SMILE: Switch Over Study of Biosimilar AGA for Fabry Disease

Sponsor

Bio Sidus SA (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05843916

Collaborator

(none)

Enrollment

Location

Arm

11.6

Anticipated Duration (Months)

1.7

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Condition or Disease	Intervention/Treatment	Phase
Fabry Disease	Drug: Recombinant human alpha galactosidase A (agalsidase beta) Drug: Recombinant human alpha-galactosidase A (agalsidase beta)	Phase 3

Detailed Description

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 plasma Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as plasma level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by plasma level of the marker Lyso-Gb3 at baseline).

Study Design

Study Type:

Interventional

Anticipated Enrollment :

20 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model 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®.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®.

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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®

Actual Study Start Date :

Dec 13, 2022

Anticipated Primary Completion Date :

Dec 1, 2023

Anticipated Study Completion Date :

Dec 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: AGA BETA BS The study has a single-arm. First, all participants will receive 2 doses of Fabrazyme® with approximately 14 days between them, and afterwards all participants will switch treatment and receive AGA BETA BS for 54 weeks	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 Other Names: Fabrazyme 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 Other Names: AGA BETA BS

Arm

Intervention/Treatment

Experimental: AGA BETA BS

The study has a single-arm. First, all participants will receive 2 doses of Fabrazyme® with approximately 14 days between them, and afterwards all participants will switch treatment and receive AGA BETA BS for 54 weeks

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

Other Names:

Fabrazyme

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

Other Names:

AGA BETA BS

Outcome Measures

Primary Outcome Measures

Equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment [6 months]
• 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. Endpoint: • Mean plasma Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as plasma level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by plasma level of the marker Lyso-Gb3 at baseline.

Secondary Outcome Measures

Difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year of treatment [1 year]
• To evaluate the difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year of treatment in participants with Fabry disease previously stabilized with Fabrazyme®, by measuring disease biomarker. Endpoint: • Mean plasma Lyso-Gb3 marker ratio after 54 weeks of treatment, defined as plasma level of the marker Lyso-Gb3 after 54 weeks (12 months) divided by plasma level of the marker Lyso-Gb3 at baseline.
Compare the pain severity before and after 26 weeks of treatment with AGA BETA BS [26 weeks]
• To compare the pain severity before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 26 of treatment.
Compare the pain severity before and after 54 weeks of treatment with AGA BETA BS [54 weeks]
• To compare the pain severity before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 54 weeks of treatment.
Compare the impact of pain on daily functions before and after 26 weeks of treatment with AGA BETA [26 weeks]
• To compare the impact of pain on daily functions before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain interference as assessed by BPI-short form pain interference items scores after 26 weeks of treatment
Compare the impact of pain on daily functions before and after 54 weeks of treatment with AGA BETA [54 weeks]
• To compare the impact of pain on daily functions before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. Endpoint: • Change from baseline in pain interference as assessed by BPI-short form pain interference items scores after 54 weeks of treatment
Compare the participants' perception of their own health before and after 26 weeks of treatment with AGA BETA BS [26 weeks]
• To compare the participants' perception of their own health before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the SF (Short Form)-36. Endpoint: • Change from baseline in SF-36 scores after 26 weeks of treatment.
Compare the participants' perception of their own health before and after 54 weeks of treatment with AGA BETA BS [54 weeks]
• To compare the participants' perception of their own health before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the SF (Short Form)-36. Endpoint: • Change from baseline in SF-36 scores after 54 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count at baseline. [Baseline]
Platelet count at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count after 14 weeks of treatment. [14 weeks]
Platelet count at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count after 26 weeks of treatment. [26 weeks]
Platelet count at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of platelet count after 54 weeks of treatment. [54 weeks]
Platelet count at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count at baseline. [Baseline]
Red blood cell count at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count after 14 weeks of treatment [14 weeks]
Red blood cell count at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count after 26 weeks of treatment [26 weeks]
Red blood cell count at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of red blood cell count after 54 weeks of treatment [54 weeks]
Red blood cell count at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin at baseline [Baseline]
Hemoglobin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin after 14 weeks of treatment [14 weeks]
Hemoglobin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin after 26 weeks of treatment [26 weeks]
Hemoglobin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hemoglobin after 54 weeks of treatment [54 weeks]
Hemoglobin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit [Baseline]
Hematocrit at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit [14 weeks]
Hematocrit at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit [26 weeks]
Hematocrit at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit [54 weeks]
Hematocrit at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume [Baseline]
Mean corpuscular volume at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume [14 weeks]
Mean corpuscular volume at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume [26 weeks]
Mean corpuscular volume at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular volume [54 weeks]
Mean corpuscular volume at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin [Baseline]
Mean corpuscular hemoglobin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin [14 weeks]
Mean corpuscular hemoglobin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin [26 weeks]
Mean corpuscular hemoglobin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean corpuscular hemoglobin [54 weeks]
Mean corpuscular hemoglobin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration [Baseline]
Mean cell hemoglobin concentration at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration [14 weeks]
Mean cell hemoglobin concentration at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration [26 weeks]
Mean cell hemoglobin concentration at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of mean cell hemoglobin concentration [54 weeks]
Mean cell hemoglobin concentration at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes [Baseline]
%reticulocytes at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes [14 weeks]
%reticulocytes at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes [26 weeks]
%reticulocytes at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes [54 weeks]
%reticulocytes at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count [Baseline]
White blood cell count at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count [14 weeks]
White blood cell count at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count [26 weeks]
White blood cell count at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of white blood cell count [54 weeks]
White blood cell count at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils [Baseline]
Neutrophils at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils [14 weeks]
Neutrophils at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils [26 weeks]
Neutrophils at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils [54 weeks]
Neutrophils at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes [Baseline]
Lymphocytes at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes [14 weeks]
Lymphocytes at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes [26 weeks]
Lymphocytes at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes [54 weeks]
Lymphocytes at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes [Baseline]
Monocytes at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes [14 weeks]
Monocytes at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes [26 weeks]
Monocytes at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes [54 weeks]
Monocytes at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils [Baseline]
Eosinophils at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils [14 weeks]
Eosinophils at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils [26 weeks]
Eosinophils at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils [54 weeks]
Eosinophils at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils [Baseline]
Basophils at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils [14 weeks]
Basophils at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils [26 weeks]
Basophils at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils [54 weeks]
Basophils at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen [Baseline]
Blood urea nitrogen at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen [14 weeks]
Blood urea nitrogen at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen [26 weeks]
Blood urea nitrogen at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of blood urea nitrogen [54 weeks]
Blood urea nitrogen at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate [Baseline]
Phosphate at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate [14 weeks]
Phosphate at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate [26 weeks]
Phosphate at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate [54 weeks]
Phosphate at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine [Baseline]
Creatinine at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine [14 weeks]
Creatinine at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine [26 weeks]
Creatinine at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine [54 weeks]
Creatinine at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol [Baseline]
Total cholesterol at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol [14 weeks]
Total cholesterol at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol [26 weeks]
Total cholesterol at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total cholesterol [54 weeks]
Total cholesterol at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL [Baseline]
Cholesterol LDL at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL [14 weeks]
Cholesterol LDL at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL [26 weeks]
Cholesterol LDL at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol LDL [54 weeks]
Cholesterol LDL at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL [Baseline]
Cholesterol HDL at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL [14 weeks]
Cholesterol HDL at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL [26 weeks]
Cholesterol HDL at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of cholesterol HDL [54 weeks]
Cholesterol HDL at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides [Baseline]
Triglycerides at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides [14 weeks]
Triglycerides at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides [26 weeks]
Triglycerides at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides [54 weeks]
Triglycerides at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia [Baseline]
Glycemia at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia [14 weeks]
Glycemia at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia [26 weeks]
Glycemia at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia [54 weeks]
Glycemia at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin [Baseline]
Total bilirrubin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin [14 weeks]
Total bilirrubin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin [26 weeks]
Total bilirrubin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total bilirrubin [54 weeks]
Total bilirrubin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin [Baseline]
Direct bilirrubin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin [14 weeks]
Direct bilirrubin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin [26 weeks]
Direct bilirrubin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of direct bilirrubin [54 weeks]
Direct bilirrubin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase [Baseline]
Aspartate aminotransferase at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase [14 weeks]
Aspartate aminotransferase at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase [26 weeks]
Aspartate aminotransferase at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of aspartate aminotransferase [54 weeks]
Aspartate aminotransferase at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase [Baseline]
Alanine aminotransferase at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase [14 weeks]
Alanine aminotransferase at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase [26 weeks]
Alanine aminotransferase at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alanine aminotransferase [54 weeks]
Alanine aminotransferase at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium [Baseline]
Sodium at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium [14 weeks]
Sodium at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium [26 weeks]
Sodium at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium [54 weeks]
Sodium at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium [Baseline]
Potassium at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium [14 weeks]
Potassium at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium [26 weeks]
Potassium at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium [54 weeks]
Potassium at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine [Baseline]
Chlorine at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine [14 weeks]
Chlorine at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine [26 weeks]
Chlorine at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine [54 weeks]
Chlorine at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate [Baseline]
Bicarbonate at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate [14 weeks]
Bicarbonate at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate [26 weeks]
Bicarbonate at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate [54 weeks]
Bicarbonate at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium [Baseline]
Magnesium at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium [14 weeks]
Magnesium at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium [26 weeks]
Magnesium at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium [54 weeks]
Magnesium at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium [Baseline]
Calcium at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium [14 weeks]
Calcium at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium [26 weeks]
Calcium at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium [54 weeks]
Calcium at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase [Baseline]
Alkaline phosphatase at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase [14 weeks]
Alkaline phosphatase at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase [26 weeks]
Alkaline phosphatase at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of alkaline phosphatase [54 weeks]
Alkaline phosphatase at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins [Baseline]
Total proteins at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins [14 weeks]
Total proteins at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins [26 weeks]
Total proteins at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of total proteins [54 weeks]
Total proteins at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin [Baseline]
Albumin at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin [14 weeks]
Albumin at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin [26 weeks]
Albumin at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin [54 weeks]
Albumin at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase [Baseline]
Gamma glutamyl transferase at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase [14 weeks]
Gamma glutamyl transferase at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase [26 weeks]
Gamma glutamyl transferase at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of Gamma glutamyl transferase [54 weeks]
Gamma glutamyl transferase at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate [Baseline]
estimated Glomerular Filtration Rate at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate [14 weeks]
estimated Glomerular Filtration Rate at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate [26 weeks]
estimated Glomerular Filtration Rate at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of estimated Glomerular Filtration Rate [54 weeks]
estimated Glomerular Filtration Rate at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio [Baseline]
Urine albumin-creatinine ratio at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio [14 weeks]
Urine albumin-creatinine ratio at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio [26 weeks]
Urine albumin-creatinine ratio at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of urine albumin-creatinine ratio [54 weeks]
Urine albumin-creatinine ratio at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine [Baseline]
First morning urine at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine [14 weeks]
First morning urine at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine [26 weeks]
First morning urine at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the general evaluation of first morning urine [54 weeks]
First morning urine at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of electrocardiograms. [Baseline]
General evaluation of cardiac function based on the analysis of electrocardiogram exams performed at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of electrocardiograms. [26 weeks]
General evaluation of cardiac function based on the analysis of electrocardiogram exam performed after 26 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of electrocardiograms. [54 weeks]
General evaluation of cardiac function based on the analysis of electrocardiogram exam performed after 54 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of echocardiograms. [Baseline]
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exam performed at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of echocardiograms. [26 weeks]
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed after 26 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® regarding cardiac function through the evaluation of echocardiograms. [54 weeks]
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed after 54 weeks of treatment.
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples. [Baseline]
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at baseline
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples. [14 weeks]
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 14 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples. [26 weeks]
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 26 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of anti-AGA levels from blood samples. [54 weeks]
Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 54 weeks
To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the analysis of physical assessments, adverse events and infusion-related reactions. [54 weeks]
Analysis of data obtained from clinical and physical assessments, and from reported adverse events and infusion-related reactions throughout the clinical trial. This outcome will be measured in terms of Presence/Absence of relevant clinical findings.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Sex and Age

Male or female participant with ≥18 and ≤60 years of age at the time of signing the informed consent form (ICF).

Reproduction

Female participants who are not pregnant, breastfeeding, donating eggs (ova, oocytes), or considering becoming pregnant during the study and for 3 months after the last dose of study treatment.
All women of childbearing potential (WOCBP) must have a negative urine pregnancy test at the Screening visit and at Baseline visit (prior to the first dose of experimental intervention).
WOCBP must use one highly effective form of birth control contraception through the study and for 3 months after the last dose of study treatment (refer to Appendix 1 in Section 10.1).
Male participants who are not considering fathering a child during the study and for 3 months after the last dose of study treatment.
Male sexually active participant with female partner(s) of childbearing potential must agree to use male condoms during the study and for 3 months after the last dose of study treatment or have documented successful surgical sterilization.

Informed Consent

Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.

Type of Participant and Characteristics

Confirmed previous diagnosis of FD.
Women: preferably present genetic testing showing pathogenic GLA mutation consistent with FD at screening.
Men: preferably present leukocyte α-Gal A activity below normal range and/ or pathogenic GLA mutation consistent with FD at screening.
At least 50% of the participants will be male with classic FD phenotype. The remaining percentage will consist of male late onset and classic women FD phenotype.
Participants who have been on stable Fabrazyme® treatment for at least 6 months prior to Baseline visit.
Patients that in the last 3 months before the baseline visit have been receiving ≥80% of Fabrazyme®'s labeled dose/kg, this calculation includes both infusions provided by Biosidus during the Lead in period.
Disease status considered clinically stabilized, at Investigators' discretion.
Estimated glomerular filtration rate (eGFR) ≥45 mL/minute/1.73 m2 by CKD-EPI equation at Screening visit.
If receiving pain killers, angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs), participants must be in a stable dose for ≥ 4 weeks.

Exclusion Criteria:

Medical Conditions

Chronic kidney disease in stage 3b, 4, or 5.
History of dialysis, kidney transplant or participants who are on the waiting list for a kidney transplant.
Proteinuria ≥1 g/day at screening.
Participants who have suffered a clinical cardiovascular event (such as but not limited to myocardial infarction, transient ischemic attack) within 6 months prior to Screening visit.
Participants who have clinically significant unstable cardiac disease (such as but not limited to uncontrolled symptomatic arrhythmia, unstable angina, congestive heart failure New York Heart Association class III or IV).
Participants who have suffered a clinical cerebrovascular event (such as but not limited to stroke, transient ischemic attack) within 6 months prior to Screening visit.
History of anaphylaxis or other type I hypersensitivity reactions to agalsidase beta.
History of acute kidney injury in the 12 months prior to Screening visit (such as but not limited to acute interstitial nephritis, acute renal failure of glomerular origin or caused by vasculitis).
Presence of any medical, emotional, behavioral, or psychological condition that, according to the Investigator, would interfere with the participant's compliance with the requirements of the study.

Prior/Concomitant Therapy

Treatment initiation or change of dose of ACE inhibitors or ARBs in the 4 weeks before the screening.

Prior/Concurrent Clinical Trial Experience

Current participation in an interventional study, in which the participant received any drug within 90 days before the Screening visit.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Instituto de Investigaciones Clínicas Quilmes	Buenos Aires		Argentina

Sponsors and Collaborators

Bio Sidus SA

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Bio Sidus SA

ClinicalTrials.gov Identifier:

NCT05843916

Other Study ID Numbers:

BIO-AGA-Fase III-001

First Posted:

May 6, 2023

Last Update Posted:

May 6, 2023

Last Verified:

Apr 1, 2023

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Product Manufactured in and Exported from the U.S.:

Additional relevant MeSH terms:

Fabry Disease

Study Results

No Results Posted as of May 6, 2023