Switching from Fabrazyme to a new biosimilar treatment for Fabry Disease

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

This study is testing if switching from Fabrazyme to a new treatment called AGA BETA BS is just as effective for people with Fabry Disease who are already stable on Fabrazyme.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 18 Years to 60 Years
• Sex: All
• Sponsor: Bio Sidus SA (Industry-sponsored)
• Locations: 4 sites (Pergamino, Buenos Aires and 3 other locations)
• Trial ID: NCT05843916 on ClinicalTrials.gov

What this trial studies

This Phase III clinical trial evaluates the efficacy and safety of AGA BETA BS, a biosimilar treatment for Fabry Disease, in patients who have been stabilized on Fabrazyme. The study consists of two parts: a 5-week lead-in period where participants receive Fabrazyme, followed by a 54-week treatment period where they switch to AGA BETA BS. The primary objective is to assess the equivalence in efficacy by measuring the disease biomarker Lyso-Gb3 after 6 months of treatment. A total of up to 20 participants will be enrolled across multiple centers.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 18 to 60 who have been previously stabilized on Fabrazyme.

Not a fit: Patients who are not currently stabilized on Fabrazyme or are outside the age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide an effective alternative to Fabrazyme for patients with Fabry Disease.

How similar studies have performed: Other studies have shown success with biosimilar treatments in similar conditions, indicating potential for this approach.

Eligibility criteria

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.

Where this trial is running

Pergamino, Buenos Aires and 3 other locations

• 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)
• Clínica Universitaria Reina Fabiola — Córdoba, Argentina (Recruiting)

Study contacts

• Principal investigator: Alberto A Fernández, MD — Instituto de Investigaciones Clínicas Quilmes
• Study coordinator: Viridiana Berstein, MD

Conditions Fabry Disease