A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
Age range
2 Years – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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 (AUCτ)
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 (Cτ)
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