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A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (FIELD)

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ClinicalTrials.gov Identifier: NCT00701415
Recruitment Status : Completed
First Posted : June 19, 2008
Results First Posted : June 29, 2016
Last Update Posted : June 29, 2016
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fabry Disease
Intervention Biological: Agalsidase beta
Enrollment 31
Recruitment Details The study was conducted at 12 sites in 9 countries. A total of 44 participants were screened between 17 June 2008 and 12 April 2010.
Pre-assignment Details Of 44 screened participants, 31 were randomized in 1:1 ratio to fabrazyme 0.5 mg/kg and fabrazyme 1.0 mg/kg within each age stratum (5 to ≤11 years [children] and 12 to ≤18 years [adolescents]). 13 participants were screen failure due to failure to meet inclusion criteria or withdrawal of consent prior to all screening assessments being completed.
Arm/Group Title Fabrazyme 0.5 mg/kg Fabrazyme 1.0 mg/kg
Hide Arm/Group Description Fabrazyme 0.5 mg/kg was administered every 2 weeks (up to 131 infusions) up to 260 weeks, the total infusion time was not less than 45 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks. Fabrazyme 1.0 mg/kg was administered every 4 weeks (up to 66 infusions) up to 260 weeks, the total infusion time was not less than 90 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks.
Period Title: Overall Study
Started 16 15
Completed 15 14
Not Completed 1 1
Reason Not Completed
Adverse Event             0             1
Social/family issues and needle phobia             1             0
Arm/Group Title Fabrazyme 0.5 mg/kg Fabrazyme 1.0 mg/kg Total
Hide Arm/Group Description Fabrazyme 0.5 mg/kg was administered every 2 weeks (up to 131 infusion) up to 260 weeks, the total infusion time was not less than 45 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks. Fabrazyme 1.0 mg/kg was administered every 4 weeks (up to 66 infusion) up to 260 weeks, the total infusion time was not less than 90 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks. Total of all reporting groups
Overall Number of Baseline Participants 16 15 31
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants 15 participants 31 participants
11.2  (4) 11.9  (4.5) 11.5  (4.2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 15 participants 31 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
16
 100.0%
15
 100.0%
31
 100.0%
1.Primary Outcome
Title Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium
Hide Description Skin biopsies were taken at Baseline, Week 52, Week 156 and Week 260 or early withdrawal and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was scored for GL-3 accumulation on a severity score-scale of none, mild, moderate, severe (0-1-2-3). Scores are categorized as normal (score = 0) or abnormal (score = 1, 2 or 3). Data was summarized in terms of number of participants with none/trace, mild, moderate and severe biopsy scores.
Time Frame Baseline, Week 52, Week 156 and Week 260
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on Full analysis set (FAS), which included all randomized participants who received at least 1 infusion of study treatment.
Arm/Group Title Fabrazyme 0.5 mg/kg Fabrazyme 1.0 mg/kg
Hide Arm/Group Description:
Fabrazyme 0.5 mg/kg was administered every 2 weeks (up to 131 infusions) up to 260 weeks, the total infusion time was not less than 45 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks.
Fabrazyme 1.0 mg/kg was administered every 4 weeks (up to 66 infusions) up to 260 weeks, the total infusion time was not less than 90 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks.
Overall Number of Participants Analyzed 16 15
Measure Type: Number
Unit of Measure: Percentage of participants
Zero (0) Skin GL-3 Score at Baseline 18.8 33.3
Zero (0) Skin GL-3 Score at Week 52 75 80
Zero (0) Skin GL-3 Score at Week 156 56.3 80
Zero (0) Skin GL-3 Score at Week 260 68.8 66.7
Mild (1) Skin GL-3 Score at Baseline 6.3 0
Mild (1) Skin GL-3 Score at Week 52 6.3 13.3
Mild (1) Skin GL-3 Score at Week 156 18.8 0
Mild (1) Skin GL-3 Score at Week 260 12.5 20
Moderate (2) Skin GL-3 Score at Baseline 75 66.7
Moderate (2) Skin GL-3 Score at Week 52 0 0
Moderate (2) Skin GL-3 Score at Week 156 6.3 13.3
Moderate (2) Skin GL-3 Score at Week 260 0 6.7
Severe (3) Skin GL-3 Score at Baseline 0 0
Severe (3) Skin GL-3 Score at Week 52 0 0
Severe (3) Skin GL-3 Score at Week 156 0 0
Severe (3) Skin GL-3 Score at Week 260 0 0
Missing Skin GL-3 Score at Baseline 0 0
Missing Skin GL-3 Score at Week 52 18.8 6.7
Missing Skin GL-3 Score at Week 156 18.8 6.7
Missing Skin GL-3 Score at Week 260 18.8 6.7
2.Secondary Outcome
Title Percent Change From Baseline in GL-3 Clearance From Plasma
Hide Description Plasma samples were assayed for GL-3 clearance using a validated tandem mass spectrometry with an upper limit of normal plasma GL-3 level of 7.0 μg/mL. Number of participants analyzed=participants with both baseline and post-baseline GL-3 plasma clearance assessment. Here 'n' signifies number of participants with available data for specified category.
Time Frame Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS.
Arm/Group Title Fabrazyme 0.5 mg/kg Fabrazyme 1.0 mg/kg
Hide Arm/Group Description:
Fabrazyme 0.5 mg/kg was administered every 2 weeks (up to 131 infusions) up to 260 weeks, the total infusion time was not less than 45 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks.
Fabrazyme 1.0 mg/kg was administered every 4 weeks (up to 66 infusions) up to 260 weeks, the total infusion time was not less than 90 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks.
Overall Number of Participants Analyzed 14 14
Mean (Standard Deviation)
Unit of Measure: Percent change
Week 12 (n=14, 11) -52.37  (10.12) -52.74  (6.79)
Week 28 (n=14, 14) -49.06  (15.43) -47.55  (16.75)
Week 40 (n=13, 14) -52.01  (11.29) -50.82  (12.87)
Week 52 (n=14, 14) -52.29  (10.48) -45.87  (16.01)
Week 80 (n=13, 14) -52.91  (13.93) -48.93  (14.75)
Week 104 (n=13, 14) -51.08  (20.45) -39.92  (18.69)
Week 132 (n=11, 14) -61.39  (10.71) -52.97  (17.1)
Week 156 (n=11, 14) -48.72  (18.48) -44.83  (14.14)
Week 184 (n=12, 14) -53.62  (19.38) -49.08  (17.93)
Week 208 (n=12, 14) -48.83  (16.80) -46.09  (16.84)
Week 236 (n=12, 14) -56.44  (12.08) -47.25  (13.04)
Week 260 (n=11, 14) -59.95  (12.39) -46.34  (14.01)
3.Secondary Outcome
Title Percent Change From Baseline in GL-3 Clearance From Urine
Hide Description Plasma samples were assayed for total urine GL-3 clearance using a validated tandem mass spectrometry with an upper limit of normal of <0.030 mg/mmoL of creatinine. Number of participants analyzed=participants with both baseline and post-baseline GL-3 urine clearance assessment. Here 'n' signifies number of participants with available data for specified category.
Time Frame Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS.
Arm/Group Title Fabrazyme 0.5 mg/kg Fabrazyme 1.0 mg/kg
Hide Arm/Group Description:
Fabrazyme 0.5 mg/kg was administered every 2 weeks (up to 131 infusions) up to 260 weeks, the total infusion time was not less than 45 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks.
Fabrazyme 1.0 mg/kg was administered every 4 weeks (up to 66 infusions) up to 260 weeks, the total infusion time was not less than 90 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks.
Overall Number of Participants Analyzed 15 15
Mean (Standard Deviation)
Unit of Measure: Percent change
Week 12 (n=15, 14) -50.77  (64.59) -63.39  (38.81)
Week 28 (n=15, 15) -50.84  (100.61) -52.55  (58.59)
Week 40 (n=15, 14) -44.22  (105.03) -63.87  (24.41)
Week 52 (n=15, 14) -70.1  (41.4) -20.72  (156.1)
Week 80 (n=14, 14) -35.84  (102.31) 35.22  (238.61)
Week 104 (n=14, 14) -21.92  (138.78) -56.39  (42.39)
Week 132 (n=13, 14) -48.79  (100.1) -45.61  (48.84)
Week 156 (n=13, 14) -65.57  (52.11) -28.92  (84.32)
Week 184 (n=13, 14) -76.54  (38.93) -10.5  (146.86)
Week 208 (n=13, 14) -60.94  (67.99) -50.93  (46.52)
Week 236 (n=13, 14) -69.08  (51.72) -40.09  (77.04)
Week 260 (n=13, 14) -57.59  (93.74) -28.27  (56.79)
Time Frame All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (Week 264) regardless of seriousness or relationship to investigational product.
Adverse Event Reporting Description Reported adverse events are treatment-emergent adverse events that is AEs that developed/worsened during the ‘on treatment period’ (the time from the first infusion of the study drug up to 28 days after the last infusion of study drug). Safety population included all randomized participants who received at least one infusion of Fabrazyme.
 
Arm/Group Title Fabrazyme 0.5 mg/kg Fabrazyme 1.0 mg/kg
Hide Arm/Group Description Fabrazyme 0.5 mg/kg was administered every 2 weeks (up to 131 infusion) up to 260 weeks, the total infusion time was not less than 45 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks. Fabrazyme 1.0 mg/kg was administered every 4 weeks (up to 66 infusion) up to 260 weeks, the total infusion time was not less than 90 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks.
All-Cause Mortality
Fabrazyme 0.5 mg/kg Fabrazyme 1.0 mg/kg
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Fabrazyme 0.5 mg/kg Fabrazyme 1.0 mg/kg
Affected / at Risk (%) Affected / at Risk (%)
Total   6/16 (37.50%)   5/15 (33.33%) 
Gastrointestinal disorders     
GASTROINTESTINAL HAEMORRHAGE  1  0/16 (0.00%)  1/15 (6.67%) 
General disorders     
MEDICAL DEVICE COMPLICATION  1  0/16 (0.00%)  1/15 (6.67%) 
PYREXIA  1  1/16 (6.25%)  1/15 (6.67%) 
CHILLS  1  1/16 (6.25%)  0/15 (0.00%) 
Infections and infestations     
CELLULITIS  1  1/16 (6.25%)  0/15 (0.00%) 
ERYSIPELAS  1  0/16 (0.00%)  1/15 (6.67%) 
OTITIS MEDIA  1  0/16 (0.00%)  1/15 (6.67%) 
PNEUMONIA  1  0/16 (0.00%)  1/15 (6.67%) 
SEPSIS  1  0/16 (0.00%)  1/15 (6.67%) 
TINEA PEDIS  1  0/16 (0.00%)  1/15 (6.67%) 
Injury, poisoning and procedural complications     
MULTIPLE INJURIES  1  1/16 (6.25%)  0/15 (0.00%) 
OVERDOSE  1  1/16 (6.25%)  0/15 (0.00%) 
Investigations     
BODY MASS INDEX DECREASED  1  1/16 (6.25%)  0/15 (0.00%) 
Nervous system disorders     
PARAESTHESIA  1  1/16 (6.25%)  1/15 (6.67%) 
TREMOR  1  0/16 (0.00%)  1/15 (6.67%) 
Psychiatric disorders     
SUICIDAL IDEATION  1  1/16 (6.25%)  0/15 (0.00%) 
Renal and urinary disorders     
MICROALBUMINURIA  1  1/16 (6.25%)  1/15 (6.67%) 
Respiratory, thoracic and mediastinal disorders     
COUGH  1  0/16 (0.00%)  1/15 (6.67%) 
Skin and subcutaneous tissue disorders     
DERMATITIS  1  0/16 (0.00%)  1/15 (6.67%) 
Surgical and medical procedures     
CENTRAL VENOUS CATHETERISATION  1  1/16 (6.25%)  0/15 (0.00%) 
ELECTIVE SURGERY  1  1/16 (6.25%)  0/15 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 18.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Fabrazyme 0.5 mg/kg Fabrazyme 1.0 mg/kg
Affected / at Risk (%) Affected / at Risk (%)
Total   16/16 (100.00%)   15/15 (100.00%) 
Blood and lymphatic system disorders     
ANAEMIA  1  1/16 (6.25%)  1/15 (6.67%) 
EOSINOPHILIA  1  0/16 (0.00%)  1/15 (6.67%) 
LEUKOPENIA  1  0/16 (0.00%)  2/15 (13.33%) 
LYMPHADENOPATHY  1  2/16 (12.50%)  0/15 (0.00%) 
MONOCYTOPENIA  1  0/16 (0.00%)  1/15 (6.67%) 
Cardiac disorders     
ARRHYTHMIA  1  0/16 (0.00%)  1/15 (6.67%) 
TACHYCARDIA  1  2/16 (12.50%)  1/15 (6.67%) 
Ear and labyrinth disorders     
CONDUCTIVE DEAFNESS  1  1/16 (6.25%)  2/15 (13.33%) 
DEAFNESS  1  1/16 (6.25%)  0/15 (0.00%) 
DEAFNESS BILATERAL  1  1/16 (6.25%)  0/15 (0.00%) 
DEAFNESS NEUROSENSORY  1  1/16 (6.25%)  0/15 (0.00%) 
DEAFNESS UNILATERAL  1  1/16 (6.25%)  2/15 (13.33%) 
MIDDLE EAR EFFUSION  1  0/16 (0.00%)  1/15 (6.67%) 
OTORRHOEA  1  0/16 (0.00%)  1/15 (6.67%) 
TINNITUS  1  3/16 (18.75%)  4/15 (26.67%) 
TYMPANIC MEMBRANE HYPERAEMIA  1  1/16 (6.25%)  0/15 (0.00%) 
Endocrine disorders     
HYPERPARATHYROIDISM SECONDARY  1  1/16 (6.25%)  1/15 (6.67%) 
Eye disorders     
CONJUNCTIVAL HYPERAEMIA  1  0/16 (0.00%)  2/15 (13.33%) 
CORNEAL DEPOSITS  1  1/16 (6.25%)  0/15 (0.00%) 
DRY EYE  1  1/16 (6.25%)  0/15 (0.00%) 
LACRIMATION INCREASED  1  0/16 (0.00%)  1/15 (6.67%) 
OCULAR VASCULAR DISORDER  1  0/16 (0.00%)  1/15 (6.67%) 
PHOTOPHOBIA  1  1/16 (6.25%)  0/15 (0.00%) 
VISION BLURRED  1  0/16 (0.00%)  1/15 (6.67%) 
VISUAL ACUITY REDUCED  1  1/16 (6.25%)  0/15 (0.00%) 
PERIORBITAL OEDEMA  1  1/16 (6.25%)  0/15 (0.00%) 
Gastrointestinal disorders     
ABDOMINAL PAIN  1  9/16 (56.25%)  6/15 (40.00%) 
ABDOMINAL PAIN LOWER  1  1/16 (6.25%)  0/15 (0.00%) 
ABDOMINAL PAIN UPPER  1  4/16 (25.00%)  4/15 (26.67%) 
CONSTIPATION  1  2/16 (12.50%)  0/15 (0.00%) 
DENTAL CARIES  1  1/16 (6.25%)  1/15 (6.67%) 
DIARRHOEA  1  10/16 (62.50%)  8/15 (53.33%) 
EPIGASTRIC DISCOMFORT  1  0/16 (0.00%)  1/15 (6.67%) 
FOOD POISONING  1  1/16 (6.25%)  0/15 (0.00%) 
FREQUENT BOWEL MOVEMENTS  1  0/16 (0.00%)  1/15 (6.67%) 
GASTROINTESTINAL DISORDER  1  2/16 (12.50%)  1/15 (6.67%) 
NAUSEA  1  9/16 (56.25%)  7/15 (46.67%) 
ORAL MUCOSAL ERYTHEMA  1  0/16 (0.00%)  1/15 (6.67%) 
SALIVARY HYPERSECRETION  1  0/16 (0.00%)  1/15 (6.67%) 
STOMATITIS  1  1/16 (6.25%)  0/15 (0.00%) 
TOOTHACHE  1  0/16 (0.00%)  1/15 (6.67%) 
VOMITING  1  9/16 (56.25%)  7/15 (46.67%) 
ABDOMINAL TENDERNESS  1  1/16 (6.25%)  0/15 (0.00%) 
IRRITABLE BOWEL SYNDROME  1  1/16 (6.25%)  0/15 (0.00%) 
General disorders     
ASTHENIA  1  3/16 (18.75%)  1/15 (6.67%) 
CATHETER SITE PAIN  1  0/16 (0.00%)  1/15 (6.67%) 
CHEST DISCOMFORT  1  0/16 (0.00%)  2/15 (13.33%) 
CHILLS  1  6/16 (37.50%)  2/15 (13.33%) 
FACE OEDEMA  1  2/16 (12.50%)  0/15 (0.00%) 
FATIGUE  1  5/16 (31.25%)  2/15 (13.33%) 
FEELING COLD  1  3/16 (18.75%)  1/15 (6.67%) 
FEELING HOT  1  0/16 (0.00%)  3/15 (20.00%) 
FEELING OF BODY TEMPERATURE CHANGE  1  0/16 (0.00%)  1/15 (6.67%) 
INFLUENZA LIKE ILLNESS  1  2/16 (12.50%)  0/15 (0.00%) 
INFUSION SITE PAIN  1  1/16 (6.25%)  0/15 (0.00%) 
INJECTION SITE HAEMORRHAGE  1  1/16 (6.25%)  0/15 (0.00%) 
MALAISE  1  2/16 (12.50%)  1/15 (6.67%) 
MEDICAL DEVICE COMPLICATION  1  1/16 (6.25%)  1/15 (6.67%) 
OEDEMA PERIPHERAL  1  3/16 (18.75%)  2/15 (13.33%) 
PAIN  1  4/16 (25.00%)  3/15 (20.00%) 
PYREXIA  1  12/16 (75.00%)  8/15 (53.33%) 
SOFT TISSUE INFLAMMATION  1  0/16 (0.00%)  1/15 (6.67%) 
GENERAL PHYSICAL HEALTH DETERIORATION  1  1/16 (6.25%)  0/15 (0.00%) 
INFUSION SITE EXTRAVASATION  1  1/16 (6.25%)  0/15 (0.00%) 
Immune system disorders     
ALLERGY TO ARTHROPOD BITE  1  0/16 (0.00%)  1/15 (6.67%) 
Infections and infestations     
ACUTE SINUSITIS  1  1/16 (6.25%)  0/15 (0.00%) 
BRONCHITIS  1  1/16 (6.25%)  1/15 (6.67%) 
CATHETER SITE INFECTION  1  0/16 (0.00%)  1/15 (6.67%) 
CHLAMYDIAL INFECTION  1  0/16 (0.00%)  1/15 (6.67%) 
CONJUNCTIVITIS  1  0/16 (0.00%)  1/15 (6.67%) 
COXSACKIE VIRAL INFECTION  1  1/16 (6.25%)  0/15 (0.00%) 
EAR INFECTION  1  5/16 (31.25%)  1/15 (6.67%) 
ENTEROBIASIS  1  0/16 (0.00%)  1/15 (6.67%) 
ERYSIPELAS  1  0/16 (0.00%)  1/15 (6.67%) 
GASTROENTERITIS  1  2/16 (12.50%)  2/15 (13.33%) 
GASTROENTERITIS VIRAL  1  3/16 (18.75%)  1/15 (6.67%) 
HERPES SIMPLEX  1  1/16 (6.25%)  1/15 (6.67%) 
HERPES VIRUS INFECTION  1  0/16 (0.00%)  1/15 (6.67%) 
IMPETIGO  1  2/16 (12.50%)  1/15 (6.67%) 
INFLUENZA  1  3/16 (18.75%)  3/15 (20.00%) 
LOWER RESPIRATORY TRACT INFECTION  1  0/16 (0.00%)  1/15 (6.67%) 
MOLLUSCUM CONTAGIOSUM  1  0/16 (0.00%)  1/15 (6.67%) 
NASOPHARYNGITIS  1  7/16 (43.75%)  5/15 (33.33%) 
ORAL HERPES  1  2/16 (12.50%)  0/15 (0.00%) 
OTITIS MEDIA CHRONIC  1  0/16 (0.00%)  1/15 (6.67%) 
PARASITIC GASTROENTERITIS  1  0/16 (0.00%)  1/15 (6.67%) 
PHARYNGITIS  1  2/16 (12.50%)  0/15 (0.00%) 
PHARYNGITIS STREPTOCOCCAL  1  2/16 (12.50%)  0/15 (0.00%) 
PNEUMONIA  1  0/16 (0.00%)  1/15 (6.67%) 
RESPIRATORY TRACT INFECTION  1  0/16 (0.00%)  2/15 (13.33%) 
RESPIRATORY TRACT INFECTION VIRAL  1  1/16 (6.25%)  0/15 (0.00%) 
RHINITIS  1  5/16 (31.25%)  3/15 (20.00%) 
SINUSITIS  1  3/16 (18.75%)  1/15 (6.67%) 
STAPHYLOCOCCAL INFECTION  1  1/16 (6.25%)  0/15 (0.00%) 
TINEA INFECTION  1  0/16 (0.00%)  1/15 (6.67%) 
TINEA VERSICOLOUR  1  1/16 (6.25%)  0/15 (0.00%) 
TONSILLITIS  1  1/16 (6.25%)  0/15 (0.00%) 
TOOTH INFECTION  1  0/16 (0.00%)  1/15 (6.67%) 
UPPER RESPIRATORY TRACT INFECTION  1  6/16 (37.50%)  5/15 (33.33%) 
VIRAL INFECTION  1  2/16 (12.50%)  1/15 (6.67%) 
VIRAL PHARYNGITIS  1  0/16 (0.00%)  1/15 (6.67%) 
VIRAL RHINITIS  1  1/16 (6.25%)  0/15 (0.00%) 
VIRAL UPPER RESPIRATORY TRACT INFECTION  1  1/16 (6.25%)  1/15 (6.67%) 
HORDEOLUM  1  1/16 (6.25%)  0/15 (0.00%) 
Injury, poisoning and procedural complications     
ANIMAL BITE  1  1/16 (6.25%)  1/15 (6.67%) 
ARTHROPOD BITE  1  0/16 (0.00%)  1/15 (6.67%) 
ARTHROPOD STING  1  0/16 (0.00%)  2/15 (13.33%) 
CONCUSSION  1  1/16 (6.25%)  0/15 (0.00%) 
CONTUSION  1  2/16 (12.50%)  1/15 (6.67%) 
EAR INJURY  1  0/16 (0.00%)  1/15 (6.67%) 
EXCORIATION  1  0/16 (0.00%)  1/15 (6.67%) 
FALL  1  1/16 (6.25%)  0/15 (0.00%) 
FROSTBITE  1  1/16 (6.25%)  0/15 (0.00%) 
GENITAL INJURY  1  1/16 (6.25%)  0/15 (0.00%) 
HAND FRACTURE  1  1/16 (6.25%)  0/15 (0.00%) 
HEAD INJURY  1  0/16 (0.00%)  1/15 (6.67%) 
HUMERUS FRACTURE  1  1/16 (6.25%)  0/15 (0.00%) 
JOINT INJURY  1  0/16 (0.00%)  1/15 (6.67%) 
LACERATION  1  0/16 (0.00%)  1/15 (6.67%) 
LIGAMENT RUPTURE  1  1/16 (6.25%)  0/15 (0.00%) 
LIGAMENT SPRAIN  1  1/16 (6.25%)  1/15 (6.67%) 
LIMB INJURY  1  1/16 (6.25%)  1/15 (6.67%) 
MUSCLE RUPTURE  1  1/16 (6.25%)  0/15 (0.00%) 
MUSCLE STRAIN  1  0/16 (0.00%)  1/15 (6.67%) 
OVERDOSE  1  1/16 (6.25%)  0/15 (0.00%) 
POST-TRAUMATIC PAIN  1  0/16 (0.00%)  1/15 (6.67%) 
PROCEDURAL PAIN  1  0/16 (0.00%)  2/15 (13.33%) 
ROAD TRAFFIC ACCIDENT  1  1/16 (6.25%)  1/15 (6.67%) 
SKIN ABRASION  1  1/16 (6.25%)  1/15 (6.67%) 
SOFT TISSUE INJURY  1  0/16 (0.00%)  1/15 (6.67%) 
TOOTH FRACTURE  1  0/16 (0.00%)  2/15 (13.33%) 
WOUND COMPLICATION  1  1/16 (6.25%)  0/15 (0.00%) 
WOUND DEHISCENCE  1  1/16 (6.25%)  0/15 (0.00%) 
TENDON RUPTURE  1  1/16 (6.25%)  0/15 (0.00%) 
Investigations     
BETA 2 MICROGLOBULIN INCREASED  1  0/16 (0.00%)  1/15 (6.67%) 
BLOOD PRESSURE INCREASED  1  0/16 (0.00%)  1/15 (6.67%) 
BODY TEMPERATURE INCREASED  1  0/16 (0.00%)  1/15 (6.67%) 
EOSINOPHIL COUNT INCREASED  1  1/16 (6.25%)  0/15 (0.00%) 
HEART RATE INCREASED  1  0/16 (0.00%)  1/15 (6.67%) 
NEUTROPHIL COUNT INCREASED  1  1/16 (6.25%)  1/15 (6.67%) 
URINE ALBUMIN/CREATININE RATIO ABNORMAL  1  1/16 (6.25%)  0/15 (0.00%) 
VITAMIN B12 DECREASED  1  0/16 (0.00%)  2/15 (13.33%) 
VITAMIN D DECREASED  1  1/16 (6.25%)  1/15 (6.67%) 
PROTEIN URINE PRESENT  1  1/16 (6.25%)  0/15 (0.00%) 
Metabolism and nutrition disorders     
FAILURE TO THRIVE  1  0/16 (0.00%)  1/15 (6.67%) 
HYPOPHOSPHATAEMIA  1  0/16 (0.00%)  1/15 (6.67%) 
IRON DEFICIENCY  1  1/16 (6.25%)  0/15 (0.00%) 
OBESITY  1  0/16 (0.00%)  1/15 (6.67%) 
UNDERWEIGHT  1  1/16 (6.25%)  1/15 (6.67%) 
VITAMIN D DEFICIENCY  1  0/16 (0.00%)  1/15 (6.67%) 
VITAMIN B12 DEFICIENCY  1  1/16 (6.25%)  0/15 (0.00%) 
Musculoskeletal and connective tissue disorders     
ARTHRALGIA  1  4/16 (25.00%)  1/15 (6.67%) 
BACK PAIN  1  2/16 (12.50%)  2/15 (13.33%) 
FOOT DEFORMITY  1  1/16 (6.25%)  0/15 (0.00%) 
GROIN PAIN  1  1/16 (6.25%)  0/15 (0.00%) 
HAEMARTHROSIS  1  0/16 (0.00%)  1/15 (6.67%) 
INTERVERTEBRAL DISC PROTRUSION  1  1/16 (6.25%)  0/15 (0.00%) 
JOINT RANGE OF MOTION DECREASED  1  0/16 (0.00%)  1/15 (6.67%) 
MUSCLE SPASMS  1  1/16 (6.25%)  0/15 (0.00%) 
MUSCULOSKELETAL PAIN  1  1/16 (6.25%)  0/15 (0.00%) 
MYALGIA  1  2/16 (12.50%)  0/15 (0.00%) 
NECK PAIN  1  3/16 (18.75%)  0/15 (0.00%) 
PAIN IN EXTREMITY  1  8/16 (50.00%)  5/15 (33.33%) 
TENDONITIS  1  0/16 (0.00%)  1/15 (6.67%) 
JOINT SWELLING  1  1/16 (6.25%)  0/15 (0.00%) 
MUSCULAR WEAKNESS  1  1/16 (6.25%)  0/15 (0.00%) 
MUSCULOSKELETAL CHEST PAIN  1  1/16 (6.25%)  1/15 (6.67%) 
Nervous system disorders     
BURNING SENSATION  1  1/16 (6.25%)  1/15 (6.67%) 
DISTURBANCE IN ATTENTION  1  1/16 (6.25%)  0/15 (0.00%) 
DIZZINESS  1  4/16 (25.00%)  4/15 (26.67%) 
DYSGEUSIA  1  1/16 (6.25%)  0/15 (0.00%) 
HEADACHE  1  8/16 (50.00%)  4/15 (26.67%) 
MIGRAINE  1  1/16 (6.25%)  1/15 (6.67%) 
PARAESTHESIA  1  6/16 (37.50%)  3/15 (20.00%) 
PRESYNCOPE  1  2/16 (12.50%)  1/15 (6.67%) 
SLOW RESPONSE TO STIMULI  1  1/16 (6.25%)  0/15 (0.00%) 
SPEECH DISORDER  1  0/16 (0.00%)  1/15 (6.67%) 
TREMOR  1  1/16 (6.25%)  0/15 (0.00%) 
HYPOAESTHESIA  1  1/16 (6.25%)  0/15 (0.00%) 
Psychiatric disorders     
ABNORMAL BEHAVIOUR  1  1/16 (6.25%)  0/15 (0.00%) 
ANXIETY  1  1/16 (6.25%)  1/15 (6.67%) 
CONFUSIONAL STATE  1  1/16 (6.25%)  0/15 (0.00%) 
DEPRESSION  1  0/16 (0.00%)  1/15 (6.67%) 
DEPRESSIVE SYMPTOM  1  1/16 (6.25%)  0/15 (0.00%) 
DISORIENTATION  1  1/16 (6.25%)  0/15 (0.00%) 
DYSPHORIA  1  1/16 (6.25%)  0/15 (0.00%) 
HALLUCINATION  1  1/16 (6.25%)  0/15 (0.00%) 
INSOMNIA  1  2/16 (12.50%)  0/15 (0.00%) 
LISTLESS  1  1/16 (6.25%)  0/15 (0.00%) 
PANIC ATTACK  1  0/16 (0.00%)  1/15 (6.67%) 
SLEEP DISORDER  1  2/16 (12.50%)  0/15 (0.00%) 
Renal and urinary disorders     
HAEMATURIA  1  2/16 (12.50%)  1/15 (6.67%) 
HYDRONEPHROSIS  1  1/16 (6.25%)  0/15 (0.00%) 
HYDROURETER  1  1/16 (6.25%)  0/15 (0.00%) 
MICROALBUMINURIA  1  0/16 (0.00%)  1/15 (6.67%) 
Reproductive system and breast disorders     
PENILE DISCHARGE  1  0/16 (0.00%)  1/15 (6.67%) 
PENILE PAIN  1  0/16 (0.00%)  1/15 (6.67%) 
SCROTAL ANGIOKERATOMA  1  2/16 (12.50%)  1/15 (6.67%) 
Respiratory, thoracic and mediastinal disorders     
ALLERGIC COUGH  1  1/16 (6.25%)  0/15 (0.00%) 
ALLERGIC RESPIRATORY SYMPTOM  1  0/16 (0.00%)  1/15 (6.67%) 
BRONCHIAL OBSTRUCTION  1  1/16 (6.25%)  1/15 (6.67%) 
BRONCHOSPASM  1  1/16 (6.25%)  1/15 (6.67%) 
COUGH  1  8/16 (50.00%)  5/15 (33.33%) 
DRY THROAT  1  0/16 (0.00%)  1/15 (6.67%) 
DYSPNOEA  1  3/16 (18.75%)  2/15 (13.33%) 
EPISTAXIS  1  3/16 (18.75%)  1/15 (6.67%) 
NASAL CONGESTION  1  5/16 (31.25%)  1/15 (6.67%) 
OROPHARYNGEAL PAIN  1  3/16 (18.75%)  6/15 (40.00%) 
PHARYNGEAL ERYTHEMA  1  1/16 (6.25%)  0/15 (0.00%) 
RESPIRATORY FAILURE  1  1/16 (6.25%)  0/15 (0.00%) 
RESPIRATORY TRACT CONGESTION  1  0/16 (0.00%)  1/15 (6.67%) 
RHINITIS ALLERGIC  1  1/16 (6.25%)  0/15 (0.00%) 
RHINORRHOEA  1  1/16 (6.25%)  1/15 (6.67%) 
SINUS CONGESTION  1  1/16 (6.25%)  1/15 (6.67%) 
SNEEZING  1  1/16 (6.25%)  0/15 (0.00%) 
TACHYPNOEA  1  0/16 (0.00%)  1/15 (6.67%) 
THROAT IRRITATION  1  0/16 (0.00%)  1/15 (6.67%) 
TONSILLAR HYPERTROPHY  1  1/16 (6.25%)  0/15 (0.00%) 
UPPER RESPIRATORY TRACT CONGESTION  1  0/16 (0.00%)  1/15 (6.67%) 
WHEEZING  1  1/16 (6.25%)  0/15 (0.00%) 
CATARRH  1  1/16 (6.25%)  0/15 (0.00%) 
Skin and subcutaneous tissue disorders     
ACNE  1  1/16 (6.25%)  0/15 (0.00%) 
ANGIOKERATOMA  1  9/16 (56.25%)  5/15 (33.33%) 
BLISTER  1  0/16 (0.00%)  1/15 (6.67%) 
DERMATITIS  1  0/16 (0.00%)  1/15 (6.67%) 
DERMATITIS CONTACT  1  2/16 (12.50%)  2/15 (13.33%) 
DRY SKIN  1  1/16 (6.25%)  0/15 (0.00%) 
ECZEMA  1  1/16 (6.25%)  1/15 (6.67%) 
ERYTHEMA  1  4/16 (25.00%)  1/15 (6.67%) 
HYPERHIDROSIS  1  0/16 (0.00%)  1/15 (6.67%) 
MILIARIA  1  0/16 (0.00%)  1/15 (6.67%) 
NIGHT SWEATS  1  0/16 (0.00%)  1/15 (6.67%) 
PETECHIAE  1  0/16 (0.00%)  1/15 (6.67%) 
RASH  1  4/16 (25.00%)  1/15 (6.67%) 
RASH ERYTHEMATOUS  1  1/16 (6.25%)  0/15 (0.00%) 
RASH MACULAR  1  1/16 (6.25%)  1/15 (6.67%) 
SWELLING FACE  1  2/16 (12.50%)  1/15 (6.67%) 
TELANGIECTASIA  1  0/16 (0.00%)  1/15 (6.67%) 
UMBILICAL ERYTHEMA  1  0/16 (0.00%)  1/15 (6.67%) 
PRURITUS  1  1/16 (6.25%)  2/15 (13.33%) 
Surgical and medical procedures     
SINUS OPERATION  1  1/16 (6.25%)  0/15 (0.00%) 
Vascular disorders     
ANGIOPATHY  1  0/16 (0.00%)  1/15 (6.67%) 
FLUSHING  1  1/16 (6.25%)  0/15 (0.00%) 
HYPOTENSION  1  1/16 (6.25%)  1/15 (6.67%) 
PALLOR  1  2/16 (12.50%)  1/15 (6.67%) 
RAYNAUD'S PHENOMENON  1  0/16 (0.00%)  1/15 (6.67%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 18.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
Results Point of Contact
Name/Title: Trial Transparency Team
Organization: Sanofi
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00701415     History of Changes
Other Study ID Numbers: AGAL06207
2007-005668-28 ( EudraCT Number )
EFC12821 ( Other Identifier: Sanofi )
First Submitted: June 17, 2008
First Posted: June 19, 2008
Results First Submitted: May 20, 2016
Results First Posted: June 29, 2016
Last Update Posted: June 29, 2016