Status:
COMPLETED
A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
Lead Sponsor:
Sanofi
Conditions:
Fabry's Disease
Eligibility:
All Genders
2-65 years
Phase:
PHASE4
Brief Summary
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to ...
Detailed Description
The total duration will be up to 7 months
Eligibility Criteria
Inclusion
- \- Participants with confirmed diagnosis of FD who are ≥2 and ≤65 years of age at the time of signing the informed consent form (ICF) or assent, if applicable.
- Cohort 1: female participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 2: non-classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 3: classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 4: participants with body weight \<30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Women of childbearing potential must use a highly effective method of contraception through the study.
Exclusion
- Female participants who are pregnant or breastfeeding.
- History of significant allergic disease or hypersensitivity to Fabrazyme or other medicinal products.
- Contraindication to Fabrazyme or any of the premedications or rescue medications (diphenhydramine, loratadine, cetirizine, fexofenadine, acetaminophen, montelukast, dexamethasone).
- Any other medical condition considered to make the increased infusion rate not tolerable at the Investigator's discretion.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Key Trial Info
Start Date :
November 10 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 25 2024
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT06019728
Start Date
November 10 2023
End Date
October 25 2024
Last Update
October 21 2025
Active Locations (5)
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1
UCLA Medical Center Site Number : 1003
Santa Monica, California, United States, 90404
2
Emory University School of Medicine Site Number : 1005
Atlanta, Georgia, United States, 30322-1007
3
Infusion Associates Site Number : 1001
Grand Rapids, Michigan, United States, 49525
4
Metropolitan Hospital Center Site Number : 1004
New York, New York, United States, 10021