A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
- Conditions
- Fabry's Disease
- Interventions
- Drug: AGALSIDASE BETA (GZ419828)
- Registration Number
- NCT06019728
- Lead Sponsor
- Sanofi
- 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 provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.
- Detailed Description
The total duration will be up to 7 months
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 8
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description agalsidase beta AGALSIDASE BETA (GZ419828) agalsidase beta 1 mg/kg infusion once every other week agalsidase beta Acetaminophen agalsidase beta 1 mg/kg infusion once every other week agalsidase beta Diphenhydramine agalsidase beta 1 mg/kg infusion once every other week agalsidase beta Dexamethasone agalsidase beta 1 mg/kg infusion once every other week agalsidase beta Montelukast agalsidase beta 1 mg/kg infusion once every other week agalsidase beta Loratadine agalsidase beta 1 mg/kg infusion once every other week agalsidase beta Cetirizine agalsidase beta 1 mg/kg infusion once every other week agalsidase beta Fexofenadine agalsidase beta 1 mg/kg infusion once every other week
- Primary Outcome Measures
Name Time Method Reduction of infusion duration from pretrial average of recent 3 infusions Baseline to month 4 Reduction of infusion duration from initial 120 minutes Baseline to month 4 Shortest infusion duration each participant tolerates Baseline to month 4
- Secondary Outcome Measures
Name Time Method Percentage of participants achieving the shortest planned duration of infusion time Baseline to month 4 Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR Baseline to month 4 Number of participants achieving the shortest planned duration of infusion time Baseline to month 4 Number of participants achieving infusion duration without experiencing any IAR Baseline to month 4 Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR Baseline to month 4
Trial Locations
- Locations (5)
UCLA Medical Center Site Number : 1003
🇺🇸Santa Monica, California, United States
Emory University School of Medicine Site Number : 1005
🇺🇸Atlanta, Georgia, United States
Infusion Associates Site Number : 1001
🇺🇸Grand Rapids, Michigan, United States
Metropolitan Hospital Center Site Number : 1004
🇺🇸New York, New York, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 1002
🇺🇸Fairfax, Virginia, United States