Trial Outcomes & Findings for PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion (NCT NCT05343715)

NCT ID: NCT05343715

Last Updated: 2025-10-15

Results Overview

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")

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 24 participants
• Primary outcome timeframe: 0, 1, 3, 5, 5.5, 6, 7, 8, 10 and 12 hours from the beginning of the infusion
• Results posted on: 2025-10-15

Participant Flow

Participant milestones

Measure	Agalsidase Beta from Biosidus Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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
Overall Study STARTED	12	12
Overall Study COMPLETED	12	12
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Agalsidase Beta from Biosidus n=12 Participants Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) n=12 Participants Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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	Total n=24 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=5 Participants	12 Participants n=7 Participants	24 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	12 Participants n=7 Participants	24 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	12 Participants n=5 Participants	12 Participants n=7 Participants	24 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment Argentina	12 participants n=5 Participants	12 participants n=7 Participants	24 participants n=5 Participants
Weight	71 Kilograms STANDARD_DEVIATION 5.7 • n=5 Participants	64.5 Kilograms STANDARD_DEVIATION 7.9 • n=7 Participants	67.6 Kilograms STANDARD_DEVIATION 7.4 • n=5 Participants
Height	174.5 Centimeters STANDARD_DEVIATION 6.6 • n=5 Participants	169.5 Centimeters STANDARD_DEVIATION 9.3 • n=7 Participants	172 Centimeters STANDARD_DEVIATION 8.2 • n=5 Participants
Body Mass Index (BMI)	23.32 Kg/m2 STANDARD_DEVIATION 1.56 • n=5 Participants	22.4 Kg/m2 STANDARD_DEVIATION 1.7 • n=7 Participants	22.8 Kg/m2 STANDARD_DEVIATION 1.6 • n=5 Participants

PRIMARY outcome

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

Population: Per protocol analysis

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")

Outcome measures

Measure	Agalsidase Beta from Biosidus n=11 Participants Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) n=11 Participants Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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
Maximum Serum Concentration of Agalsidase Beta (Cmax)	95.51 mU/ml Standard Deviation 24.9	109.65 mU/ml Standard Deviation 24.32

PRIMARY outcome

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

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")

Outcome measures

Measure	Agalsidase Beta from Biosidus n=11 Participants Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) n=11 Participants Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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
Area Under the Curve of Serum Concentration Between Times 0 and 12 Hours of Agalsidase Beta	418.21 mU*h/ml Standard Deviation 84.81	472.15 mU*h/ml Standard Deviation 100.47

PRIMARY outcome

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

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")

Outcome measures

Measure	Agalsidase Beta from Biosidus n=11 Participants Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) n=11 Participants Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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
Time at Which Maximum Serum Concentration of Agalsidase Beta is Observed (Tmax)	3.83 h Standard Deviation 1.53	3.36 h Standard Deviation 1.21

PRIMARY outcome

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

Population: Per protocol population

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")

Outcome measures

Measure	Agalsidase Beta from Biosidus n=11 Participants Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) n=11 Participants Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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
Area Under the Curve of Serum Concentration, Resulting From the Extrapolation From Time 0 to Infinite Time	419.81 mU*h/ml Standard Deviation 85.1	473.51 mU*h/ml Standard Deviation 100.87

SECONDARY outcome

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

Comparison between enzymatic activity of both formulations of agalsidase beta, assessing the difference between the 5 hour timepoint and the basal determination, to estimate their biological activity as part of the pharmacodynamic analysis. A fluorometric assay will be used. The enzymatic activity will be measured in mU/ml.

The variation of enzymatic activity between the sample taken at the end of the infusion (5 hours) and pre-infusion will be analyzed.

Outcome measures

Measure	Agalsidase Beta from Biosidus n=11 Participants Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) n=11 Participants Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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
Enzymatic Activity on Plasma Samples Measured With Fluorometric Method	98.45 mU/ml Interval 86.63 to 110.27	109.65 mU/ml Interval 97.59 to 121.71

SECONDARY outcome

Timeframe: From infusion to 35 days after

Evaluation of the product safety with analysis of incidence of adverse events and tolerance.

Outcome measures

Measure	Agalsidase Beta from Biosidus n=12 Participants Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) n=12 Participants Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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
Number of Patients With Adverse Events	5 Participants	7 Participants

SECONDARY outcome

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

The presence of neutralizing antibodies to agalsidase beta activity was analyzed in serum samples from 24 volunteers (0h, 12h and 35 days), by biological activity neutralization method.

The percentage of inhibition in the samples was calculated by comparing the activity value of 1 ng of agalsidase beta alone with that of agalsidase treated with the serum samples. Those samples with percentage inhibition >50% were defined as positive.

Outcome measures

Measure	Agalsidase Beta from Biosidus n=12 Participants Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) n=12 Participants Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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
Number of Participants With Positive Results for the Presence of Neutralizing Antibodies Against Agalsidase Beta 0 hours	0 Participants with positive result	0 Participants with positive result
Number of Participants With Positive Results for the Presence of Neutralizing Antibodies Against Agalsidase Beta 12 hours after the end of the infusion	0 Participants with positive result	0 Participants with positive result
Number of Participants With Positive Results for the Presence of Neutralizing Antibodies Against Agalsidase Beta 35 days after the end of the infusion	0 Participants with positive result	0 Participants with positive result

Adverse Events

Agalsidase Beta from Biosidus

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Fabrazyme (Sanofi-Genzyme)

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Agalsidase Beta from Biosidus n=12 participants at risk Participants received a single infusion at a dose 1 mg/kg Agalsidase beta from Biosidus 1 mg/kg: 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	Fabrazyme (Sanofi-Genzyme) n=12 participants at risk Participants received a single infusion at a dose 1 mg/kg Fabrazyme (agalsidase beta) 1 mg/kg: 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
Vascular disorders Increased dyastolic blood pressure	8.3% 1/12 • 35 days	0.00% 0/12 • 35 days
General disorders Headache	8.3% 1/12 • 35 days	33.3% 4/12 • 35 days
Renal and urinary disorders Increased proteinuria	16.7% 2/12 • 35 days	16.7% 2/12 • 35 days
Blood and lymphatic system disorders Increased erythrocyte sedimentation rate	8.3% 1/12 • 35 days	8.3% 1/12 • 35 days
Infections and infestations COVID-19	8.3% 1/12 • 35 days	0.00% 0/12 • 35 days
Vascular disorders Hypotension	8.3% 1/12 • 35 days	0.00% 0/12 • 35 days
Vascular disorders Hypertension	0.00% 0/12 • 35 days	8.3% 1/12 • 35 days
Hepatobiliary disorders Increased GTP	0.00% 0/12 • 35 days	8.3% 1/12 • 35 days

Additional Information

Director of Clinical Trials

Biosidus S.A.U.

Phone: +541149098044

Email: v.berstein@biosidus.com.ar

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place