NCT04840667

A Study of Replagal in Treatment-naïve Adults With Fabry Disease

A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects With Fabry Disease

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

In this study, adults with Fabry Disease who have not had any treatment for this condition will be treated with Replagal. The main aim of the study is to check if Replagal improves kidney function and heart structure of participants with Fabry Disease. Participants will receive one Replagal infusion every other week for up to 104 weeks. They will visit the clinic every 12 to 14 weeks during treatment with a follow-up visit 2 weeks after treatment.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	REPLAGAL	Participants will receive REPLAGAL 0.2 mg/kg body weight of IV infusion for 104 weeks.

Timeline

• Start date: 2021-12-28
• Primary completion: 2022-12-16
• Completion: 2022-12-16
• First posted: 2021-04-12
• Last updated: 2024-06-17
• Results posted: 2024-06-17

Locations

28 sites across 8 countries: Canada, Finland, Germany, Greece, Poland, Portugal, Spain, Sweden