A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
Participants with the provision of informed consent from their legal guardians
Boys and girls aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to <18 years (Cohort C).
Confirmed diagnosis of Fabry disease
Presence of at least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma.
Estimated glomerular filtration rate (eGFR) at screening < 80 mL/min/1.73 m2.
History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or any component of the study drug.
Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB) or a dose change in ongoing treatment in the four weeks before screening.
Urine protein to creatinine ratio (UPCR) > 0.5 g/g (0.5 mg/mg or 500 mg/g) if not treated with an ACE inhibitor or ARB.
Single group
PRX-102 1 mg/kg every two weeks
DrugDrug: PRX-102 1 mg/kg every two weeks
Incidence of Treatment Emergent Adverse Events (TEAEs)
Incidence of Infusion Related Reactions (IRRs)
Incidence of Injection site reactions (ISRs)
Change in Tanner stage
Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.
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Chiesi Farmaceutici S.p.A.
Lead sponsor
ICON plc
Collaborator
