Switch Over Study of Biosimilar AGA for Fabry Disease

Bio Sidus SA20 enrolled

Overview

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 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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Fabry Disease

Interventions

Recombinant human alpha galactosidase A (agalsidase beta)DRUG

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

Recombinant human alpha-galactosidase A (agalsidase beta)DRUG

All participants will receive AGA BETA BS for 54 weeks. The dose will be 1 mg/kg of body mass every 2 weeks

Eligibility

Sex: ALLMin age: 18 YearsMax age: 60 Years

Medical Language ↔ Plain English

Inclusion Criteria: Sex and Age 1. Male or female participant with ≥18 and ≤60 years of age at the time of signing the informed consent form (ICF). Reproduction 2. 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. 3. 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). 4. 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). 5. Male participants who are not considering fathering a child during the study and for 3 months after the last dose of study treatment. 6. 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 7. 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 8. Confirmed previous diagnosis of FD. 1. Women: preferably present genetic testing showing pathogenic GLA mutation consistent with FD at screening. 2. Men: preferably present leukocyte α-Gal A activity below normal range and/ or pathogenic GLA mutation consistent with FD at screening. 3. 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. 9. Participants who have been on stable Fabrazyme® treatment for at least 6 months prior to Baseline visit. 10. 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. 11. Disease status considered clinically stabilized, at Investigators' discretion. 12. Estimated glomerular filtration rate (eGFR) ≥45 mL/minute/1.73 m2 by CKD-EPI equation at Screening visit. 13. 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 1. Chronic kidney disease in stage 3b, 4, or 5. 2. History of dialysis, kidney transplant or participants who are on the waiting list for a kidney transplant. 3. Proteinuria ≥1 g/day at screening. 4. 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. 5. 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). 6. 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. 7. History of anaphylaxis or other type I hypersensitivity reactions to agalsidase beta. 8. 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). 9. 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 10. Treatment initiation or change of dose of ACE inhibitors or ARBs in the 4 weeks before the screening. Prior/Concurrent Clinical Trial Experience 11. Current participation in an interventional study, in which the participant received any drug within 90 days before the Screening visit.

Locations (4)

Instituto de Nefrología Pergamino S.R.L

Pergamino, Buenos Aires, Argentina

RECRUITING

Centro Médico Santa María de la Salud

San Isidro, Buenos Aires, Argentina

RECRUITING

Instituto de Investigaciones Clínicas Quilmes

Buenos Aires, Argentina

RECRUITING

Outcomes

Primary Outcomes

Equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment

• 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.

Time frame: 6 months

Secondary Outcomes

Difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year of treatment

• 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.

Time frame: 1 year

Compare the pain severity before and after 26 weeks of treatment with AGA BETA BS

• 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.

Time frame: 26 weeks

Compare the pain severity before and after 54 weeks of treatment with AGA BETA BS

Central Contacts

Viridiana Berstein, MD

CONTACT

+5491159597061 v.berstein@biosidus.com.ar

Roberto A Diez, MD

CONTACT

+541149098000 r.diez@biosidus.com.ar

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Clínica Universitaria Reina Fabiola

Córdoba, Argentina

RECRUITING

Time frame: 54 weeks

Compare the impact of pain on daily functions before and after 26 weeks of treatment with AGA BETA

• 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

Time frame: 26 weeks

Compare the impact of pain on daily functions before and after 54 weeks of treatment with AGA BETA

Time frame: 54 weeks

Compare the participants' perception of their own health before and after 26 weeks of treatment with AGA BETA BS

• 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.

Time frame: 26 weeks

Compare the participants' perception of their own health before and after 54 weeks of treatment with AGA BETA BS

Time frame: 54 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 at baseline.

Platelet count at baseline

Time frame: 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.

Platelet count at 14 weeks

Time frame: 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.

Platelet count at 26 weeks

Time frame: 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.

Platelet count at 54 weeks

Time frame: 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.

Red blood cell count at baseline

Time frame: 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

Red blood cell count at 14 weeks

Time frame: 14 weeks

Red blood cell count at 26 weeks

Time frame: 26 weeks

Red blood cell count at 54 weeks

Time frame: 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

Hemoglobin at baseline

Time frame: 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

Hemoglobin at 14 weeks

Time frame: 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

Hemoglobin at 26 weeks

Time frame: 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

Hemoglobin at 54 weeks

Time frame: 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

Hematocrit at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of hematocrit

Hematocrit at 14 weeks

Time frame: 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

Hematocrit at 26 weeks

Time frame: 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

Hematocrit at 54 weeks

Time frame: 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

Mean corpuscular volume at baseline

Time frame: 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

Mean corpuscular volume at 14 weeks

Time frame: 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

Mean corpuscular volume at 26 weeks

Time frame: 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

Mean corpuscular volume at 54 weeks

Time frame: 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

Mean corpuscular hemoglobin at baseline

Time frame: 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

Mean corpuscular hemoglobin at 14 weeks

Time frame: 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

Mean corpuscular hemoglobin at 26 weeks

Time frame: 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

Mean corpuscular hemoglobin at 54 weeks

Time frame: 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

Mean cell hemoglobin concentration at baseline

Time frame: 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

Mean cell hemoglobin concentration at 14 weeks

Time frame: 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

Mean cell hemoglobin concentration at 26 weeks

Time frame: 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

Mean cell hemoglobin concentration at 54 weeks

Time frame: 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

%reticulocytes at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of %reticulocytes

%reticulocytes at 14 weeks

Time frame: 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

%reticulocytes at 26 weeks

Time frame: 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

%reticulocytes at 54 weeks

Time frame: 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

White blood cell count at baseline

Time frame: 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

White blood cell count at 14 weeks

Time frame: 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

White blood cell count at 26 weeks

Time frame: 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

White blood cell count at 54 weeks

Time frame: 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

Neutrophils at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of neutrophils

Neutrophils at 14 weeks

Time frame: 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

Neutrophils at 26 weeks

Time frame: 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

Neutrophils at 54 weeks

Time frame: 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

Lymphocytes at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of lymphocytes

Lymphocytes at 14 weeks

Time frame: 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

Lymphocytes at 26 weeks

Time frame: 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

Lymphocytes at 54 weeks

Time frame: 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

Monocytes at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of monocytes

Monocytes at 14 weeks

Time frame: 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

Monocytes at 26 weeks

Time frame: 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

Monocytes at 54 weeks

Time frame: 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

Eosinophils at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of eosinophils

Eosinophils at 14 weeks

Time frame: 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

Eosinophils at 26 weeks

Time frame: 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

Eosinophils at 54 weeks

Time frame: 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

Basophils at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of basophils

Basophils at 14 weeks

Time frame: 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

Basophils at 26 weeks

Time frame: 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

Basophils at 54 weeks

Time frame: 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

Blood urea nitrogen at baseline

Time frame: 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

Blood urea nitrogen at 14 weeks

Time frame: 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

Blood urea nitrogen at 26 weeks

Time frame: 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

Blood urea nitrogen at 54 weeks

Time frame: 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

Phosphate at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of phosphate

Phosphate at 14 weeks

Time frame: 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

Phosphate at 26 weeks

Time frame: 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

Phosphate at 54 weeks

Time frame: 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

Creatinine at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of creatinine

Creatinine at 14 weeks

Time frame: 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

Creatinine at 26 weeks

Time frame: 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

Creatinine at 54 weeks

Time frame: 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

Total cholesterol at baseline

Time frame: 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

Total cholesterol at 14 weeks

Time frame: 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

Total cholesterol at 26 weeks

Time frame: 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

Total cholesterol at 54 weeks

Time frame: 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

Cholesterol LDL at baseline

Time frame: 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

Cholesterol LDL at 14 weeks

Time frame: 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

Cholesterol LDL at 26 weeks

Time frame: 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

Cholesterol LDL at 54 weeks

Time frame: 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

Cholesterol HDL at baseline

Time frame: 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

Cholesterol HDL at 14 weeks

Time frame: 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

Cholesterol HDL at 26 weeks

Time frame: 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

Cholesterol HDL at 54 weeks

Time frame: 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

Triglycerides at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of triglycerides

Triglycerides at 14 weeks

Time frame: 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

Triglycerides at 26 weeks

Time frame: 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

Triglycerides at 54 weeks

Time frame: 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

Glycemia at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of glycemia

Glycemia at 14 weeks

Time frame: 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

Glycemia at 26 weeks

Time frame: 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

Glycemia at 54 weeks

Time frame: 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

Total bilirrubin at baseline

Time frame: 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

Total bilirrubin at 14 weeks

Time frame: 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

Total bilirrubin at 26 weeks

Time frame: 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

Total bilirrubin at 54 weeks

Time frame: 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

Direct bilirrubin at baseline

Time frame: 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

Direct bilirrubin at 14 weeks

Time frame: 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

Direct bilirrubin at 26 weeks

Time frame: 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

Direct bilirrubin at 54 weeks

Time frame: 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

Aspartate aminotransferase at baseline

Time frame: 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

Aspartate aminotransferase at 14 weeks

Time frame: 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

Aspartate aminotransferase at 26 weeks

Time frame: 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

Aspartate aminotransferase at 54 weeks

Time frame: 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

Alanine aminotransferase at baseline

Time frame: 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

Alanine aminotransferase at 14 weeks

Time frame: 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

Alanine aminotransferase at 26 weeks

Time frame: 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

Alanine aminotransferase at 54 weeks

Time frame: 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

Sodium at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of sodium

Sodium at 14 weeks

Time frame: 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

Sodium at 26 weeks

Time frame: 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

Sodium at 54 weeks

Time frame: 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

Potassium at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of potassium

Potassium at 14 weeks

Time frame: 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

Potassium at 26 weeks

Time frame: 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

Potassium at 54 weeks

Time frame: 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

Chlorine at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of chlorine

Chlorine at 14 weeks

Time frame: 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

Chlorine at 26 weeks

Time frame: 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

Chlorine at 54 weeks

Time frame: 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

Bicarbonate at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of bicarbonate

Bicarbonate at 14 weeks

Time frame: 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

Bicarbonate at 26 weeks

Time frame: 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

Bicarbonate at 54 weeks

Time frame: 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

Magnesium at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of magnesium

Magnesium at 14 weeks

Time frame: 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

Magnesium at 26 weeks

Time frame: 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

Magnesium at 54 weeks

Time frame: 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

Calcium at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of calcium

Calcium at 14 weeks

Time frame: 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

Calcium at 26 weeks

Time frame: 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

Calcium at 54 weeks

Time frame: 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

Alkaline phosphatase at baseline

Time frame: 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

Alkaline phosphatase at 14 weeks

Time frame: 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

Alkaline phosphatase at 26 weeks

Time frame: 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

Alkaline phosphatase at 54 weeks

Time frame: 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

Total proteins at baseline

Time frame: 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

Total proteins at 14 weeks

Time frame: 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

Total proteins at 26 weeks

Time frame: 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

Total proteins at 54 weeks

Time frame: 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

Albumin at baseline

Time frame: Baseline

To characterize the safety of AGA BETA BS treatment in participants with Fabry disease previously stabilized with Fabrazyme® through the evaluation of albumin

Albumin at 14 weeks

Time frame: 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

Albumin at 26 weeks

Time frame: 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

Albumin at 54 weeks

Time frame: 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

Gamma glutamyl transferase at baseline

Time frame: 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

Gamma glutamyl transferase at 14 weeks

Time frame: 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

Gamma glutamyl transferase at 26 weeks

Time frame: 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

Gamma glutamyl transferase at 54 weeks

Time frame: 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

estimated Glomerular Filtration Rate at baseline

Time frame: 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

estimated Glomerular Filtration Rate at 14 weeks

Time frame: 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

estimated Glomerular Filtration Rate at 26 weeks

Time frame: 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

estimated Glomerular Filtration Rate at 54 weeks

Time frame: 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

Urine albumin-creatinine ratio at baseline

Time frame: 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

Urine albumin-creatinine ratio at 14 weeks

Time frame: 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

Urine albumin-creatinine ratio at 26 weeks

Time frame: 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

Urine albumin-creatinine ratio at 54 weeks

Time frame: 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

First morning urine at baseline

Time frame: 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

First morning urine at 14 weeks

Time frame: 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

First morning urine at 26 weeks

Time frame: 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

First morning urine at 54 weeks

Time frame: 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.

General evaluation of cardiac function based on the analysis of electrocardiogram exams performed at baseline

Time frame: 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.

General evaluation of cardiac function based on the analysis of electrocardiogram exam performed after 26 weeks of treatment.

Time frame: 26 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.

General evaluation of cardiac function based on the analysis of electrocardiogram exam performed after 54 weeks of treatment.

Time frame: 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 echocardiograms.

Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exam performed at baseline

Time frame: 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.

Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed after 26 weeks of treatment.

Time frame: 26 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 echocardiograms.

Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed after 54 weeks of treatment.

Time frame: 54 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.

Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at baseline

Time frame: 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.

Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 14 weeks

Time frame: 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.

Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 26 weeks

Time frame: 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.

Evaluation of immunogenicity based on the analysis of anti-AGA levels from blood samples collected at 54 weeks

Time frame: 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.

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.

Time frame: 54 weeks