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Safety and Exercise Study of Two Doses of BMN 110 for 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: NCT01609062
Recruitment Status : Terminated
First Posted : May 31, 2012
Results First Posted : February 1, 2016
Last Update Posted : February 1, 2016
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Mucopolysaccharidosis IVA
Morquio A Syndrome
MPS IVA
Intervention Drug: BMN 110
Enrollment 25
Recruitment Details  
Pre-assignment Details  
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week
Hide Arm/Group Description BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase. BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Period Title: Primary Phase
Started 15 10
Completed 15 10
Not Completed 0 0
Period Title: Extension Phase
Started 15 10
Completed 0 0 [1]
Not Completed 15 10
Reason Not Completed
Terminated by Sponsor             14             9
Discontinued drug and then was pregnant             1             0
Anticipated surgery & transition to EAP             0             1
[1]
EAP = Expanded Access Program
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week Total
Hide Arm/Group Description BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase. BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase. Total of all reporting groups
Overall Number of Baseline Participants 15 10 25
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 15 participants 10 participants 25 participants
14.9  (9.32) 12.0  (3.16) 13.7  (7.52)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 15 participants 10 participants 25 participants
7 to 11 years 9 5 14
12 to 18 years 3 5 8
>=19 years 3 0 3
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 10 participants 25 participants
Female
12
  80.0%
4
  40.0%
16
  64.0%
Male
3
  20.0%
6
  60.0%
9
  36.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 10 participants 25 participants
Hispanic or Latino
4
  26.7%
2
  20.0%
6
  24.0%
Not Hispanic or Latino
11
  73.3%
8
  80.0%
19
  76.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 15 participants 10 participants 25 participants
American Indian or Alaska Native 0 0 0
Asian 0 0 0
Native Hawaiian or Other Pacific Islander 0 0 0
Black or African American 0 0 0
White 14 9 23
Other 1 1 2
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 15 participants 10 participants 25 participants
Canada 5 1 6
Germany 0 3 3
United Kingdom 1 1 2
United States 9 5 14
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 192 weeks or ETV (early termination visit)
Hide 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 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 15 10 25
Measure Type: Number
Unit of Measure: participants
Serious Adverse Events 3 2 5
Other (Not Including Serious) Adverse Events 15 10 25
2.Secondary Outcome
Title 6-minute Walk Test (6MWT)
Hide Description Change from baseline to Week 12, 24, and 52 as measured in distance walked (meters) in 6MWT.
Time Frame Baseline, Week 12, 24, and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Modified Intent-to-Treat Analysis Set
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 15 10 25
Mean (Standard Deviation)
Unit of Measure: meters
Week 12 14.7  (30.90) -8.9  (83.52) 5.3  (57.55)
Week 24 -13.0  (54.12) 0.1  (34.70) -7.8  (46.94)
Week 52 9.0  (48.41) 31.8  (54.56) 18.5  (51.19)
3.Secondary Outcome
Title 3-minute Stair Climb Test (3MSCT)
Hide Description Change from baseline to Week 12, 24, and 52 as measured in speed (stairs/min) in 3MSCT.
Time Frame Baseline, Week 12, 24, and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Modified Intent-to-Treat Analysis Set
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 15 10 25
Mean (Standard Deviation)
Unit of Measure: stairs/min
Week 12 -3.7  (13.16) 1.7  (11.28) -1.5  (12.49)
Week 24 -2.2  (11.01) 12.5  (15.26) 3.9  (14.65)
Week 52 -4.4  (9.82) 10.0  (16.88) 1.3  (14.66)
4.Secondary Outcome
Title Respiratory Function Test (MVV and FVC)
Hide Description

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

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

Time Frame Baseline, Week 12, 24, and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Modified Intent-to-treat Analysis Set
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 15 10 25
Mean (Standard Deviation)
Unit of Measure: Percent Change
MVV Week 12 12.8  (21.77) 32.4  (140.73) 21.7  (94.00)
MVV Week 24 8.8  (17.20) -0.6  (27.00) 4.3  (22.34)
MVV Week 52 2.2  (41.24) -17.8  (38.61) -6.9  (40.43)
FVC Week 12 1.7  (8.77) 3.2  (10.63) 2.3  (9.39)
FVC Week 24 3.4  (9.01) 13.0  (16.30) 7.4  (13.16)
FVC Week 52 4.8  (12.68) 15.1  (15.94) 8.9  (14.69)
5.Secondary Outcome
Title Normalized Urine Keratan Sulfate (uKS)
Hide Description

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

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

Time Frame Baseline, Week 12, 24, and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Modified Intent-to-treat Analysis Set
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 15 10 25
Mean (Standard Deviation)
Unit of Measure: Percent Change
Week 12 (%) -27.6  (25.10) -50.9  (14.00) -36.7  (24.07)
Week 24 (%) -37.4  (23.21) -55.5  (12.62) -44.5  (21.39)
Week 52 (%) -20.5  (36.95) -52.8  (5.74) -31.3  (33.69)
6.Secondary Outcome
Title Cardiopulmonary Exercise Testing (CPET) - Duration of Exercise
Hide Description

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

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

Time Frame Baseline, Week 25 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
A subset of mITT subjects who were scheduled to perform CPET
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 10 5 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
CPET Duration of Exercise Week 25 (min) 0.8  (0.88) 1.1  (1.43) 0.9  (1.05)
CPET Duration of Exercise Week 52 (min) 0.6  (1.34) 0.7  (1.46) 0.6  (1.33)
7.Secondary Outcome
Title Cardiopulmonary Exercise Testing (CPET) - Peak Workload
Hide Description

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

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

Time Frame Baseline, Week 25 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
A subset of mITT subjects who were scheduled to perform CPET
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 10 5 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
Week 25 23.6  (20.87) 38.4  (44.66) 28.5  (30.04)
Week 52 24.7  (26.17) 13.0  (22.69) 20.8  (24.90)
8.Secondary Outcome
Title Cardiopulmonary Exercise Testing (CPET) - O2 Pulse
Hide Description

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

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

Time Frame Baseline, Week 25 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
A subset of mITT subjects who were scheduled to perform CPET
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 10 5 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
Week 25 6.8  (14.17) 13.5  (17.65) 9.0  (15.13)
Week 52 7.6  (27.89) 2.0  (31.74) 5.6  (28.22)
9.Secondary Outcome
Title Cardiopulmonary Exercise Testing (CPET) - Aerobic Efficiency
Hide Description

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

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

Note that decline in Aerobic Efficiency translate into an improvement

Time Frame Baseline, Week 25 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
A subset of mITT subjects who were scheduled to perform CPET
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 10 5 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
Week 25 -8.9  (15.41) -6.0  (8.29) -7.9  (13.02)
Week 52 -7.5  (28.39) -9.8  (33.14) -8.2  (28.52)
10.Secondary Outcome
Title Muscle Strength Testing (MST) - Knee Extension Test
Hide Description Change from baseline to Week 25 and 52 as measured by the peak force in MST knee extension test (newton meters).
Time Frame Baseline, Week 25 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Modified Intent-to-treat Analysis Set
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 15 10 25
Mean (Standard Deviation)
Unit of Measure: Percent Change
Week 25 -0.6  (9.76) 5.2  (7.63) 1.7  (9.26)
Week 52 3.3  (6.88) 6.7  (8.87) 4.7  (7.74)
11.Secondary Outcome
Title Muscle Strength Testing (MST) - Knee Flexion Test
Hide Description Percent change from baseline to Week 25 and 52 as measured by the peak force in MST knee flexion test (newton meters).
Time Frame Baseline, Week 25 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Modified Intent-to-treat Analysis Set
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 15 10 25
Mean (Standard Deviation)
Unit of Measure: Percent Change
Week 25 -4.2  (24.83) 9.4  (44.87) 1.5  (34.39)
Week 52 -10.8  (22.72) 8.2  (41.57) -2.7  (32.69)
12.Secondary Outcome
Title Muscle Strength Testing (MST) - Elbow Flexion Test
Hide Description Percent change from baseline to Week 25 and 52 as measured by the peak force in MST elbow flexion test (newton meters).
Time Frame Baseline, Week 25 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Modified Intent-to-treat Analysis Set
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 15 10 25
Mean (Standard Deviation)
Unit of Measure: Percent Change
Week 25 12.0  (55.42) 17.7  (41.82) 14.1  (49.67)
Week 52 49.7  (86.83) 50.7  (59.81) 50.1  (75.60)
13.Secondary Outcome
Title Adolescent Pediatric Pain Tool (APPT) - Pain Intensity
Hide Description

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

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

Time Frame Baseline, Week 12, 24, and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Modified Intent-to-treat
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week All Subjects
Hide Arm/Group Description:
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
Both groups combined
Overall Number of Participants Analyzed 15 10 25
Mean (Standard Deviation)
Unit of Measure: units on a scale
APPT Word Graphic Rating Scale Week 12 -2.3  (3.37) -1.2  (2.23) -1.8  (2.93)
APPT Word Graphic Rating Scale Week 24 -2.2  (3.68) -1.2  (2.87) -1.8  (3.33)
APPT Word Graphic Rating Scale Week 52 -0.8  (3.41) -0.8  (3.04) -0.8  (3.17)
Time Frame Study Period, up to 192 weeks or ETV
Adverse Event Reporting Description All Adverse Events
 
Arm/Group Title BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week
Hide Arm/Group Description BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase. BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
All-Cause Mortality
BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
BMN 110 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/15 (20.00%)      2/10 (20.00%)    
Gastrointestinal disorders     
Inflammatory bowel disease  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Umbilical hernia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Knee deformity  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Kyphoscoliosis  1  1/15 (6.67%)  2 0/10 (0.00%)  0
Renal and urinary disorders     
Enuresis  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Surgical and medical procedures     
Medical device removal  1  0/15 (0.00%)  0 1/10 (10.00%)  1
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 Weekly at 2.0 mg/kg/Week BMN 110 Weekly at 4.0 mg/kg/Week
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   15/15 (100.00%)      10/10 (100.00%)    
Blood and lymphatic system disorders     
Lymph node pain  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Cardiac disorders     
Bradycardia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Sinus bradycardia  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Tachycardia  1  0/15 (0.00%)  0 1/10 (10.00%)  2
Congenital, familial and genetic disorders     
Pectus carinatum  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Ear and labyrinth disorders     
Ear discomfort  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Ear pain  1  2/15 (13.33%)  3 2/10 (20.00%)  2
Ear pruritus  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Motion sickness  1  2/15 (13.33%)  3 0/10 (0.00%)  0
Otorrhoea  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Tinnitus  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Eye disorders     
Eye pruritus  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Photophobia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Vision blurred  1  1/15 (6.67%)  2 0/10 (0.00%)  0
Vitreous floaters  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Gastrointestinal disorders     
Abdominal discomfort  1  1/15 (6.67%)  3 0/10 (0.00%)  0
Abdominal pain  1  6/15 (40.00%)  18 3/10 (30.00%)  23
Abdominal pain lower  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Abdominal pain upper  1  4/15 (26.67%)  8 4/10 (40.00%)  11
Aerophagia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Anal pruritus  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Aphthous stomatitis  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Constipation  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Diarrhoea  1  6/15 (40.00%)  21 4/10 (40.00%)  10
Dyspepsia  1  1/15 (6.67%)  1 1/10 (10.00%)  6
Gastrooesophageal reflux disease  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Haematochezia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Lip oedema  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Nausea  1  10/15 (66.67%)  33 5/10 (50.00%)  6
Oral pain  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Retching  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Swollen tongue  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Toothache  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Vomiting  1  11/15 (73.33%)  34 5/10 (50.00%)  10
General disorders     
Catheter site pain  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Chest pain  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Chills  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Cyst  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Device malfunction  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Extravasation  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Face oedema  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Fatigue  1  8/15 (53.33%)  12 2/10 (20.00%)  5
Influenza like illness  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Infusion site bruising  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Infusion site erythema  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Infusion site extravasation  1  3/15 (20.00%)  6 2/10 (20.00%)  4
Infusion site swelling  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Injection site pain  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Injection site pruritus  1  0/15 (0.00%)  0 1/10 (10.00%)  2
Injection site reaction  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Irritability  1  2/15 (13.33%)  3 0/10 (0.00%)  0
Local swelling  1  2/15 (13.33%)  3 1/10 (10.00%)  1
Malaise  1  1/15 (6.67%)  1 1/10 (10.00%)  1
Medical device pain  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Oedema peripheral  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Pain  1  2/15 (13.33%)  2 1/10 (10.00%)  1
Pyrexia  1  9/15 (60.00%)  24 3/10 (30.00%)  9
Immune system disorders     
Seasonal allergy  1  0/15 (0.00%)  0 2/10 (20.00%)  2
Infections and infestations     
Ear infection  1  2/15 (13.33%)  3 0/10 (0.00%)  0
Enterobiasis  1  1/15 (6.67%)  3 0/10 (0.00%)  0
Fungal infection  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Gastroenteritis  1  2/15 (13.33%)  3 3/10 (30.00%)  5
Gastroenteritis viral  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Gastrointestinal infection  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Influenza  1  4/15 (26.67%)  4 1/10 (10.00%)  1
Nasopharyngitis  1  6/15 (40.00%)  10 7/10 (70.00%)  16
Oral herpes  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Otitis media  1  2/15 (13.33%)  2 1/10 (10.00%)  1
Pharyngitis  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Pharyngitis streptococcal  1  2/15 (13.33%)  5 0/10 (0.00%)  0
Pneumonia  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Rhinitis  1  0/15 (0.00%)  0 2/10 (20.00%)  2
Sinusitis  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Upper respiratory tract infection  1  5/15 (33.33%)  13 5/10 (50.00%)  7
Urinary tract infection  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Injury, poisoning and procedural complications     
Contusion  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Excoriation  1  1/15 (6.67%)  1 1/10 (10.00%)  1
Fall  1  2/15 (13.33%)  2 2/10 (20.00%)  3
Hand fracture  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Head injury  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Inadequate analgesia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Infusion related reaction  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Injection related reaction  1  1/15 (6.67%)  2 0/10 (0.00%)  0
Joint injury  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Laceration  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Ligament sprain  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Limb crushing injury  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Limb injury  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Muscle strain  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Procedural pain  1  5/15 (33.33%)  8 2/10 (20.00%)  2
Wound complication  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Investigations     
Blood glucose decreased  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Haemoglobin increased  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Urine leukocyte esterase positive  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Urine output decreased  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Weight decreased  1  1/15 (6.67%)  1 0/10 (0.00%)  0
White blood cells urine positive  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Metabolism and nutrition disorders     
Decreased appetite  1  1/15 (6.67%)  1 1/10 (10.00%)  1
Hyperglycaemia  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Hypophosphataemia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Increased appetite  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Arthralgia  1  6/15 (40.00%)  28 5/10 (50.00%)  11
Back pain  1  6/15 (40.00%)  13 1/10 (10.00%)  1
Elbow deformity  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Flank pain  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Joint swelling  1  2/15 (13.33%)  2 1/10 (10.00%)  1
Knee deformity  1  1/15 (6.67%)  1 1/10 (10.00%)  1
Kyphoscoliosis  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Muscle spasms  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Musculoskeletal chest pain  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Musculoskeletal pain  1  3/15 (20.00%)  4 0/10 (0.00%)  0
Musculoskeletal stiffness  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Myalgia  1  2/15 (13.33%)  5 0/10 (0.00%)  0
Osteoarthritis  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Osteochondrosis  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Osteopenia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Pain in extremity  1  8/15 (53.33%)  22 4/10 (40.00%)  5
Nervous system disorders     
Coordination abnormal  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Dizziness  1  6/15 (40.00%)  8 2/10 (20.00%)  3
Dysgeusia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Headache  1  13/15 (86.67%)  89 7/10 (70.00%)  44
Lethargy  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Neuralgia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Poor quality sleep  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Somnolence  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Syncope  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Psychiatric disorders     
Anxiety  1  2/15 (13.33%)  2 1/10 (10.00%)  3
Depression  1  1/15 (6.67%)  2 0/10 (0.00%)  0
Insomnia  1  3/15 (20.00%)  3 0/10 (0.00%)  0
Phobia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Renal and urinary disorders     
Dysuria  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Enuresis  1  1/15 (6.67%)  2 0/10 (0.00%)  0
Pollakiuria  1  1/15 (6.67%)  2 0/10 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Asthma  1  1/15 (6.67%)  2 1/10 (10.00%)  1
Cough  1  6/15 (40.00%)  9 4/10 (40.00%)  8
Dysphonia  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Dyspnoea  1  4/15 (26.67%)  4 0/10 (0.00%)  0
Epistaxis  1  3/15 (20.00%)  5 1/10 (10.00%)  1
Hypoventilation  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Nasal congestion  1  7/15 (46.67%)  9 2/10 (20.00%)  2
Oropharyngeal pain  1  7/15 (46.67%)  11 4/10 (40.00%)  6
Productive cough  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Respiratory distress  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Rhinorrhoea  1  1/15 (6.67%)  2 1/10 (10.00%)  1
Throat irritation  1  1/15 (6.67%)  1 2/10 (20.00%)  2
Wheezing  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Skin and subcutaneous tissue disorders     
Acne  1  1/15 (6.67%)  1 2/10 (20.00%)  4
Decubitus ulcer  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Dermatitis allergic  1  1/15 (6.67%)  1 1/10 (10.00%)  1
Dermatitis contact  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Dry skin  1  1/15 (6.67%)  1 1/10 (10.00%)  2
Ecchymosis  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Eczema  1  2/15 (13.33%)  2 0/10 (0.00%)  0
Erythema  1  4/15 (26.67%)  9 2/10 (20.00%)  2
Nail disorder  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Pruritus  1  3/15 (20.00%)  6 1/10 (10.00%)  1
Rash  1  6/15 (40.00%)  7 1/10 (10.00%)  3
Rash erythematous  1  0/15 (0.00%)  0 1/10 (10.00%)  1
Rash generalised  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Rash pruritic  1  1/15 (6.67%)  1 2/10 (20.00%)  3
Swelling face  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Urticaria  1  3/15 (20.00%)  11 0/10 (0.00%)  0
Surgical and medical procedures     
Catheterisation venous  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Central venous catheterisation  1  1/15 (6.67%)  1 1/10 (10.00%)  1
Medical device removal  1  0/15 (0.00%)  0 2/10 (20.00%)  2
Tooth extraction  1  1/15 (6.67%)  2 0/10 (0.00%)  0
Vascular disorders     
Flushing  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Hot flush  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Hypotension  1  1/15 (6.67%)  2 0/10 (0.00%)  0
Pallor  1  1/15 (6.67%)  1 0/10 (0.00%)  0
Peripheral coldness  1  1/15 (6.67%)  1 0/10 (0.00%)  0
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 up to 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
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: NCT01609062     History of Changes
Other Study ID Numbers: MOR-008
First Submitted: May 24, 2012
First Posted: May 31, 2012
Results First Submitted: November 12, 2015
Results First Posted: February 1, 2016
Last Update Posted: February 1, 2016