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

Fabry's Disease Clinical Trial

— SHORTEN  

Official title:

A Study to Investigate Safety and Tolerability of Higher Infusion Rate to shORten the duraTion of FabrazymE Infusion

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06019728
• Other study ID #: LPS17726
• Secondary ID: U1111-1287-8570
• Status: Recruiting
• Phase: Phase 4
• Start date: November 10, 2023
• Est. completion date: October 6, 2025

Study information

• Verified date: March 2024
• Source: Sanofi
• Contact: Trial Transparency email recommended (Toll free for US & Canada)
• Phone: 800-633-1610
• Email: Contact-US[@]sanofi.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

The total duration will be up to 6 months

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 18
• Est. completion date: October 6, 2025
• Est. primary completion date: October 6, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Fabry Disease
• Fabry's Disease

Intervention

Drug:
• AGALSIDASE BETA (GZ419828)
Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion
• Acetaminophen
Tablet or solution; Oral
• Diphenhydramine
Tablet or solution; Oral
• Dexamethasone
Tablet or solution; Oral
• Montelukast
Tablet or chewable tablet or oral granules; Oral

Locations

Country	Name	City	State
United States	Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 1002	Fairfax	Virginia
United States	Infusion Associates Site Number : 1001	Grand Rapids	Michigan
United States	Metropolitan Hospital Center Site Number : 1004	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Sanofi

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Reduction of infusion duration from pretrial average of recent 3 infusions		Baseline to month 4
Primary	Reduction of infusion duration from initial 120 minutes		Baseline to month 4
Primary	Shortest infusion duration each participant tolerates		Baseline to month 4
Secondary	Number of participants achieving the shortest planned duration of infusion time		Baseline to month 4
Secondary	Percentage of participants achieving the shortest planned duration of infusion time		Baseline to month 4
Secondary	Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR		Baseline to month 4
Secondary	Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR		Baseline to month 4
Secondary	Number of participants achieving infusion duration without experiencing any IAR		Baseline to month 4