A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
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Incidence of Treatment Emergent Adverse Events (TEAEs)
Timeframe: 12 Months
Incidence of Infusion Related Reactions (IRRs)
Timeframe: 12 Months
Incidence of Injection site reactions (ISRs)
Timeframe: 12 Months
Change in Tanner stage
Timeframe: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate
Timeframe: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: PR Interval
Timeframe: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QRS Duration
Timeframe: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QT Interval
Timeframe: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QTc Interval
Timeframe: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: ST Segment
Timeframe: Baseline and 12 Months
Incidence of treatment-emergent Anti-Drug Antibodies (ADAs)
Timeframe: Baseline and 12 Months
Incidence of premedication use at each visit and change of infusion premedications from baseline
Timeframe: Baseline and 12 Months
Pharmacokinetics: Time to maximum plasma concentration (tmax)
Timeframe: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetic : Area under the plasma concentration-time curve from time 0 to time t (AUC0 t)
Timeframe: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Area under the curve from time 0 to 2 weeks (AUC0-2wk)
Timeframe: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Area under the curve from time 0 to infinity (AUC0-∞)
Timeframe: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Terminal half-life (t1/2)
Timeframe: Baseline, week 2, week 4, week 12, week 26 and week 52]
Pharmacokinetics: Area under the curve over a dosing interval (AUCÏ„)
Timeframe: Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Observed drug concentration at the end of the dosing interval (CÏ„)
Timeframe: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Clearance (Cl)
Timeframe: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Volume of distribution (Vz)
Timeframe: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Change in eGFR
Timeframe: Baseline and 12 Months
Change in annualized eGFR slope
Timeframe: Baseline and 12 Months
Change in urine albumin levels
Timeframe: Baseline and 12 Months
Change in urine protein levels
Timeframe: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Timeframe: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Timeframe: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Timeframe: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Timeframe: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Timeframe: Baseline and 12 Months
Incidence of any cardiac arrythmias as assessed by Holter ECG
Timeframe: Baseline and 12 Months
Change in plasma levels of cardiac biomarkers
Timeframe: Baseline and 12 Months
Change in plasma level of Gb3 concentration (nM)
Timeframe: Baseline and 12 Months
Change in plasma level of lyso-Gb3 (nM)
Timeframe: Baseline and 12 Months
Change in urine level of lyso-Gb3 (nM)
Timeframe: Baseline and 12 Months
Incidence of change from baseline in the number of different pain medications
Timeframe: Baseline and 12 Months
Incidence of Fabry Clinical Events
Timeframe: 12 Months
Change from baseline of Mainz Severity Score Index (MSSI) scores
Timeframe: Baseline and 12 Months
Change from baseline of PedsQL-GI (or GSRS for subjects who reaches 18 yrs of age) scores
Timeframe: Baseline and 12 Months
Change from baseline of FPHPQ scores
Timeframe: Baseline and 12 Months
Change from baseline of PedsQL-PPQ (or BPI-SF for subjects who reaches 18 yrs of age) scores
Timeframe: Baseline and 12 Months
Change from baseline of EQ-5D-Y (or EQ-5D-5L for subjects who reaches 18 yrs of age) scores
Timeframe: Baseline and 12 Months