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.

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

Fabry's Disease

The total duration will be up to 7 months

AGALSIDASE BETA (GZ419828)

Acetaminophen

Diphenhydramine

Dexamethasone

Montelukast

Loratadine

Cetirizine

Fexofenadine

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

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).

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.

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.

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.

Number of participants who tolerated 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. Participants with an initial planned target duration <90 minutes were considered a responder if they tolerated at least 1 infusion during the study treatment period with actual duration <90 minutes.

Number of participants who tolerated 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. Participants with an initial planned target duration <90 minutes were considered a responder if they tolerated at least 1 infusion during the study treatment period with actual duration <90 minutes.

Number of participants who tolerated each planned infusion duration 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. The planned duration of infusion time was 120 minutes, 90 minutes, 60 minutes, 40 minutes, and 20 minutes.

Number of participants who tolerated each planned infusion duration 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. The planned duration of infusion time was 120 minutes, 90 minutes, 60 minutes, 40 minutes, and 20 minutes.

agalsidase beta 1 mg/kg infusion once every other week

UCLA Medical Center Site Number : 1003

Emory University School of Medicine Site Number : 1005

Infusion Associates Site Number : 1001

Metropolitan Hospital Center Site Number : 1004

Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 1002

Analysis was performed on the safety analysis set which included all participants who received any amount of study drug (Fabrazyme®) during study treatment period.

All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).

Fabrazyme®

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

The Intent-to-treat (ITT) analysis set included all participants who were enrolled and allocated to study treatment.

Trial Transparency Team

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).

The ITT analysis set included all participants who were enrolled and allocated to study treatment.

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

Shortest Actual Tolerated Infusion Duration

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 a Second Infusion Associated Reaction

Number of participants who tolerated 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. Participants with an initial planned target duration \<90 minutes were considered a responder if they tolerated at least 1 infusion during the study treatment period with actual duration \<90 minutes.

Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing Any Infusion Associated Reaction

Number of participants who tolerated 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. Participants with an initial planned target duration \<90 minutes were considered a responder if they tolerated at least 1 infusion during the study treatment period with actual duration \<90 minutes.

Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing a Second Infusion Associated Reaction

120 minutes

90 minutes

60 minutes

40 minutes

20 minutes

The ITT analysis set included all participants who were enrolled and allocated to study treatment. Only those participants with data collected at specified timepoints are reported.

Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction

Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction

All participants received Fabrazyme® 1 milligram per kilogram (mg/kg) every 2 weeks (Q2W) via intravenous (IV) infusion for up to 16 weeks (up to 9 infusions).

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

About this trial

Study ID

Status

Type

Phase

Placebo

Accepting

Trial Timing

What is this trial about?

What are the participation requirements?