A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
Study Overview
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.
Study details
The total duration will be up to 6 months
Eligibility Criteria
You may be eligible for this study if you meet the following criteria:
-
Conditions:
Fabry's Disease
-
Age: Between 2 Years - 65 Years
-
Gender: All
Inclusion Criteria:
- 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.
- Cohort 5: ERT-naïve participants. Women of childbearing potential must use a highly effective method of contraception through the study.
Exclusion Criteria:
- 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, 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.
Updated on
27 Mar 2024.
Study ID: NCT06019728
Find a Study Location to Connect
,
You have contacted , on
Your message has been sent to the study team at ,
A copy of the message has been sent to your email
What happens next?
- You can expect the study team to contact you via email or phone in the next few days.
- Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.
You are contacting
Primary Contact
