protocolSection identificationModule nctId: NCT06328608, orgStudyIdInfo id: CLI-06657AA1-01, secondaryIdInfos id: 2022-503128-29, type: REGISTRY, domain: Clinical Trial Information System (CTIS) - EU, briefTitle: A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease, acronym: FLY, statusModule overallStatus: NOT_YET_RECRUITING, startDateStruct date: 2024-10, primaryCompletionDateStruct date: 2027-12, completionDateStruct date: 2028-03, studyFirstPostDateStruct date: 2024-03-25, lastUpdatePostDateStruct date: 2024-03-25, sponsorCollaboratorsModule leadSponsor name: Chiesi Farmaceutici S.p.A., class: INDUSTRY, collaborators name: ICON plc, descriptionModule briefSummary: A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease., conditionsModule conditions: Fabry Disease, designModule studyType: INTERVENTIONAL, phases: PHASE2, phases: PHASE3, designInfo allocation: NA, interventionModel: SINGLE_GROUP, primaryPurpose: TREATMENT, maskingInfo masking: NONE, enrollmentInfo count: 22, type: ESTIMATED, armsInterventionsModule interventions name: PRX-102 1 mg/kg every two weeks, outcomesModule primaryOutcomes measure: Incidence of Treatment Emergent Adverse Events (TEAEs), primaryOutcomes measure: Incidence of Infusion Related Reactions (IRRs), primaryOutcomes measure: Incidence of Injection site reactions (ISRs), primaryOutcomes measure: Change in Tanner stage, primaryOutcomes measure: Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate, primaryOutcomes measure: Change from baseline of 12-lead ECG quantitative parameters: PR Interval, primaryOutcomes measure: Change from baseline of 12-lead ECG quantitative parameters: QRS Duration, primaryOutcomes measure: Change from baseline of 12-lead ECG quantitative parameters: QT Interval, primaryOutcomes measure: Change from baseline of 12-lead ECG quantitative parameters: QTc Interval, primaryOutcomes measure: Change from baseline of 12-lead ECG quantitative parameters: ST Segment, primaryOutcomes measure: Incidence of treatment-emergent Anti-Drug Antibodies (ADAs), primaryOutcomes measure: Incidence of premedication use at each visit and change of infusion premedications from baseline, primaryOutcomes measure: Pharmacokinetics: Time to maximum plasma concentration (tmax), primaryOutcomes measure: Pharmacokinetic : Area under the plasma concentration-time curve from time 0 to time t (AUC0 t), primaryOutcomes measure: Pharmacokinetics: Area under the curve from time 0 to 2 weeks (AUC0-2wk), primaryOutcomes measure: Pharmacokinetics: Area under the curve from time 0 to infinity (AUC0-∞), primaryOutcomes measure: Pharmacokinetics: Terminal half-life (t1/2), primaryOutcomes measure: Pharmacokinetics: Area under the curve over a dosing interval (AUCτ), primaryOutcomes measure: Pharmacokinetics: Observed drug concentration at the end of the dosing interval (Cτ), primaryOutcomes measure: Pharmacokinetics: Clearance (Cl), primaryOutcomes measure: Pharmacokinetics: Volume of distribution (Vz), primaryOutcomes measure: Change in eGFR, primaryOutcomes measure: Change in annualized eGFR slope, primaryOutcomes measure: Change in urine albumin levels, primaryOutcomes measure: Change in urine protein levels, primaryOutcomes measure: Change from baseline in LVMi as assessed by echocardiogram, primaryOutcomes measure: Change from baseline in LVMi as assessed by echocardiogram, primaryOutcomes measure: Change from baseline in LVMi as assessed by echocardiogram, primaryOutcomes measure: Change from baseline in LVMi as assessed by echocardiogram, primaryOutcomes measure: Change from baseline in LVMi as assessed by echocardiogram, primaryOutcomes measure: Incidence of any cardiac arrythmias as assessed by Holter ECG, primaryOutcomes measure: Change in plasma levels of cardiac biomarkers, primaryOutcomes measure: Change in plasma level of Gb3 concentration (nM), primaryOutcomes measure: Change in plasma level of lyso-Gb3 (nM), primaryOutcomes measure: Change in urine level of lyso-Gb3 (nM), primaryOutcomes measure: Incidence of change from baseline in the number of different pain medications, primaryOutcomes measure: Incidence of Fabry Clinical Events, primaryOutcomes measure: Change from baseline of Mainz Severity Score Index (MSSI) scores, primaryOutcomes measure: Change from baseline of PedsQL-GI (or GSRS for subjects who reaches 18 yrs of age) scores, primaryOutcomes measure: Change from baseline of FPHPQ scores, primaryOutcomes measure: Change from baseline of PedsQL-PPQ (or BPI-SF for subjects who reaches 18 yrs of age) scores, primaryOutcomes measure: Change from baseline of EQ-5D-Y (or EQ-5D-5L for subjects who reaches 18 yrs of age) scores, eligibilityModule sex: ALL, minimumAge: 2 Years, maximumAge: 17 Years, stdAges: CHILD, contactsLocationsModule, hasResults: False