NCT05710692

Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease (RISE)

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: Chiesi Farmaceutici S.p.A. · Industry
• Sex: All
• Age: 13 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Investigators are doing this study to find out if treatment with pegunigalsidase alfa will prevent or reduce the development of health problems caused by Fabry disease and thereby improve patients' health and quality of life. pegunigalsidase alfa (PRX-102) is a drug made using genetic engineering techniques and manufactured using cultured tobacco cells. It is given by intravenous infusion every 2 weeks, at a dosage of 1 milligram per kilogram (mg/kg) of body weight. The study consists of a main study that is divided into two stages, each of which will last one year, followed by an optional extension study. In stage II of main study and in the optional extension study, the participants may receive PRX-102 intravenous infusion every 2 weeks, at a dosage of 1 milligram per kilogram (mg/kg) of body weight or every 4 weeks at a dosage of 2 milligrams per kilogram (mg/kg) of body weight. There are three groups (cohorts) in this study, with adults enrolled in either Cohort A or B and adolescents in Cohort C. Whether an adult is assigned to Cohort A or Cohort B depends on their kidney function and treatment history. This study will start with a screening visit of up to 6 weeks. It will be followed up by infusion visits every 2 weeks or 4 weeks. For subjects not continuing in the extension stage, a follow-up call is to be made 30 days after the last study drug infusion.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	PRX-102 1 mg/kg every 2 weeks	PRX-102 1 mg/kg every 2 weeks
DRUG	PRX-102 2 mg/kg every 4 weeks	PRX-102 2 mg/kg every 4 weeks

Timeline

• Start date: 2023-08-01
• Primary completion: 2027-10-01
• Completion: 2029-08-01
• First posted: 2023-02-02
• Last updated: 2026-03-18

Locations

10 sites across 1 country: Japan