NCT05843916

Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease

Phase III, Open-label, Switch Over Trial of the Efficacy and Safety of Agalsidase Beta Biosidus (AGA BETA BS) in Fabry Disease Patients Previously Stabilized With Fabrazyme®

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Bio Sidus SA · Industry
• Sex: All
• Age: 16 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and previously stabilized with Fabrazyme®.

Detailed description

BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and previously stabilized with Fabrazyme®. The study will be conducted in 2 parts: a 5-week Lead-in period (period 1) and 54 week treatment period (period 2). During period 1 all participants will receive 2 intravenous (IV) infusions of Fabrazyme®, provided by Biosidus. After that, in period 2 all participants will switch treatment to AGA BETA BS. A total of up to 20 participants are planned for the study. •The primary objective of the study is to evaluate the equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment in participants with Fabry disease previously stabilized with Fabrazyme, by measuring disease biomarker (mean serum Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as serum level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by serum level of the marker Lyso-Gb3 at baseline).

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Recombinant human alpha galactosidase A (agalsidase beta)	All participants will receive 2 doses of Fabrazyme® with approximately 14 days between them. The dose will be 1 mg/kg of body mass every 2 weeks
DRUG	Recombinant human alpha-galactosidase A (agalsidase beta)	All participants will receive AGA BETA BS for 54 weeks. The dose will be 1 mg/kg of body mass every 2 weeks

Timeline

• Start date: 2022-12-13
• Primary completion: 2024-09-19
• Completion: 2025-03-31
• First posted: 2023-05-06
• Last updated: 2026-02-18
• Results posted: 2026-02-18

Locations

5 sites across 1 country: Argentina