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