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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06019728
Other study ID # LPS17726
Secondary ID U1111-1287-8570
Status Recruiting
Phase Phase 4
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
See also
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Completed NCT02450604 - Prevalence of Fabry's Disease in a Population of Patients With Chronic Pain N/A
Recruiting NCT04847713 - Early Detection and Follow-Up of Patients With Fabry's Disease