drugset / Trial / NCT05710692
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
NaSingle-groupOpen-labelTreatment
Summary
The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease. It is planned of a total of approximately 16 male and female Fabry disease patients between the ages of 13 and 70 years to be part of the study. The study is conducted in Japan.
Timeline
- Start
- 2023-08-01
- Primary completion
- 2027-10
- Completion
- 2029-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | pegunigalsidase alfa | Protein / enzyme biologic | 1 mg/kg | Intravenous |
| Subject | pegunigalsidase alfa | Protein / enzyme biologic | 2 mg/kg | Intravenous |