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

Study on Safe Infusion Rates for Fabry Disease Treatment

Recruiting
2 years - 65 years
All
Phase 4
18 participants needed
3 Locations

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: 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 06 Mar 2024. Study ID: NCT06019728

This study investigates the safety and tolerability of an investigational medication for Fabry Disease (FD). Fabry Disease is a genetic disorder that affects the body's ability to break down a specific type of fat, leading to a buildup in the body's cells. The purpose of this study is to see if the infusion rate of the investigational medication can be safely increased while reducing the infusion volume.

Participants will receive the investigational medication through an infusion. The study will involve different groups, called study arms, based on participants' characteristics and previous treatment experience. Participants will be monitored for any reactions to the infusion to ensure safety.

  • Who can participate: Participants aged 2 to 65 years with a confirmed diagnosis of Fabry Disease can join. They must weigh at least 30 kg and have been treated with Fabrazyme for at least 3 months without reactions, except for those who have not received enzyme replacement therapy before.
  • Study details: Participants will receive infusions of Fabrazyme. The study will explore different rates and volumes of infusion to assess safety and tolerability without using a placebo.
  • Study Timelines: The study will last up to 6 months.

Find a study site near you

We have submitted the contact information you provided to the research team at {{SITE_NAME}}. A copy of the message has been sent to your email for your records.
Would you like to be notified about other trials? Sign up for Patient Notification Services.
Sign up

Send a message

Enter your contact details to connect with study team

Investigator Avatar

Primary Contact

First name*
Last name*
Email*
Phone number*
Other language