Study of PRX-102 Enzyme Replacement Therapy in Children and Adolescents Ages 2 to 17 With Fabry Disease to Assess Safety and Effects

What this Trial Is About

Researchers are evaluating the safety and effects of the study drug pegunigalsidase alfa PRX-102 in children and adolescents aged 2 to 17 years with Fabry disease. This enzyme replacement therapy is given intravenously every two weeks. The study focuses on finding the safest and most effective dose and understanding how PRX-102 impacts symptoms of Fabry disease including kidney and heart function, pain, and digestive issues. The trial includes 20 to 22 boys and girls divided into three age groups 2 to 7 years, 8 to 12 years, and 13 to under 18 years. The study has three stages Stage I to find the right dose, Stage II to confirm safety and how well the drug works, and an optional Stage III extension that continues until the drug is commercially available or the study ends. PRX-102 is given every two weeks during study visits, and doses may be adjusted based on early results. Participants will have regular visits every two weeks for treatment and assessments. These include physical exams, heart ultrasounds and ECGs, questionnaires on symptoms and quality of life, and blood and urine tests to monitor safety, disease severity, and how the drug behaves in the body. Blood samples are taken multiple times over several days to study drug levels. The main outcomes measured include side effects, heart and kidney function, pain levels, and quality of life over 12 months.

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease