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

NCT ID: NCT05343715

Last Updated: 2025-10-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-10-23
• Study Completion Date: 2022-04-17

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

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: SINGLE

Study Groups

Agalsidase Beta from Biosidus

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

Group Type: EXPERIMENTAL

Agalsidase beta from Biosidus 1 mg/kg

Intervention Type: 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

Fabrazyme (Sanofi-Genzyme)

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

Group Type: ACTIVE_COMPARATOR

Fabrazyme (agalsidase beta) 1 mg/kg

Intervention Type: 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

Interventions

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

Intervention Type: BIOLOGICAL

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

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. Male subjects between the ages of 18 and 40

2. Body mass index (BMI) between 19 and 25 kg/m².

3. 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.

4. 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)

5. Volunteers who are well disposed to the study and have signed the approved informed consent prior to the start of the study

Exclusion Criteria

1. History of clinically significant allergies (except for untreated asymptomatic seasonal allergies)

2. 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

3. Volunteers who are receiving other drugs (prescription or over-the-counter) or who have within the 2 weeks prior to the study

4. Volunteers with history of autoimmune diseases.

5. Chronic disorders of the CNS, psychological and/or psychiatric disorders: bipolar disorder, severe depression, insomnia, changes in personality

6. Active or chronic infections

7. Having received live or inactivated viral or bacterial vaccines, within the fifteen days prior to visits 1 or 2

8. 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.

9. Known allergies to any of the components of the formulations

10. Active smoker, of more than 10 cigarettes/day

11. Current clinical evidence of severe digestive disorders, surgeries of the gastrointestinal tract (except for appendectomy)

12. Current clinical evidence of kidney disease

13. Current clinical evidence of liver disorders

14. Current clinical evidence of respiratory and cardiac disease

15. Presence of diabetes mellitus, thyroid dysfunction or other endocrine disorders

16. Evidence of active gastroduodenal disease

17. History of peripheral thrombotic phenomena

18. Underlying neurological disease

19. Presence of a current progressive chronic disease

20. History of drug or alcohol abuse or addiction within the last three years

21. Participation in a clinical study within the last three months

22. 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.

23. 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.

24. 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)

25. Significant abnormalities in the electrocardiogram

26. Positive PCR test for COVID-19

27. Positive serology for HIV, hepatitis B or hepatitis C

28. Abnormal clinical laboratory test results (that according to the principal investigator's judgement are considered clinically significant)

29. Uncooperative volunteers

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 40 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: Yes

Sponsors

Bio Sidus SA

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Eduardo Pirotzky, MD

Role: PRINCIPAL_INVESTIGATOR

Sanatorio Nuestra Señora del Pilar

Locations

Sanatorio Nuestra Señora del Pilar

Ciudadela, Buenos Aires, Argentina

Countries

Argentina

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

AGA Biosidus

Identifier Type: -

Identifier Source: org_study_id