A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

Completed · Phase 4

Conditions studied: Fabry's Disease

In brief

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.

Key facts

Study ID

NCT06019728

Run by

Sanofi

People needed

8

Starts

2023-11-10

Expected to finish

2024-10-25

Last updated by the study team

2025-10-21

Who can join

Age: 2 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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