nasal congestion  1  1/3 (33.33%)  1 0/3 (0.00%)  0 1/16 (6.25%)  1 2/22 (9.09%)  2
Oropharyngeal pain  1  1/3 (33.33%)  1 0/3 (0.00%)  0 3/16 (18.75%)  3 4/22 (18.18%)  4
Pharyngeal oedema  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Respiratory distress  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Rhinorrhoea  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Throat irritation  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Skin and subcutaneous tissue disorders         
Cold sweat  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  2 1/22 (4.55%)  2
Dermatitis allergic  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Eczema  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  2 2/22 (9.09%)  2
Hyperhidrosis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  2 1/22 (4.55%)  2
Pruritus  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  4 2/22 (9.09%)  4
Rash  1  0/3 (0.00%)  0 0/3 (0.00%)  0 4/16 (25.00%)  5 4/22 (18.18%)  5
Rash erythematous  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Rash maculo-papular  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Rash pruritic  1  0/3 (0.00%)  0 1/3 (33.33%)  1 1/16 (6.25%)  4 2/22 (9.09%)  5
Skin discolouration  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Urticaria  1  0/3 (0.00%)  0 1/3 (33.33%)  1 1/16 (6.25%)  13 2/22 (9.09%)  14
Vascular disorders         
Flushing  1  1/3 (33.33%)  5 0/3 (0.00%)  0 1/16 (6.25%)  1 2/22 (9.09%)  6
Haematoma  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Hot flush  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Hypertension  1  0/3 (0.00%)  0 0/3 (0.00%)  0 3/16 (18.75%)  5 3/22 (13.64%)  5
Hypotension  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Pallor  1  0/3 (0.00%)  0 0/3 (0.00%)  0 2/16 (12.50%)  2 2/22 (9.09%)  2
Poor venous access  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/16 (0.00%)  0 1/22 (4.55%)  1
Systolic hypertension  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
Venous thrombosis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/16 (6.25%)  1 1/22 (4.55%)  1
1
Term from vocabulary, MedDRA 16.1
Indicates events were collected by systematic assessment
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: Manager, Clinical Operations
Organization: BioMarin Pharmaceutical Inc.
Phone: 415-455-7448
EMail: Slava.Titov@bmrn.com
Layout table for additonal information
Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01230801     History of Changes
Other Study ID Numbers: POM-001
2010-023561-22 ( EudraCT Number )
First Submitted: October 27, 2010
First Posted: October 29, 2010
Results First Submitted: March 19, 2018
Results First Posted: June 11, 2018
Last Update Posted: June 11, 2018