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

Phase 4

Recruiting

• Conditions: Fabry's Disease
• Interventions: Drug: AGALSIDASE BETA (GZ419828); Drug: Acetaminophen; Drug: Diphenhydramine; Drug: Dexamethasone; Drug: Montelukast

• 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 6 months

Basic Information
|
Recruitment & Eligibility
|
Study & Design
|
Trial Locations

Study Timeline

• Study First Submitted: 2023-08-25
• Study First Submitted QC: 2023-08-30
• Study First Posted: 2023-08-31
• Study Start: 2023-11-10
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-08
• Last Update Posted: 2024-08-09
• Primary Completion: 2025-10-06
• Study Completion: 2025-10-06

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 18

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.

Read More

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.

Read More

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

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)

Emory University School of Medicine Site Number : 1005; 🇺🇸 Atlanta, Georgia, 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, United States

UCLA Medical Center Site Number : 1003; 🇺🇸 Santa Monica, California, United States