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

Phase 4CompletedNCT06019728

Sanofi8 enrolled

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.

The total duration will be up to 7 months

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Fabry's Disease

Interventions

AGALSIDASE BETA (GZ419828)DRUG

Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion

AcetaminophenDRUG

Tablet or solution; Oral

DiphenhydramineDRUG

Tablet or solution; Oral

Eligibility

Sex: ALLMin age: 2 YearsMax age: 65 Years

Medical Language ↔ Plain English

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. 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, loratadine, cetirizine, fexofenadine, 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.

Locations (5)

UCLA Medical Center Site Number : 1003

Santa Monica, California, United States

Emory University School of Medicine Site Number : 1005

Atlanta, Georgia, United States

Infusion Associates Site Number : 1001

Grand Rapids, Michigan, United States

Metropolitan Hospital Center Site Number : 1004

New York, New York, United States

Outcomes

Primary Outcomes

Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions

Percent reduction was with respect to the shortest tolerated infusion for the participant. The shortest infusion duration tolerated was the actual shortest duration (highest infusion rate) of a completed infusion tolerated by the participant without experiencing a second infusion associated reaction (IAR). The pre-study infusion duration was derived as the average of the duration of 3 most recent infusions the participant received prior to participating to this study. Percent reduction of infusion duration was calculated as: 100 x (average of pre-study infusion duration \[minutes\] - actual shortest infusion duration tolerated \[minutes\]) / average of pre-study infusion duration (minutes).

Time frame: Up to 16 weeks

Shortest Actual Tolerated Infusion Duration

The shortest infusion duration tolerated was the actual shortest duration (highest infusion rate) of a completed infusion tolerated by the participant without experiencing a second IAR. IARs were events recorded as adverse event of special interest (AESI) and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun.

Time frame: Up to 16 weeks

Secondary Outcomes

Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing Any Infusion Associated Reaction

Number of participants who achieved the shortest planned duration of infusion time without experiencing any IAR is reported. ARs were events recorded as AESI and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun.

Time frame: Up to 16 weeks

Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing a Second Infusion Associated Reaction

Number of participants who achieved the shortest planned duration of infusion time without experiencing a second IAR is reported. ARs were events recorded as AESI and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun.

Data from ClinicalTrials.gov

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