PRX-102 in Children and Adolescents with Fabry disease
Research type
Research Study
Full title
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 (FLY)
IRAS ID
1010087
Contact name
Giovanni Piotti
Contact email
Sponsor organisation
Chiesi Farmaceutici S.p.A.
ISRCTN Number
ISRCTN14012649
Clinicaltrials.gov Identifier
Research summary
Fabry disease is a genetic disorder in which the body does not properly produce an enzyme that is needed to break down a type of fat called (Gb3 for short). If this enzyme is missing or not working properly, Gb3 builds up in cells, which can lead to serious health problems, such as heart disease, kidney failure and strokes. The drug being tested is called pegunigalsidase alfa or PRX-102 which is approved for sale and has shown to be effective in treating adult patients affected by Fabry disease. It is a type of enzyme replacement therapy, meaning that like the natural enzyme, it acts to break down Gb3. It is given by intravenous infusion (through a needle placed in a vein) every 2 weeks. PRX-102 is an investigational drug in children and adolescents, meaning that it is not approved for sale in these age groups and is being studied to see how well it works and how safe it is.
We are conducting this study in children and adolescents with Fabry disease to learn more about the drug that is designed to prevent or reduce the development of health problems caused by this disease. Since the symptoms of Fabry disease appear at a young age and get worse over time, a treatment that would help to slow or stop the irreversible damage that the disease causes to organs would improve patients’ health and quality of life. At this time, not many treatments have been approved for children, so there is an important need to develop new therapies. The goals of this study are to see if this new drug is safe in children and adolescents and to see how well it works.
The study is divided into three parts, or “stages”: a dose-finding stage (Stage I), to try and determine the best dose for each age group which will last up to 6 months, a confirmatory stage (Stage II), which will last for up to 12 months; and an optional extension stage.
The study will take place in six countries globally including at one UK site and approximately 20-22 participants will be enrolled in the study globally.REC name
North of Scotland Research Ethics Committee 1
REC reference
24/NS/0061
Date of REC Opinion
2 Jul 2024
REC opinion
Further Information Favourable Opinion