Fabry DiseasePhase 1NCT05343715
PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion
A Phase 1 study testing Agalsidase beta from Biosidus 1 mg/kg and Fabrazyme (agalsidase beta) 1 mg/kg in people with Fabry Disease, run by Bio Sidus SA at 1 site in Argentina.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 40 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Agalsidase beta from Biosidus 1 mg/kg and Fabrazyme (agalsidase beta) 1 mg/kg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteArgentina
- Study dates
- 23 Oct 2021to 17 Apr 2022
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 40 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- Male subjects between the ages of 18 and 40
- Body mass index (BMI) between 19 and 25 kg/m².
- Volunteers whose complementary exams (ECG, chest X-ray, blood and urine, PCR for COVID-19), performed prior to their inclusion are within the normal range and/or are not clinically significant according to the investigator's judgement.
- 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
Who cannot join
- 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
Show all 29Show fewer
- 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
- Evidence of active gastroduodenal disease
- History of peripheral thrombotic phenomena
- Underlying neurological disease
- Presence of a current progressive chronic disease
- History of drug or alcohol abuse or addiction within the last three years
- Participation in a clinical study within the last three months
- Use of any drug within the fourteen days prior to the start of the study that, according to the principal investigator's judgement may interfere with the biodistribution of the medicinal product.
- Subjects who have donated or suffered from blood loss within the twelve weeks prior to the start of the study, or intends to donate blood within the following three months after the completion of the study.
- Excessive drinker of tea, cocoa, mate, coffee and/or caffeinated drinks (>5 cups/day) or of wine (>0.5 L/day) or alcohol (>50 ml/day)
- Significant abnormalities in the electrocardiogram
- Positive PCR test for COVID-19
- Positive serology for HIV, hepatitis B or hepatitis C
- Abnormal clinical laboratory test results (that according to the principal investigator's judgement are considered clinically significant)
- Uncooperative volunteers
Trial locations and contacts
1 site in 1 country.
Argentina1 site
Sanatorio Nuestra Señora del Pilar
Ciudadela, Buenos Aires, 1702
About this study
Official title: Comparative Pharmacokinetic and Pharmacodynamic Study Between 2 Agalsidase Beta Formulations at a Single Dose of 1 mg/kg of Agalsidase (Biosidus) and Fabrazyme (Sanofi Genzyme) as Intravenous Infusion in Male Healthy Volunteers
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
Read the detailed description
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.
- Conditions
- Fabry Disease
- Being tested
- Agalsidase beta from Biosidus 1 mg/kg, Fabrazyme (agalsidase beta) 1 mg/kg
- Keywords
- Fabry Disease, agalsidase beta, phase 1
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Agalsidase beta from Biosidus 1 mg/kgBiological
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 (agalsidase beta) 1 mg/kgBiological
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
| Study group | Role | What they receive |
|---|---|---|
| Agalsidase Beta from Biosidus | Experimental | Participants received a single infusion at a dose 1 mg/kg Receives: Biological: Agalsidase beta from Biosidus 1 mg/kg |
| Fabrazyme (Sanofi-Genzyme) | Active comparator | Participants received a single infusion at a dose 1 mg/kg Receives: Biological: Fabrazyme (agalsidase beta) 1 mg/kg |
What is being measured
The primary outcome is the main question the study is designed to answer.
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")
Measured: 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")
Measured: 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")
Measured: 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")
Measured: 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
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.
Measured: 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.
Measured: 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.
Measured: 0 hours,12 hours and 35 days after the end of the infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2022
- Study start23 Oct 2021
- Primary completion6 Mar 2022
- Study completion17 Apr 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bio Sidus SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Eduardo Pirotzky, MD, Principal investigator, Sanatorio Nuestra Señora del Pilar
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05343715
- Sponsor's protocol ID
- AGA Biosidus
- Status last verified
- Oct 2025
- First submitted
- 24 Jan 2022
- Last updated
- 15 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparative Pharmacokinetic and Pharmacodynamic Study Between 2 Agalsidase Beta Formulations at a Single Dose of 1 mg/kg of Agalsidase (Biosidus) and Fabrazyme (Sanofi Genzyme) as Intravenous Infusion in Male Healthy Volunteers
- ClinicalTrials.gov ID
- NCT05343715
- Acronym / sponsor ID
- AGA Biosidus
- Conditions
- Fabry Disease
- Keywords
- Fabry Disease, agalsidase beta, phase 1
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Bio Sidus SAIndustry (pharma, biotech, devices)
- Study start
- 23 Oct 2021 (actual)
- Primary completion
- 6 Mar 2022 (actual)
- Study completion
- 17 Apr 2022 (actual)
- First posted
- 25 Apr 2022 (actual)
- Results
- Posted 15 Mar 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Agalsidase Beta from Biosidus | Experimental | Biological: Agalsidase beta from Biosidus 1 mg/kgParticipants received a single infusion at a dose 1 mg/kg |
| Fabrazyme (Sanofi-Genzyme) | Active comparator | Biological: Fabrazyme (agalsidase beta) 1 mg/kgParticipants received a single infusion at a dose 1 mg/kg |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Male subjects between the ages of 18 and 40
- Body mass index (BMI) between 19 and 25 kg/m².
- Volunteers whose complementary exams (ECG, chest X-ray, blood and urine, PCR for COVID-19), performed prior to their inclusion are within the normal range and/or are not clinically significant according to the investigator's judgement.
- 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
Key exclusion criteria (29 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Agalsidase beta from Biosidus 1 mg/kgPatients 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 | Biological | Agalsidase Beta from Biosidus |
| Fabrazyme (agalsidase beta) 1 mg/kgPatients received an infusion of 1 mg/kg of Fabrazyme. Final infusion dose is achieved with lyophilized drug at concentration 35 mg and 5 mg | Biological | Fabrazyme (Sanofi-Genzyme) |
Primary outcome
Maximum Serum Concentration of Agalsidase Beta (Cmax)
Time frame: 0, 1, 3, 5, 5.5, 6, 7, 8, 10 and 12 hours from the beginning of the infusion
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")
Area Under the Curve of Serum Concentration Between Times 0 and 12 Hours of Agalsidase Beta
Time frame: 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")
Time at Which Maximum Serum Concentration of Agalsidase Beta is Observed (Tmax)
Time frame: 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")
Area Under the Curve of Serum Concentration, Resulting From the Extrapolation From Time 0 to Infinite Time
Time frame: 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 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")
3 secondary outcomes
Enzymatic Activity on Plasma Samples Measured With Fluorometric Method
Time frame: Pre-infusion and 5 hours post administration (end of infusion)
Number of Patients With Adverse Events
Time frame: From infusion to 35 days after
Number of Participants With Positive Results for the Presence of Neutralizing Antibodies Against Agalsidase Beta
Time frame: 0 hours,12 hours and 35 days after the end of the infusion.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Eduardo Pirotzky, MD principal investigator
Sanatorio Nuestra Señora del Pilar
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Agalsidase beta from Biosidus 1 mg/kg and Fabrazyme (agalsidase beta) 1 mg/kg in people with Fabry Disease, run by Bio Sidus SA at 1 site in Argentina.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Argentina. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Serum Concentration of Agalsidase Beta (Cmax) (time frame: 0, 1, 3, 5, 5.5, 6, 7, 8, 10 and 12 hours from the beginning of the infusion). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2025.