Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease

NCT05843916 | Completed Phase 3 Results

• 1 other identifier: BIO-AGA-Fase III-001
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 5

Brief Summary

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

Conditions

Fabry Disease

Keywords

Biosimilar; Fabry disease; Fabry; agalsidase beta; Switch over

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Lyso Gb3 Serum Levels

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

  Baseline (after 5 week lead-in period on Fabrazyme®) and 26 weeks (after the switch to AGA BETA BS)

Secondary Outcomes (14)

• Change From Baseline in Lyso-Gb3 Serum Levels

  Baseline (after 5 week lead-in period on Fabrazyme®) and 54 weeks (after the switch to AGA BETA BS)

• Change From Baseline in Pain Severity as Assessed by Brief Pain Inventory-short Form Pain Severity Items Scores

  Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).

• Change From Baseline in Pain Interference as Assessed by BPI-short Form Pain Interference Items Scores

  Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).

• Change From Baseline in SF-36 Health Survey Scores

  Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).

• Mean Blood Urea Nitrogen (BUN) Levels at Specified Timepoints

  Baseline (after 5 week lead-in period on Fabrazyme®), 14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS).

Study Arms (1)

Fabrazyme® then AGA BETA BS

EXPERIMENTAL

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); Drug: Recombinant human alpha-galactosidase A (agalsidase beta)

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

Also known as: Fabrazyme

Fabrazyme® then AGA BETA BS

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

Also known as: AGA BETA BS

Fabrazyme® then AGA BETA BS

Eligibility Criteria

• Age: 16 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Sex and Age
• Male or female participant with ≥16 and ≤60 years of age at the time of signing the informed consent form (ICF).
• Reproduction
• Female participants who are not pregnant, breastfeeding, donating eggs (ova, oocytes), or considering becoming pregnant during the study and for 3 months after the last dose of study treatment.
• All women of childbearing potential (WOCBP) must have a negative urine pregnancy test at the Screening visit and at Baseline visit (prior to the first dose of experimental intervention).
• WOCBP must use one highly effective form of birth control contraception through the study and for 3 months after the last dose of study treatment.
• Male participants who are not considering fathering a child during the study and for 3 months after the last dose of study treatment.
• Male sexually active participant with female partner(s) of childbearing potential must agree to use male condoms during the study and for 3 months after the last dose of study treatment or have documented successful surgical sterilization.
• Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
• Confirmed previous diagnosis of FD.
• Women: preferably present genetic testing showing pathogenic GLA mutation consistent with FD at screening.
• Men: preferably present leukocyte α-Gal A activity below normal range and/ or pathogenic GLA mutation consistent with FD at screening.
• Male with classic FD phenotype, female with classic FD and men with late onset may be included.
• Participants who have been on stable Fabrazyme® treatment for at least 6 months prior to Baseline visit.
• Patients that in the last 3 months before the baseline visit have been receiving ≥80% of Fabrazyme®'s labeled dose/kg, this calculation includes both infusions provided by Biosidus during the Lead in period.

You may not qualify if:

• Chronic kidney disease in stage 3b, 4, or 5.
• History of dialysis, kidney transplant or participants who are on the waiting list for a kidney transplant.
• Proteinuria ≥1 g/day at screening.
• Participants who have suffered a clinical cardiovascular event (such as but not limited to myocardial infarction, transient ischemic attack) within 6 months prior to
• Participants who have clinically significant unstable cardiac disease (such as but not limited to uncontrolled symptomatic arrhythmia, unstable angina, congestive heart failure New York Heart Association class III or IV).
• Participants who have suffered a clinical cerebrovascular event (such as but not limited to stroke, transient ischemic attack) within 6 months prior to Screening visit.
• History of anaphylaxis or other type I hypersensitivity reactions to agalsidase beta.
• History of acute kidney injury in the 12 months prior to Screening visit (such as but not limited to acute interstitial nephritis, acute renal failure of glomerular origin or caused by vasculitis).
• Presence of any medical, emotional, behavioral, or psychological condition that, according to the Investigator, would interfere with the participant's compliance with the requirements of the study.
• Treatment initiation or change of dose of ACE inhibitors or ARBs in the 4 weeks before the screening.
• Current participation in an interventional study, in which the participant received any drug within 90 days before the Screening visit.

Sponsors & Collaborators

• Bio Sidus SA: lead
• Amsterdam UMC: collaborator
• IQVIA Solutions: collaborator
• IQVIA RDS Inc.: collaborator

Study Sites (5)

Instituto de Nefrología Pergamino S.R.L

Pergamino, Buenos Aires, 2700, Argentina

Location

Centro Médico Santa María de la Salud

San Isidro, Buenos Aires, 1642, Argentina

Location

Instituto de Investigaciones Clínicas Quilmes

Buenos Aires, Argentina

Location

Clínica Universitaria Reina Fabiola

Córdoba, X5004, Argentina

Location

Centro Oncológico Riojano Integral

La Rioja, F5300, Argentina

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase beta

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Limitations and Caveats

This switch-over study was conducted in a small, heterogeneous cohort typical of rare-disease research. Limitations include the absence of a parallel control group, limited sample size, and a follow-up duration that may be insufficient for long-term assessment in a chronic progressive disease. These constraints are inherent to studies in rare diseases and were anticipated a priori.

Results Point of Contact

• Title: Nicolás Manuel Antognoni
• Organization: Biosidus S.A.U.

n.antognoni@biosidus.com.ar

+54 9 1140983909

Study Officials

• Alberto A Fernández, MD

  Instituto de Investigaciones Clínicas Quilmes

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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 Record Dates

• First Submitted: November 28, 2022
• First Posted: May 6, 2023
• Study Start: December 13, 2022
• Primary Completion: September 19, 2024
• Study Completion: March 31, 2025
• Last Updated: February 18, 2026
• Results First Posted: February 18, 2026

Record last verified: 2026-01

Locations

AR (5)