A Phase 1 interventional study of Agalsidase beta from Biosidus 1 mg/kg and Fabrazyme (agalsidase beta) 1 mg/kg in Fabry Disease, sponsored by Bio Sidus SA. Completed at 1 site in Argentina. Open to male participants aged 18 Years to 40 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-10-15.
Sponsored by Bio Sidus SA · Phase 1, Interventional, and Treatment
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
Volunteers will receive either one or the other agalsidase beta formulation. Drug will be administered as an intravenous 5h infusion. Plasma samples will be taken at different time points, from before infusion up to 12h later. Pharmacokinetic (PK) and pharmacodynamic (PD) profile will be determined, and bioequivalence evaluated.
244 studies on the registry are indexed under Fabry Disease; 55 are open to participants now.
This study's enrollment of 24 is close to the median of 22 across 108 interventional studies indexed under Fabry Disease.
Browse Fabry Disease studies →Bio Sidus SA is the lead sponsor of 6 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants received a single infusion at a dose 1 mg/kg
Biological: Agalsidase beta from Biosidus 1 mg/kg
Participants received a single infusion at a dose 1 mg/kg
Biological: Fabrazyme (agalsidase beta) 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
Drug page: Agalsidase beta
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)
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")
Time frame: 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")
Time frame: 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")
Time frame: 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")
Time frame: 0, 1, 3, 5, 5.5, 6, 7, 8, 10 and 12 hours from the beginning of the infusion
Enzymatic Activity on Plasma Samples Measured With Fluorometric Method
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.
Time frame: Pre-infusion and 5 hours post administration (end of infusion)
Number of Patients With Adverse Events
Evaluation of the product safety with analysis of incidence of adverse events and tolerance.
Time frame: From infusion to 35 days after
Number of Participants With Positive Results for the Presence of Neutralizing Antibodies Against Agalsidase Beta
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.
Time frame: 0 hours,12 hours and 35 days after the end of the infusion.
| Milestone | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) |
|---|---|---|
| Started | 12 | 12 |
| Completed | 12 | 12 |
| Not completed | 0 | 0 |
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")
| mU/ml | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) |
|---|---|---|
| Maximum Serum Concentration of Agalsidase Beta (Cmax) | 95.51 ± 24.9 | 109.65 ± 24.32 |
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")
| mU*h/ml | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) |
|---|---|---|
| Area Under the Curve of Serum Concentration Between Times 0 and 12 Hours of Agalsidase Beta | 418.21 ± 84.81 | 472.15 ± 100.47 |
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")
| h | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) |
|---|---|---|
| Time at Which Maximum Serum Concentration of Agalsidase Beta is Observed (Tmax) | 3.83 ± 1.53 | 3.36 ± 1.21 |
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")
| mU*h/ml | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) |
|---|---|---|
| Area Under the Curve of Serum Concentration, Resulting From the Extrapolation From Time 0 to Infinite Time | 419.81 ± 85.1 | 473.51 ± 100.87 |
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.
| mU/ml | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) |
|---|---|---|
| Enzymatic Activity on Plasma Samples Measured With Fluorometric Method | 98.45 (86.63 to 110.27) | 109.65 (97.59 to 121.71) |
Evaluation of the product safety with analysis of incidence of adverse events and tolerance.
| Participants | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) |
|---|---|---|
| Number of Patients With Adverse Events | 5 | 7 |
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.
| Participants with positive result | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) |
|---|---|---|
| 0 hours | 0 | 0 |
| 12 hours after the end of the infusion | 0 | 0 |
| 35 days after the end of the infusion | 0 | 0 |
Collected over 35 days. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Agalsidase Beta from Biosidus | 0/12 (0%) | 0/12 (0%) | 7/12 (58.3%) |
| Fabrazyme (Sanofi-Genzyme) | 0/12 (0%) | 0/12 (0%) | 9/12 (75%) |
| Event | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) |
|---|---|---|
| HeadacheGeneral disorders | 1/12 | 4/12 |
| Increased proteinuriaRenal and urinary disorders | 2/12 | 2/12 |
| Increased dyastolic blood pressureVascular disorders | 1/12 | 0/12 |
| Increased erythrocyte sedimentation rateBlood and lymphatic system disorders | 1/12 | 1/12 |
| COVID-19Infections and infestations | 1/12 | 0/12 |
| HypotensionVascular disorders | 1/12 | 0/12 |
| HypertensionVascular disorders | 0/12 | 1/12 |
| Increased GTPHepatobiliary disorders | 0/12 | 1/12 |
Healthy volunteers who have no detectable enzyme activity in circulating blood.
| Age, Categorical(Participants) | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 12 | 12 | 24 |
| >=65 years | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 12 | 12 | 24 |
| Ethnicity (NIH/OMB)(Participants) | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) | Total |
|---|---|---|---|
| Hispanic or Latino | 12 | 12 | 24 |
| Not Hispanic or Latino | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(participants) | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) | Total |
|---|---|---|---|
| Argentina | 12 | 12 | 24 |
| Weight(Kilograms) | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) | Total |
|---|---|---|---|
| Mean | 71 ± 5.7 | 64.5 ± 7.9 | 67.6 ± 7.4 |
| Height(Centimeters) | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) | Total |
|---|---|---|---|
| Mean | 174.5 ± 6.6 | 169.5 ± 9.3 | 172 ± 8.2 |
| Body Mass Index (BMI)(Kg/m2) | Agalsidase Beta from Biosidus | Fabrazyme (Sanofi-Genzyme) | Total |
|---|---|---|---|
| Mean | 23.32 ± 1.56 | 22.4 ± 1.7 | 22.8 ± 1.6 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Oct 2021 to primary completion in Mar 2022, 4 months.
Results were first posted in Mar 2024, 2 years after primary completion.
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