Fabry Disease
Conditions
Keywords
α-GAL, α-Galactosidase-A, r-hαGAL
Brief summary
The purpose of this study was to determine whether 2 alternative dosing regimens of Fabrazyme (Agalsidase beta) (1.0 mg/kg every 4 weeks or 0.5 mg/kg every 2 weeks) were effective in treatment-naïve pediatric participants without severe symptoms. Participants were to be treated for 5 years.
Interventions
BIOLOGICALAgalsidase beta
Powder for concentrate for solution for infusion 1.0 mg/kg/4 weeks
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
5 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* The participant and/or participant's parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed. * The participant must had a confirmed diagnosis of Fabry disease as documented by leukocyte α-Galactosidase A (αGAL) activity of \<4 nmol/hr/mg leukocyte (preferred assay; resulted from a central laboratory). If the leukocyte αGAL activity assay was difficult to obtain, the participant might be enrolled based on documented plasma αGAL \<1.5 nmol/hr/mL, with the agreement of the Medical Monitor (resulted from a central laboratory). * The participant must had evidence of globotriaosylceramide (GL-3) accumulation as documented by plasma GL-3 (\>7.0 µg/mL) or urinary GL-3 (\>0.3 mg GL-3/mmol creatinine) levels (results from a central laboratory). * The participant must be male ≥5 and ≤18 years of age.
Exclusion criteria
* Participant had albuminuria (first morning void urinary albumin/creatinine ratio \>30 mg/g on at least 2 out of 3 consecutive samples, each at least 1 week apart). * Participant had a Glomerular Filtration Rate (GFR) by iohexol \<90 L/min/1.73m\^2. In case of properly documented low protein intake, values as low as 80 mL/min/1.73 m\^2 might be acceptable, after consultation with the Medical Monitor. * Participant had documented evidence of stroke or transient ischemic attack (TIA), or if a brain magnetic resonance imaging (MRI) had been performed, bright lesions \>2 mm on T2- or fluid attenuated inversion recovery (FLAIR)- weighted images within the white matter or the basal ganglia. * Participant had severe and recurrent acroparesthesia, judged by the physician as frequent (more than once a week) pain episodes for at least 3 months that influenced daily activities, irrespective of medication. * Participant had an end-diastolic left ventricular posterior wall thickness (LVPWTd) and/or an end-diastolic interventricular septum thickness (IVSTd)≥2 standard deviations (SD) compared to normal (based on body surface area \[BSA\] normal ranges from Kampmann, et al 2000) as read at the study site. * Participant had received prior treatment specific to Fabry Disease. * Participant had participated in a study employing an investigational drug within 30 days of the start of their participation in this study. * Participant had any medical condition or extenuating circumstance, which in the opinion of the Study Investigator, could interfere with study compliance. * Participant had any medical condition or extenuating circumstance, for example diabetes mellitus, which in the opinion of the Study Investigator, could interfere with the interpretation of study results. * Participant was on treatment with angiotensin converting enzyme inhibitors/angiotensin receptor blockers (ACEIs/ARBs). * Participant had any contra-indication mentioned in the labeling of Fabrazyme and/or iohexol (Omnipaque). * Participant or parent(s)/legal guardian(s) was unwilling to comply with the requirements of the protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Baseline, Week 52, Week 156 and Week 260 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change From Baseline in GL-3 Clearance From Plasma | Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260 | 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. |
| Percent Change From Baseline in GL-3 Clearance From Urine | Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260 | 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. |
Countries
Argentina, Brazil, Canada, Czechia, Netherlands, Norway, Poland, United Kingdom, United States
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.
Participants by arm
| Arm | Count |
|---|---|
| Fabrazyme 0.5 mg/kg 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. | 16 |
| Fabrazyme 1.0 mg/kg 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. | 15 |
| Total | 31 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 0 | 1 |
| Overall Study | Social/family issues and needle phobia | 1 | 0 |
Baseline characteristics
| Characteristic | Fabrazyme 0.5 mg/kg | Fabrazyme 1.0 mg/kg | Total |
|---|---|---|---|
| Age, Continuous | 11.2 years STANDARD_DEVIATION 4 | 11.9 years STANDARD_DEVIATION 4.5 | 11.5 years STANDARD_DEVIATION 4.2 |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 16 Participants | 15 Participants | 31 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 16 / 16 | 15 / 15 |
| serious Total, serious adverse events | 6 / 16 | 5 / 15 |
Outcome results
Primary
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium
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
Population: Analysis was performed on Full analysis set (FAS), which included all randomized participants who received at least 1 infusion of study treatment.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Zero (0) Skin GL-3 Score at Baseline | 18.8 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Zero (0) Skin GL-3 Score at Week 52 | 75 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Zero (0) Skin GL-3 Score at Week 156 | 56.3 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Zero (0) Skin GL-3 Score at Week 260 | 68.8 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Mild (1) Skin GL-3 Score at Baseline | 6.3 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Mild (1) Skin GL-3 Score at Week 52 | 6.3 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Mild (1) Skin GL-3 Score at Week 156 | 18.8 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Mild (1) Skin GL-3 Score at Week 260 | 12.5 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Moderate (2) Skin GL-3 Score at Baseline | 75 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Moderate (2) Skin GL-3 Score at Week 52 | 0 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Moderate (2) Skin GL-3 Score at Week 156 | 6.3 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Moderate (2) Skin GL-3 Score at Week 260 | 0 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Severe (3) Skin GL-3 Score at Baseline | 0 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Severe (3) Skin GL-3 Score at Week 52 | 0 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Severe (3) Skin GL-3 Score at Week 156 | 0 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Severe (3) Skin GL-3 Score at Week 260 | 0 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Missing Skin GL-3 Score at Baseline | 0 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Missing Skin GL-3 Score at Week 52 | 18.8 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Missing Skin GL-3 Score at Week 156 | 18.8 Percentage of participants |
| Fabrazyme 0.5 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Missing Skin GL-3 Score at Week 260 | 18.8 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Missing Skin GL-3 Score at Week 52 | 6.7 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Zero (0) Skin GL-3 Score at Baseline | 33.3 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Moderate (2) Skin GL-3 Score at Week 156 | 13.3 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Zero (0) Skin GL-3 Score at Week 52 | 80 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Severe (3) Skin GL-3 Score at Week 260 | 0 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Zero (0) Skin GL-3 Score at Week 156 | 80 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Moderate (2) Skin GL-3 Score at Week 260 | 6.7 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Zero (0) Skin GL-3 Score at Week 260 | 66.7 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Missing Skin GL-3 Score at Week 260 | 6.7 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Mild (1) Skin GL-3 Score at Baseline | 0 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Severe (3) Skin GL-3 Score at Baseline | 0 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Mild (1) Skin GL-3 Score at Week 52 | 13.3 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Missing Skin GL-3 Score at Baseline | 0 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Mild (1) Skin GL-3 Score at Week 156 | 0 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Severe (3) Skin GL-3 Score at Week 52 | 0 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Mild (1) Skin GL-3 Score at Week 260 | 20 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Missing Skin GL-3 Score at Week 156 | 6.7 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Moderate (2) Skin GL-3 Score at Baseline | 66.7 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Severe (3) Skin GL-3 Score at Week 156 | 0 Percentage of participants |
| Fabrazyme 1.0 mg/kg | Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium | Moderate (2) Skin GL-3 Score at Week 52 | 0 Percentage of participants |
Secondary
Percent Change From Baseline in GL-3 Clearance From Plasma
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
Population: Analysis was performed on FAS.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 12 (n=14, 11) | -52.37 Percent change | Standard Deviation 10.12 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 28 (n=14, 14) | -49.06 Percent change | Standard Deviation 15.43 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 40 (n=13, 14) | -52.01 Percent change | Standard Deviation 11.29 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 52 (n=14, 14) | -52.29 Percent change | Standard Deviation 10.48 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 80 (n=13, 14) | -52.91 Percent change | Standard Deviation 13.93 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 104 (n=13, 14) | -51.08 Percent change | Standard Deviation 20.45 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 132 (n=11, 14) | -61.39 Percent change | Standard Deviation 10.71 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 156 (n=11, 14) | -48.72 Percent change | Standard Deviation 18.48 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 184 (n=12, 14) | -53.62 Percent change | Standard Deviation 19.38 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 208 (n=12, 14) | -48.83 Percent change | Standard Deviation 16.8 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 236 (n=12, 14) | -56.44 Percent change | Standard Deviation 12.08 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 260 (n=11, 14) | -59.95 Percent change | Standard Deviation 12.39 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 236 (n=12, 14) | -47.25 Percent change | Standard Deviation 13.04 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 12 (n=14, 11) | -52.74 Percent change | Standard Deviation 6.79 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 132 (n=11, 14) | -52.97 Percent change | Standard Deviation 17.1 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 28 (n=14, 14) | -47.55 Percent change | Standard Deviation 16.75 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 208 (n=12, 14) | -46.09 Percent change | Standard Deviation 16.84 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 40 (n=13, 14) | -50.82 Percent change | Standard Deviation 12.87 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 156 (n=11, 14) | -44.83 Percent change | Standard Deviation 14.14 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 52 (n=14, 14) | -45.87 Percent change | Standard Deviation 16.01 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 260 (n=11, 14) | -46.34 Percent change | Standard Deviation 14.01 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 80 (n=13, 14) | -48.93 Percent change | Standard Deviation 14.75 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 184 (n=12, 14) | -49.08 Percent change | Standard Deviation 17.93 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Plasma | Week 104 (n=13, 14) | -39.92 Percent change | Standard Deviation 18.69 |
Secondary
Percent Change From Baseline in GL-3 Clearance From Urine
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
Population: Analysis was performed on FAS.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 28 (n=15, 15) | -50.84 Percent change | Standard Deviation 100.61 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 132 (n=13, 14) | -48.79 Percent change | Standard Deviation 100.1 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 52 (n=15, 14) | -70.1 Percent change | Standard Deviation 41.4 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 156 (n=13, 14) | -65.57 Percent change | Standard Deviation 52.11 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 12 (n=15, 14) | -50.77 Percent change | Standard Deviation 64.59 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 184 (n=13, 14) | -76.54 Percent change | Standard Deviation 38.93 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 80 (n=14, 14) | -35.84 Percent change | Standard Deviation 102.31 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 208 (n=13, 14) | -60.94 Percent change | Standard Deviation 67.99 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 40 (n=15, 14) | -44.22 Percent change | Standard Deviation 105.03 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 236 (n=13, 14) | -69.08 Percent change | Standard Deviation 51.72 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 260 (n=13, 14) | -57.59 Percent change | Standard Deviation 93.74 |
| Fabrazyme 0.5 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 104 (n=14, 14) | -21.92 Percent change | Standard Deviation 138.78 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 260 (n=13, 14) | -28.27 Percent change | Standard Deviation 56.79 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 12 (n=15, 14) | -63.39 Percent change | Standard Deviation 38.81 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 28 (n=15, 15) | -52.55 Percent change | Standard Deviation 58.59 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 40 (n=15, 14) | -63.87 Percent change | Standard Deviation 24.41 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 52 (n=15, 14) | -20.72 Percent change | Standard Deviation 156.1 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 80 (n=14, 14) | 35.22 Percent change | Standard Deviation 238.61 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 104 (n=14, 14) | -56.39 Percent change | Standard Deviation 42.39 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 132 (n=13, 14) | -45.61 Percent change | Standard Deviation 48.84 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 156 (n=13, 14) | -28.92 Percent change | Standard Deviation 84.32 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 184 (n=13, 14) | -10.5 Percent change | Standard Deviation 146.86 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 208 (n=13, 14) | -50.93 Percent change | Standard Deviation 46.52 |
| Fabrazyme 1.0 mg/kg | Percent Change From Baseline in GL-3 Clearance From Urine | Week 236 (n=13, 14) | -40.09 Percent change | Standard Deviation 77.04 |