Extension Study for Patients Who Have Participated in a BMN 701 Study

Sponsor
BioMarin Pharmaceutical (Industry)
Overall Status
Terminated
CT.gov ID
NCT01435772
Collaborator
(none)
21
12
3
60.8
1.8
0

Study Details

Study Description

Brief Summary

This is a Phase 2 open-label, multiple dose study of BMN 701 administered by IV infusion every 2 weeks (qow) to patients with late-onset Pompe disease.

Condition or Disease Intervention/Treatment Phase
  • Biological: BMN 701
Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
21 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study
Actual Study Start Date :
Aug 15, 2011
Actual Primary Completion Date :
Sep 9, 2016
Actual Study Completion Date :
Sep 9, 2016

Arms and Interventions

Arm Intervention/Treatment
Experimental: BMN 701 20mg/kg

BMN 701 20mg/kg IV every other week

Biological: BMN 701
GILT-tagged recombinant human GAA

Experimental: BMN 701 10mg/kg

BMN 701 10mg/kg IV every other week

Biological: BMN 701
GILT-tagged recombinant human GAA

Experimental: BMN 701 5mg/kg

BMN 701 5mg/kg IV every other week

Biological: BMN 701
GILT-tagged recombinant human GAA

Outcome Measures

Primary Outcome Measures

  1. Number of Participants With a Positive Anti-BMN 701 Antibody [Baseline, Week 144]

    Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples.

  2. Number of Participants With a Positive Anti-BMN 701 Antibody Response [Baseline, Week 144]

    Status of Anti-IGF-I antibody is corresponding to the test results of blood samples

  3. Number of Participants With a Positive Anti-BMN 701 Antibody Response [Baseline, Week 144]

    Status of Anti-IGF-II antibody is corresponding to the test results of blood samples

Secondary Outcome Measures

  1. Percent Predicted Maximal Inspiratory Pressure (MIP) [Baseline, Week 144]

    Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure

  2. Percent Predicted Maximum Expiratory Pressure (MEP) [Baseline, Week 144]

    Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure

  3. 6 Minutes Walk Test (Meters) [Baseline, Week 144]

    Distance walked within 6 minutes

  4. Maximum Voluntary Ventilation (MVV) [Baseline, Week 144]

    Pulmonary function test: Maximum Voluntary Ventilation (MVV)

  5. Percent Predicted Upright Forced Vital Capacity (FVC) [Baseline, Week 144]

    Pulmonary function test: Percent Predicted Upright Forced Vital Capacity

  6. Change From Baseline in Urine Tetrasaccharide Concentration at Week 144 [Baseline, Week 144]

    Change from Baseline in Urine Tetrasaccharide Concentration at Week 144

  7. Plasma IGF-I Concentration [Baseline, Week 144]

    Plasma IGF-I concentration from lab

  8. Plasma IGF-II Concentration [Baseline, Week 144]

    Plasma IGF-II concentration from lab

  9. Insulin-like Growth Factor Binding Protein 3 (IGFBP3) [Baseline, Week 144]

    insulin-like growth factor binding protein 3 from lab

Eligibility Criteria

Criteria

Ages Eligible for Study:
13 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
  • Have completed a prior BMN 701 clinical development study;

  • Have provided written informed consent after the nature of the study has been explained prior to performance of any study-related procedures. Minors may participate as long as they provide written assent after the nature of the study has been explained to them and after their parent, or legal guardian has provided written informed consent, prior to the performance of any study-related procedures;

  • Have been diagnosed with late-onset Pompe Disease, based on the entry criteria of a prior BMN 701 study;

  • If sexually active, be willing to use 2 known effective methods of contraception from Screening until 4 months after the last dose of study-drug;

  • If female, and not considered to be of childbearing potential, be at least 2 years post-menopausal, or have had tubal ligation at least 1 year prior to screening, or have had a total hysterectomy;

  • If female, and of childbearing potential, have a negative pregnancy test during the Screening Period and at the Baseline visit, and be willing to have additional pregnancy tests during the study;

  • Have the ability to comply with the protocol requirements, in the opinion of the Investigator.

Exclusion Criteria:
  • Have received any experimental or approved therapy for Pompe disease, other than BMN 701, subsequent to completion of a BMN 701 study and prior to entry into POM-002;

  • Have received, or are anticipated to receive, any investigational medication, other than BMN 701, within 30 days prior to the first dose of study-drug;

  • Are breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study;

  • Have a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the patient's ability to comply with the protocol requirements or compromise the patient's well being or safety.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Univ of California San Diego School of Medicine San Diego California United States 92103
2 University of Florida College of Medicine Gainesville Florida United States 32610
3 Tampa General Hospital Tampa Florida United States 33606
4 University of Kansas Medical Center Kansas City Kansas United States 66160
5 Royal Brisbane and Women's Hospital Herston Queensland Australia 4029
6 Royal Adelaide Hospital North Adelaide South Australia Australia 5006
7 Hôpital Pitié-Salpêtrière Paris France 75013
8 Villa Metabolica, ZKJM MC University Mainz Mainz Germany 55131
9 Auckland City and Starship Children's Hospital Auckland New Zealand 1142
10 University Hospitals Birmingham NHS Foundation Trust Birmingham United Kingdom B15 2TH
11 Royal Free Hospital London United Kingdom NW3 2QG
12 Salford Royal NHS Foundation Trust Salford United Kingdom M5 5AP

Sponsors and Collaborators

  • BioMarin Pharmaceutical

Investigators

  • Study Director: Medical Monitor, BioMarin Pharmaceutical

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01435772
Other Study ID Numbers:
  • POM-002
  • 2011-001805-28
First Posted:
Sep 19, 2011
Last Update Posted:
May 22, 2018
Last Verified:
Apr 1, 2018
Additional relevant MeSH terms:

Study Results

Participant Flow

Recruitment Details
Pre-assignment Detail
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Period Title: Overall Study
STARTED 3 3 15
COMPLETED 0 0 0
NOT COMPLETED 3 3 15

Baseline Characteristics

Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg Total of all reporting groups
Overall Participants 3 3 15 21
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]
52.3
(6.66)
42.7
(13.05)
50.1
(5.28)
49.4
(7.03)
Age, Customized (Count of Participants)
18-65 years
3
100%
3
100%
15
100%
21
100%
Sex: Female, Male (Count of Participants)
Female
0
0%
2
66.7%
6
40%
8
38.1%
Male
3
100%
1
33.3%
9
60%
13
61.9%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino
0
0%
1
33.3%
0
0%
1
4.8%
Not Hispanic or Latino
3
100%
2
66.7%
15
100%
20
95.2%
Unknown or Not Reported
0
0%
0
0%
0
0%
0
0%
Race/Ethnicity, Customized (Count of Participants)
American Indian or Alaska Native
0
0%
0
0%
0
0%
0
0%
Asian
0
0%
0
0%
0
0%
0
0%
Native Hawaiian or Other Pacific Islander
0
0%
0
0%
0
0%
0
0%
Black or African American
0
0%
0
0%
0
0%
0
0%
White
3
100%
3
100%
14
93.3%
20
95.2%
Other
0
0%
0
0%
1
6.7%
1
4.8%
Region of Enrollment (Count of Participants)
Australia
1
33.3%
1
33.3%
4
26.7%
6
28.6%
France
0
0%
0
0%
1
6.7%
1
4.8%
Germany
0
0%
0
0%
1
6.7%
1
4.8%
United Kingdom
0
0%
0
0%
6
40%
6
28.6%
United States
2
66.7%
2
66.7%
3
20%
7
33.3%

Outcome Measures

1. Primary Outcome
Title Number of Participants With a Positive Anti-BMN 701 Antibody
Description Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples.
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set. Please note the overall number of participants reflects the number of patients in that arm, while the outcome measurement number of participants reflects the number of patients for which data is available and analyzed.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 3 3 15
Baseline
2
66.7%
1
33.3%
11
73.3%
Week 144
2
66.7%
2
66.7%
10
66.7%
2. Primary Outcome
Title Number of Participants With a Positive Anti-BMN 701 Antibody Response
Description Status of Anti-IGF-I antibody is corresponding to the test results of blood samples
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 3 3 15
Baseline
0
0%
2
66.7%
0
0%
Week 144
0
0%
1
33.3%
5
33.3%
3. Primary Outcome
Title Number of Participants With a Positive Anti-BMN 701 Antibody Response
Description Status of Anti-IGF-II antibody is corresponding to the test results of blood samples
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 3 3 15
Baseline
1
33.3%
2
66.7%
2
13.3%
Week 144
0
0%
2
66.7%
7
46.7%
4. Secondary Outcome
Title Percent Predicted Maximal Inspiratory Pressure (MIP)
Description Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set. No data collected for outcomes with 0 participants analyzed.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 0 3 15
Baseline
39.53
(21.868)
40.2
(25.863)
Change from Baseline to Week 144
21.17
(14.537)
18.48
(18.599)
5. Secondary Outcome
Title Percent Predicted Maximum Expiratory Pressure (MEP)
Description Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set. No data collected for outcomes with 0 participants analyzed.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 0 3 15
Baseline
31.07
(6.641)
36.7
(15.939)
Change from Baseline to Week 144
-0.28
(5.841)
5.11
(16.395)
6. Secondary Outcome
Title 6 Minutes Walk Test (Meters)
Description Distance walked within 6 minutes
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 3 3 15
Baseline
334
(227.119)
360
(51.403)
363.8
(157.818)
Change from Baseline to Week 144
-36
(90.508)
-120.75
(67.529)
9.5
(77.572)
7. Secondary Outcome
Title Maximum Voluntary Ventilation (MVV)
Description Pulmonary function test: Maximum Voluntary Ventilation (MVV)
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set. No data collected for outcomes with 0 participants analyzed.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 0 3 15
Baseline
76
(41.037)
68.08
(26.74)
Change from Baseline to Week 144
13.5
(13.435)
2.24
(11.635)
8. Secondary Outcome
Title Percent Predicted Upright Forced Vital Capacity (FVC)
Description Pulmonary function test: Percent Predicted Upright Forced Vital Capacity
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 3 3 15
Baseline
69.33
(19.732)
67.33
(26.577)
57.13
(18.677)
Change from Baseline to Week 144
-8.33
(7.095)
-0.5
(4.95)
-2
(8)
9. Secondary Outcome
Title Percent Predicted Upright Forced Vital Capacity (FVC)
Description Pulmonary function test: Percent Predicted Upright Forced Vital capacity
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 3 3 15
Baseline
36.67
(17.954)
47.67
(32.578)
44.25
(21.55)
Change from Baseline to Week 144
-4.33
(9.452)
4.5
(26.163)
-3.43
(6.901)
10. Secondary Outcome
Title Change From Baseline in Urine Tetrasaccharide Concentration at Week 144
Description Change from Baseline in Urine Tetrasaccharide Concentration at Week 144
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set. No data collected for outcomes with 0 participants analyzed.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 0 2 10
Mean (Standard Deviation) [mmol/mol]
-0.2
(3.394)
-1.07
(0.975)
11. Secondary Outcome
Title Plasma IGF-I Concentration
Description Plasma IGF-I concentration from lab
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set. No data collected for outcomes with 0 participants analyzed. Please note: the overall number of participants reflects the number of patients in that arm, while the outcome measurement number of participants reflects the number of patients for which data is available and analyzed.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 2 2 15
Baseline
20.26
(6.465)
Week 144
26.98
(1.379)
18.4
(7.078)
17.62
(5.878)
12. Secondary Outcome
Title Plasma IGF-II Concentration
Description Plasma IGF-II concentration from lab
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set. No data collected for outcomes with 0 participants analyzed. Please note: the overall number of participants reflects the number of patients in that arm, while the outcome measurement number of participants reflects the number of patients for which data is available and analyzed.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 3 3 15
Baseline
702
(0)
496.83
(113.417)
Week 144
467.67
(117.142)
559.5
(140.714)
698.82
(228.948)
13. Secondary Outcome
Title Insulin-like Growth Factor Binding Protein 3 (IGFBP3)
Description insulin-like growth factor binding protein 3 from lab
Time Frame Baseline, Week 144

Outcome Measure Data

Analysis Population Description
Full Analysis Set. No data collected for outcomes with 0 participants analyzed. Please note: the overall number of participants reflects the number of patients in that arm, while the outcome measurement number of participants reflects the number of patients for which data is available and analyzed.
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg
Arm/Group Description IV infusion at dose levels of 5 mg/kg IV infusion at dose levels of 10 mg/kg IV infusion at dose levels of 20 mg/kg
Measure Participants 3 2 15
Baseline
150.45
(23.914)
Week 144
157.33
(41.477)
209
(39.598)
146.64
(33.587)

Adverse Events

Time Frame 284.9 weeks (maximum exposure) +4 weeks follow up
Adverse Event Reporting Description
Arm/Group Title BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
Arm/Group Description BMN 701 5 mg/kg BMN 701 10 mg/kg BMN 701 20 mg/kg Total
All Cause Mortality
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%) 0/3 (0%) 0/15 (0%) 0/21 (0%)
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 2/3 (66.7%) 1/3 (33.3%) 7/15 (46.7%) 10/21 (47.6%)
Cardiac disorders
Cardiac failure acute 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Myocardial infarction 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Gastrointestinal disorders
Gastrooesophageal reflux disease 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
General disorders
Fatigue 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Immune system disorders
Anaphylactoid reaction 1/3 (33.3%) 2 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 2
Infections and infestations
Pneumonia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Injury, poisoning and procedural complications
Fall 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Tendon rupture 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Metabolism and nutrition disorders
Hypoglycaemia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Nervous system disorders
Cerebrovascular accident 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Presyncope 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Renal and urinary disorders
Prerenal failure 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Respiratory, thoracic and mediastinal disorders
Chronic respiratory failure 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Respiratory failure 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Stridor 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Other (Not Including 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 3/3 (100%) 3/3 (100%) 15/15 (100%) 21/21 (100%)
Blood and lymphatic system disorders
Anaemia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Iron deficiency anaemia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Cardiac disorders
Bradycardia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Cardiac failure 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Diastolic dysfunction 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Left ventricular hypertrophy 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Sinus tachycardia 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 4 3/21 (14.3%) 4
Tachycardia 1/3 (33.3%) 1 1/3 (33.3%) 1 5/15 (33.3%) 49 7/21 (33.3%) 51
Ear and labyrinth disorders
Ear discomfort 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Tinnitus 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Vertigo positional 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Endocrine disorders
Androgen deficiency 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Eye disorders
Blindness transient 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Ocular hyperaemia 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Visual impairment 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Gastrointestinal disorders
Abdominal discomfort 1/3 (33.3%) 1 1/3 (33.3%) 1 1/15 (6.7%) 3 3/21 (14.3%) 5
Abdominal distension 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Abdominal pain 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 3 2/21 (9.5%) 3
Abdominal pain lower 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Abdominal pain upper 1/3 (33.3%) 1 1/3 (33.3%) 1 3/15 (20%) 5 5/21 (23.8%) 7
Abdominal tenderness 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Constipation 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 4 3/21 (14.3%) 4
Dental caries 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Diarrhoea 0/3 (0%) 0 0/3 (0%) 0 7/15 (46.7%) 13 7/21 (33.3%) 13
Dyspepsia 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Flatulence 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Gastrooesophageal reflux disease 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 3 3/21 (14.3%) 3
Irritable bowel syndrome 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 3 2/21 (9.5%) 3
Nausea 1/3 (33.3%) 1 1/3 (33.3%) 1 6/15 (40%) 29 8/21 (38.1%) 31
Painful defaecation 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Rectal haemorrhage 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Salivary hypersecretion 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Tooth loss 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Toothache 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 5 1/21 (4.8%) 5
Vomiting 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 4 3/21 (14.3%) 4
General disorders
Application site erythema 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Asthenia 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Catheter site erythema 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Catheter site inflammation 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Catheter site pain 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Chest discomfort 1/3 (33.3%) 1 0/3 (0%) 0 5/15 (33.3%) 19 6/21 (28.6%) 20
Chest pain 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Chills 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 5 3/21 (14.3%) 5
Discomfort 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Extravasation 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Fatigue 2/3 (66.7%) 18 0/3 (0%) 0 8/15 (53.3%) 16 10/21 (47.6%) 34
Feeling abnormal 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Feeling cold 0/3 (0%) 0 1/3 (33.3%) 1 2/15 (13.3%) 10 3/21 (14.3%) 11
Feeling hot 0/3 (0%) 0 1/3 (33.3%) 1 3/15 (20%) 7 4/21 (19%) 8
Feeling of body temperature change 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Gait disturbance 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Influenza like illness 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Infusion site extravasation 1/3 (33.3%) 2 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 2
Infusion site pain 2/3 (66.7%) 2 0/3 (0%) 0 0/15 (0%) 0 2/21 (9.5%) 2
Infusion site swelling 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Malaise 1/3 (33.3%) 1 0/3 (0%) 0 4/15 (26.7%) 5 5/21 (23.8%) 6
Non-cardiac chest pain 0/3 (0%) 0 1/3 (33.3%) 1 2/15 (13.3%) 2 3/21 (14.3%) 3
Oedema peripheral 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 8 3/21 (14.3%) 8
Pain 2/3 (66.7%) 2 0/3 (0%) 0 1/15 (6.7%) 2 3/21 (14.3%) 4
Peripheral swelling 1/3 (33.3%) 1 1/3 (33.3%) 1 3/15 (20%) 3 5/21 (23.8%) 5
Pyrexia 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Temperature intolerance 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Tenderness 0/3 (0%) 0 1/3 (33.3%) 2 0/15 (0%) 0 1/21 (4.8%) 2
Vessel puncture site bruise 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Hepatobiliary disorders
Cholelithiasis 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Immune system disorders
Seasonal allergy 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 2 1/21 (4.8%) 2
Infections and infestations
Blister infected 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Bronchitis 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Ear infection 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Fungal infection 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Gastroenteritis viral 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Herpes simplex 1/3 (33.3%) 3 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 3
Influenza 1/3 (33.3%) 1 0/3 (0%) 0 3/15 (20%) 3 4/21 (19%) 4
Lower respiratory tract infection 1/3 (33.3%) 1 1/3 (33.3%) 1 4/15 (26.7%) 9 6/21 (28.6%) 11
Lower respiratory tract infection viral 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Nasopharyngitis 0/3 (0%) 0 1/3 (33.3%) 1 4/15 (26.7%) 7 5/21 (23.8%) 8
Rhinitis 1/3 (33.3%) 1 0/3 (0%) 0 2/15 (13.3%) 2 3/21 (14.3%) 3
Sinusitis 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Upper respiratory tract infection 1/3 (33.3%) 3 2/3 (66.7%) 6 7/15 (46.7%) 11 10/21 (47.6%) 20
Urinary tract infection 0/3 (0%) 0 1/3 (33.3%) 2 1/15 (6.7%) 1 2/21 (9.5%) 3
Viral infection 0/3 (0%) 0 1/3 (33.3%) 2 1/15 (6.7%) 2 2/21 (9.5%) 4
Viral upper respiratory tract infection 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 2 1/21 (4.8%) 2
Injury, poisoning and procedural complications
Contusion 2/3 (66.7%) 5 1/3 (33.3%) 1 6/15 (40%) 9 9/21 (42.9%) 15
Epicondylitis 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Eye contusion 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Fall 3/3 (100%) 22 2/3 (66.7%) 5 7/15 (46.7%) 26 12/21 (57.1%) 53
Head injury 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Joint injury 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Laceration 1/3 (33.3%) 1 0/3 (0%) 0 1/15 (6.7%) 1 2/21 (9.5%) 2
Muscle contusion 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Muscle strain 1/3 (33.3%) 1 0/3 (0%) 0 1/15 (6.7%) 1 2/21 (9.5%) 2
Post procedural contusion 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Post procedural haemorrhage 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Procedural pain 1/3 (33.3%) 2 0/3 (0%) 0 2/15 (13.3%) 2 3/21 (14.3%) 4
Skin abrasion 2/3 (66.7%) 5 1/3 (33.3%) 1 1/15 (6.7%) 1 4/21 (19%) 7
Skin wound 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Tendon injury 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Thermal burn 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Tooth fracture 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 3 1/21 (4.8%) 3
Wound 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Investigations
Ammonia increased 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Bacterial test positive 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 2 1/21 (4.8%) 2
Blood calcium decreased 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Blood glucose decreased 0/3 (0%) 0 0/3 (0%) 0 7/15 (46.7%) 14 7/21 (33.3%) 14
Blood immunoglobulin E increased 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Cardiac murmur 1/3 (33.3%) 1 0/3 (0%) 0 1/15 (6.7%) 1 2/21 (9.5%) 2
Complement factor increased 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Lipase increased 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Pulmonary physical examination abnormal 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Troponin increased 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Tryptase increased 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Metabolism and nutrition disorders
Dehydration 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 2 1/21 (4.8%) 2
Glucose tolerance impaired 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Hyperglycaemia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 2 1/21 (4.8%) 2
Hyperkalaemia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Hypoglycaemia 1/3 (33.3%) 19 1/3 (33.3%) 30 14/15 (93.3%) 106 16/21 (76.2%) 155
Iron deficiency 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Musculoskeletal and connective tissue disorders
Arthralgia 2/3 (66.7%) 8 1/3 (33.3%) 1 7/15 (46.7%) 18 10/21 (47.6%) 27
Back pain 3/3 (100%) 6 1/3 (33.3%) 1 7/15 (46.7%) 10 11/21 (52.4%) 17
Bursitis 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Flank pain 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Groin pain 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Intervertebral disc protrusion 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Joint swelling 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Muscle spasms 1/3 (33.3%) 1 0/3 (0%) 0 3/15 (20%) 9 4/21 (19%) 10
Muscle twitching 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Muscular weakness 1/3 (33.3%) 1 0/3 (0%) 0 2/15 (13.3%) 2 3/21 (14.3%) 3
Musculoskeletal chest pain 1/3 (33.3%) 1 0/3 (0%) 0 1/15 (6.7%) 1 2/21 (9.5%) 2
Musculoskeletal discomfort 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Musculoskeletal pain 1/3 (33.3%) 1 0/3 (0%) 0 2/15 (13.3%) 3 3/21 (14.3%) 4
Myalgia 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 4 3/21 (14.3%) 4
Neck pain 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 4 3/21 (14.3%) 4
Osteopenia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Pain in extremity 1/3 (33.3%) 1 1/3 (33.3%) 1 8/15 (53.3%) 14 10/21 (47.6%) 16
Spinal pain 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Tendonitis 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Nervous system disorders
Balance disorder 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Burning sensation 0/3 (0%) 0 1/3 (33.3%) 1 2/15 (13.3%) 2 3/21 (14.3%) 3
Dizziness 1/3 (33.3%) 1 1/3 (33.3%) 5 8/15 (53.3%) 11 10/21 (47.6%) 17
Headache 2/3 (66.7%) 7 1/3 (33.3%) 4 9/15 (60%) 30 12/21 (57.1%) 41
Hypoaesthesia 1/3 (33.3%) 1 1/3 (33.3%) 1 3/15 (20%) 3 5/21 (23.8%) 5
Lethargy 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Migraine 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Paraesthesia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 2 1/21 (4.8%) 2
Presyncope 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Restless legs syndrome 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Sciatica 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Sinus headache 1/3 (33.3%) 2 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 2
Tremor 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Tunnel vision 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Product Issues
Device occlusion 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Psychiatric disorders
Anxiety 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 2 1/21 (4.8%) 2
Confusional state 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Insomnia 1/3 (33.3%) 2 1/3 (33.3%) 1 2/15 (13.3%) 2 4/21 (19%) 5
Mood swings 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Nervousness 1/3 (33.3%) 4 1/3 (33.3%) 1 4/15 (26.7%) 6 6/21 (28.6%) 11
Restlessness 1/3 (33.3%) 1 0/3 (0%) 0 2/15 (13.3%) 2 3/21 (14.3%) 3
Sleep terror 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Renal and urinary disorders
Haematuria 0/3 (0%) 0 1/3 (33.3%) 1 2/15 (13.3%) 2 3/21 (14.3%) 3
Renal impairment 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Respiratory, thoracic and mediastinal disorders
Asthma 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Chronic respiratory failure 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Cough 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 8 3/21 (14.3%) 8
Dyspnoea 1/3 (33.3%) 1 0/3 (0%) 0 9/15 (60%) 26 10/21 (47.6%) 27
Epistaxis 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 2 1/21 (4.8%) 2
Hypoxia 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 2 1/21 (4.8%) 2
Nasal congestion 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Oropharyngeal pain 1/3 (33.3%) 1 1/3 (33.3%) 2 5/15 (33.3%) 12 7/21 (33.3%) 15
Paranasal sinus hypersecretion 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Pleuritic pain 0/3 (0%) 0 0/3 (0%) 0 2/15 (13.3%) 2 2/21 (9.5%) 2
Productive cough 0/3 (0%) 0 1/3 (33.3%) 2 0/15 (0%) 0 1/21 (4.8%) 2
Pulmonary congestion 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Rales 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Respiratory failure 1/3 (33.3%) 1 0/3 (0%) 0 1/15 (6.7%) 1 2/21 (9.5%) 2
Tachypnoea 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 40 1/21 (4.8%) 40
Wheezing 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 4 1/21 (4.8%) 4
Skin and subcutaneous tissue disorders
Angioedema 1/3 (33.3%) 1 0/3 (0%) 0 1/15 (6.7%) 1 2/21 (9.5%) 2
Cold sweat 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 3 3/21 (14.3%) 3
Decubitus ulcer 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Dermal cyst 1/3 (33.3%) 4 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 4
Erythema 1/3 (33.3%) 1 1/3 (33.3%) 1 3/15 (20%) 4 5/21 (23.8%) 6
Hyperhidrosis 0/3 (0%) 0 0/3 (0%) 0 4/15 (26.7%) 14 4/21 (19%) 14
Night sweats 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Pruritus 1/3 (33.3%) 2 0/3 (0%) 0 2/15 (13.3%) 3 3/21 (14.3%) 5
Rash 1/3 (33.3%) 1 2/3 (66.7%) 5 1/15 (6.7%) 1 4/21 (19%) 7
Rash macular 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Rash papular 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Rash pruritic 1/3 (33.3%) 2 1/3 (33.3%) 1 0/15 (0%) 0 2/21 (9.5%) 3
Rosacea 1/3 (33.3%) 1 0/3 (0%) 0 0/15 (0%) 0 1/21 (4.8%) 1
Skin lesion 0/3 (0%) 0 1/3 (33.3%) 1 0/15 (0%) 0 1/21 (4.8%) 1
Skin mass 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Skin ulcer 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 1 1/21 (4.8%) 1
Urticaria 3/3 (100%) 8 1/3 (33.3%) 1 2/15 (13.3%) 3 6/21 (28.6%) 12
Vascular disorders
Flushing 0/3 (0%) 0 0/3 (0%) 0 4/15 (26.7%) 8 4/21 (19%) 8
Haematoma 1/3 (33.3%) 1 0/3 (0%) 0 1/15 (6.7%) 1 2/21 (9.5%) 2
Hot flush 1/3 (33.3%) 1 1/3 (33.3%) 1 4/15 (26.7%) 4 6/21 (28.6%) 6
Hypertension 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 6 3/21 (14.3%) 6
Hypotension 0/3 (0%) 0 0/3 (0%) 0 3/15 (20%) 6 3/21 (14.3%) 6
Pallor 0/3 (0%) 0 0/3 (0%) 0 1/15 (6.7%) 3 1/21 (4.8%) 3

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT 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

Name/Title Debra Lounsbury, Principal Scientist, Clinical Sciences
Organization BioMarin Pharmaceutical Inc.
Phone 415-506-6348
Email DLounsbury@bmrn.com
Responsible Party:
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01435772
Other Study ID Numbers:
  • POM-002
  • 2011-001805-28
First Posted:
Sep 19, 2011
Last Update Posted:
May 22, 2018
Last Verified:
Apr 1, 2018