Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease
Chiesi Farmaceutici S.p.A.
Summary
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
Description
This study aims to learn how safe pegunigalsidase alfa (PRX-102 for short) is and how it works at treating Fabry disease in children and adolescents. PRX-102 is an enzyme replacement therapy (ERT), meaning it acts like a natural enzyme. PRX-102 is given through a needle placed in a vein (intravenous infusion) every two weeks. The main questions this study aims to answer are: * Which is the safest and most effective dose to be given to children and adolescents. * Which effects PRX-102 has on signs and symptoms of Fabry disease (e.g. renal and cardiac function, pain, gastrointestinal symptoms…
Eligibility
- Age range
- 2–17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * 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. * History of Fabry pain: Fabry crises OR chronic pain. * Clinical condition that, in the investigator's opinion, requires ERT treatment. Exclusion Criteria: All Subjects: * Estimated glomerular…
Interventions
- DrugPRX-102 1 mg/kg every two weeks
Drug: PRX-102 1 mg/kg every two weeks
Locations (12)
- Phoenix Children'sPhoenix, Arizona
- Emory Genetics Clinical Trials CenterAtlanta, Georgia
- University of IowaIowa City, Iowa
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio
- University of UtahSalt Lake City, Utah
- Lysosomal and Rare Disorders Research and Treatment Center IncFairfax, Virginia