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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

Participant Flow

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
Arm/Group Description	Fabrazyme 0.5 mg/kg was administere...	Fabrazyme 1.0 mg/kg was administere...
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

Baseline Characteristics

Arm/Group Title		Fabrazyme 0.5 mg/kg	Fabrazyme 1.0 mg/kg	Total
Arm/Group Description		Fabrazyme 0.5 mg/kg was administere...	Fabrazyme 1.0 mg/kg was administere...	Total of all reporting groups
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
Baseline Analysis Population Description		[Not Specified]
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%

Outcome Measures

1.Primary Outcome


Title	Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium
Description	Skin biopsies were taken at Baseline, Week 52, Week 156 and Week 260 o...
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

Outcome Measure Data

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
Arm/Group Description:	Fabrazyme 0.5 mg/kg was administere...	Fabrazyme 1.0 mg/kg was administere...
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
Description	Plasma samples were assayed for GL-3 clearance using a validated tande...
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

Outcome Measure Data

Analysis Population Description

Analysis was performed on FAS.


Arm/Group Title	Fabrazyme 0.5 mg/kg	Fabrazyme 1.0 mg/kg
Arm/Group Description:	Fabrazyme 0.5 mg/kg was administere...	Fabrazyme 1.0 mg/kg was administere...
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
Description	Plasma samples were assayed for total urine GL-3 clearance using a val...
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

Outcome Measure Data

Analysis Population Description

Analysis was performed on FAS.


Arm/Group Title	Fabrazyme 0.5 mg/kg	Fabrazyme 1.0 mg/kg
Arm/Group Description:	Fabrazyme 0.5 mg/kg was administere...	Fabrazyme 1.0 mg/kg was administere...
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)

Adverse Events

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
Arm/Group Description	Fabrazyme 0.5 mg/kg was administere...	Fabrazyme 1.0 mg/kg was administere...
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	--/--	--/--
Serious Adverse Events 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
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

Limitations and Caveats

[Not Specified]

More Information

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

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Name/Title:	Trial Transparency Team
Organization:	Sanofi
EMail:	Contact-US@sanofi.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Wijburg FA, Bénichou B, Bichet DG, Clarke LA, Dostalova G, Fainboim A, Fellgiebel A, Forcelini C, An Haack K, Hopkin RJ, Mauer M, Najafian B, Scott CR, Shankar SP, Thurberg BL, Tøndel C, Tylki-Szymańska A, Ramaswami U. Characterization of early disease status in treatment-naive male paediatric patients with Fabry disease enrolled in a randomized clinical trial. PLoS One. 2015 May 8;10(5):e0124987. doi: 10.1371/journal.pone.0124987. eCollection 2015.

Rombach SM, Smid BE, Bouwman MG, Linthorst GE, Dijkgraaf MG, Hollak CE. Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain. Orphanet J Rare Dis. 2013 Mar 25;8:47. doi: 10.1186/1750-1172-8-47.

Layout table for additonal information

Responsible Party:	Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:	NCT00701415
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

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