PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion

NCT05343715 | Completed Phase 1 Results

• 1 other identifier: AGA Biosidus
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

Pharmacokinetic/Pharmacodynamic Study of 2 agalsidase beta Formulations in Single Dose Administered to Healthy Volunteers as intravenous infusion, at a concentration of 1 mg/kg

Conditions

Fabry Disease

Keywords

Fabry Disease; agalsidase beta; phase 1

Outcome Measures

Primary Outcomes (4)

• Maximum Serum Concentration of Agalsidase Beta (Cmax)

  Compare the maximum serum concentration of two formulations of agalsidase beta after intravenous infusion of 1 mg/kg: * Fabrazyme (Sanofi-Genzyme) (Reference Formulation, "R") * Agalsidase beta from Biosidus SA (Test Formulation, "T")

  0, 1, 3, 5, 5.5, 6, 7, 8, 10 and 12 hours from the beginning of the infusion

• Area Under the Curve of Serum Concentration Between Times 0 and 12 Hours of Agalsidase Beta

  Compare the area under the curve between times 0 and 12 hours of two formulations of agalsidase beta after intravenous infusion of 1 mg/kg: * Fabrazyme (Sanofi-Genzyme) (Reference Formulation, "R") * Agalsidase beta from Biosidus SA (Test Formulation, "T")

  0, 1, 3, 5, 5.5, 6, 7, 8, 10 and 12 hours from the beginning of the infusion

• Time at Which Maximum Serum Concentration of Agalsidase Beta is Observed (Tmax)

  Compare the time at which maximum serum concentration of agalsidase beta is observed of two formulations of agalsidase beta after intravenous infusion of 1 mg/kg: * Fabrazyme (Sanofi-Genzyme) (Reference Formulation, "R") * Agalsidase beta from Biosidus SA (Test Formulation, "T")

  0, 1, 3, 5, 5.5, 6, 7, 8, 10 and 12 hours from the beginning of the infusion

• Area Under the Curve of Serum Concentration, Resulting From the Extrapolation From Time 0 to Infinite Time

  Compare the area under the curve of serum concentration, resulting from the extrapolation from time 0 to infinite time of two formulations of agalsidase beta after intravenous infusion of 1 mg/kg: * Fabrazyme (Sanofi-Genzyme) (Reference Formulation, "R") * Agalsidase beta from Biosidus SA (Test Formulation, "T")

  0, 1, 3, 5, 5.5, 6, 7, 8, 10 and 12 hours from the beginning of the infusion

Secondary Outcomes (3)

• Enzymatic Activity on Plasma Samples Measured With Fluorometric Method

  Pre-infusion and 5 hours post administration (end of infusion)

• Number of Patients With Adverse Events

  From infusion to 35 days after

• Number of Participants With Positive Results for the Presence of Neutralizing Antibodies Against Agalsidase Beta

  0 hours,12 hours and 35 days after the end of the infusion.

Study Arms (2)

Agalsidase Beta from Biosidus

EXPERIMENTAL

Participants received a single infusion at a dose 1 mg/kg

Biological: Agalsidase beta from Biosidus 1 mg/kg

Fabrazyme (Sanofi-Genzyme)

ACTIVE COMPARATOR

Participants received a single infusion at a dose 1 mg/kg

Biological: Fabrazyme (agalsidase beta) 1 mg/kg

Interventions

Agalsidase beta from Biosidus 1 mg/kg BIOLOGICAL

Patients received an infusion of 1 mg/kg of Agalsidase Beta from Biosidus. Final infusion dose is achieved with lyophilized drug at concentration 35 mg and 5 mg

Agalsidase Beta from Biosidus

Fabrazyme (agalsidase beta) 1 mg/kg BIOLOGICAL

Patients received an infusion of 1 mg/kg of Fabrazyme. Final infusion dose is achieved with lyophilized drug at concentration 35 mg and 5 mg

Fabrazyme (Sanofi-Genzyme)

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Male subjects between the ages of 18 and 40
• Body mass index (BMI) between 19 and 25 kg/m².
• Subjects with systolic blood pressure higher than 110 mmHg and lower than 139 mmHg, diastolic blood pressure higher than 70 mmHg and lower than 89 mmHg with a heart rate higher than 50 and lower than 90 beats per minute after being seated for 5 minutes and, then, standing up (extreme values are included)
• Volunteers who are well disposed to the study and have signed the approved informed consent prior to the start of the study

You may not qualify if:

• History of clinically significant allergies (except for untreated asymptomatic seasonal allergies)
• A drop of ≥ 20 mmHg of systolic blood pressure or ≥ 10 mmHg of diastolic blood pressure within the first 3 minutes after the postural change
• Volunteers who are receiving other drugs (prescription or over-the-counter) or who have within the 2 weeks prior to the study
• Volunteers with history of autoimmune diseases.
• Chronic disorders of the CNS, psychological and/or psychiatric disorders: bipolar disorder, severe depression, insomnia, changes in personality
• Active or chronic infections
• Having received live or inactivated viral or bacterial vaccines, within the fifteen days prior to visits 1 or 2
• For COVID vaccines, a 15 day window period prior to visits #1 or #2 must be respected. This period was taken from the recommendations published by the National Ministry of Health on May 29th 2021. The volunteer will be able to receive the vaccine 35 days after receiving the investigational product.
• Known allergies to any of the components of the formulations
• Active smoker, of more than 10 cigarettes/day
• Current clinical evidence of severe digestive disorders, surgeries of the gastrointestinal tract (except for appendectomy)
• Current clinical evidence of kidney disease
• Current clinical evidence of liver disorders
• Current clinical evidence of respiratory and cardiac disease
• Presence of diabetes mellitus, thyroid dysfunction or other endocrine disorders

Sponsors & Collaborators

• Bio Sidus SA: lead

Study Sites (1)

Sanatorio Nuestra Señora del Pilar

Ciudadela, Buenos Aires, 1702, 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

Results Point of Contact

• Title: Director of Clinical Trials
• Organization: Biosidus S.A.U.

v.berstein@biosidus.com.ar

+541149098044

Study Officials

• Eduardo Pirotzky, MD

  Sanatorio Nuestra Señora del Pilar

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 24, 2022
• First Posted: April 25, 2022
• Study Start: October 23, 2021
• Primary Completion: March 6, 2022
• Study Completion: April 17, 2022
• Last Updated: October 15, 2025
• Results First Posted: March 15, 2024

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

AR (1)