Switch Over Study of Biosimilar AGA for Fabry Disease (SMILE)

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®

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®.

Study Overview

• Status: Recruiting
• Conditions: Fabry Disease
• Intervention / Treatment: Drug: Recombinant human alpha galactosidase A (agalsidase beta); Drug: Recombinant human alpha-galactosidase A (agalsidase beta)

Detailed Description

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

The study will be conducted in 2 parts: a 5-week Lead-in period (period 1) and 54 week treatment period (period 2). During period 1 all participants will receive 2 intravenous (IV) infusions of Fabrazyme®, provided by Biosidus. After that, in period 2 all participants will switch treatment to AGA BETA BS.

A total of up to 20 participants are planned for the study.

•The primary objective of the study is to evaluate the equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment in participants with Fabry disease previously stabilized with Fabrazyme, by measuring disease biomarker (mean 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
• Enrollment (Estimated): 20
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Viridiana Berstein, MD; Phone Number: +5491159597061; Email: v.berstein@biosidus.com.ar
• Study Contact Backup: Name: Roberto A Diez, MD; Phone Number: +541149098000; Email: r.diez@biosidus.com.ar

Study Locations

• Argentina
  • Buenos Aires, Argentina
    • Recruiting
    • Instituto De Investigaciones Clinicas Quilmes
    • Contact: Alberto Fernandez; Phone Number: 60634883; Email: afernan59@hotmail.com
  • Córdoba, Argentina, X5004
    • Recruiting
    • Clínica Universitaria Reina Fabiola
    • Contact: Norberto B Guelbert, MD; Phone Number: (0351)4142121; Email: gguelbert@gmail.com
    • Principal Investigator: Norberto B Guelbert, MD
  • Buenos Aires
    • Pergamino, Buenos Aires, Argentina, 2700
      • Recruiting
      • Instituto de Nefrología Pergamino S.R.L
      • Contact: Norberto R Antongiovanni, MD; Phone Number: +54 2477-430597; Email: nantongiovanni1@gmail.com
      • Principal Investigator: Norberto R Antongiovanni, MD
    • San Isidro, Buenos Aires, Argentina, 1642
      • Recruiting
      • Centro Médico Santa María de la Salud
      • Contact: Gustavo H Cabrera, MD; Phone Number: +54 114742-3598; Email: gustavo.h.cabrera@hotmail.com
      • Principal Investigator: Gustavo H Cabrera, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	26 weeks
Compare the pain severity before and after 54 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 54 weeks of treatment.	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	26 weeks
Compare the impact of pain on daily functions before and after 54 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 54 weeks of treatment	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.	26 weeks
Compare the participants' perception of their own health before and after 54 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 54 weeks of treatment.	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	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	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	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	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	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	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	Red blood cell count at 26 weeks	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	Red blood cell count at 54 weeks	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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	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.	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.	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	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.	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.	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	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	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	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	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.	54 weeks

Collaborators and Investigators

• Sponsor: Bio Sidus SA
• Investigators: Principal Investigator: Alberto A Fernández, MD, Instituto De Investigaciones Clinicas Quilmes

Study record dates

Study Major Dates

• Study Start (Actual): December 13, 2022
• Primary Completion (Estimated): December 1, 2023
• Study Completion (Estimated): December 1, 2023

Study Registration Dates

• First Submitted: November 28, 2022
• First Submitted That Met QC Criteria: April 24, 2023
• First Posted (Actual): May 6, 2023

Study Record Updates

• Last Update Posted (Actual): July 19, 2023
• Last Update Submitted That Met QC Criteria: July 18, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Metabolic Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Cerebral Small Vessel Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Fabry Disease
• Other Study ID Numbers: BIO-AGA-Fase III-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No