Trial Outcomes & Findings for A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (NCT NCT06019728)

NCT ID: NCT06019728

Last Updated: 2025-10-21

Results Overview

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).

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 8 participants
• Primary outcome timeframe: Up to 16 weeks
• Results posted on: 2025-10-21

Participant Flow

The study was conducted at 4 centers in United States. A total of 8 participants were screened from 10 November 2023 to 27 August 2024, of which none were screen failures.

A total of 8 participants were enrolled in the study. All participants received same dose in this single-group, single arm study as pre-specified in the protocol and statistical analysis plan (SAP). Note: Reason for not completed = Reason for permanent full study treatment discontinuation.

Participant milestones

Measure	Fabrazyme® 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).
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Fabrazyme® n=8 Participants All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Age, Continuous	40.0 years STANDARD_DEVIATION 17.40 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 16 weeks

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

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).

Outcome measures

Measure	Fabrazyme® n=8 Participants All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions	84.19 percent Interval 80.56 to 86.32

PRIMARY outcome

Timeframe: Up to 16 weeks

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

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.

Outcome measures

Measure	Fabrazyme® n=8 Participants All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Shortest Actual Tolerated Infusion Duration	20.00 minutes Interval 20.0 to 20.0

SECONDARY outcome

Timeframe: Up to 16 weeks

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

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.

Outcome measures

Measure	Fabrazyme® n=8 Participants All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing Any Infusion Associated Reaction	8 Participants

SECONDARY outcome

Timeframe: Up to 16 weeks

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

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.

Outcome measures

Measure	Fabrazyme® n=8 Participants All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing a Second Infusion Associated Reaction	8 Participants

SECONDARY outcome

Timeframe: Up to 16 weeks

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

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.

Outcome measures

Measure	Fabrazyme® n=8 Participants All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing Any Infusion Associated Reaction	8 Participants

SECONDARY outcome

Timeframe: Up to 16 weeks

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

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.

Outcome measures

Measure	Fabrazyme® n=8 Participants All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing a Second Infusion Associated Reaction	8 Participants

SECONDARY outcome

Timeframe: Up to 16 weeks

Population: 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 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.

Outcome measures

Measure	Fabrazyme® n=8 Participants All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction 120 minutes	6 Participants
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction 90 minutes	7 Participants
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction 60 minutes	8 Participants
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction 40 minutes	8 Participants
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction 20 minutes	8 Participants

SECONDARY outcome

Timeframe: Up to 16 weeks

Population: 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 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.

Outcome measures

Measure	Fabrazyme® n=8 Participants All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction 120 minutes	6 Participants
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction 90 minutes	7 Participants
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction 60 minutes	8 Participants
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction 40 minutes	8 Participants
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction 20 minutes	8 Participants

Adverse Events

Fabrazyme®

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Fabrazyme® n=8 participants at risk All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions).
Infections and infestations Influenza	12.5% 1/8 • Number of events 1 • Adverse events and all-cause mortality were collected from the first dose of study treatment (Day 1) up to 16 weeks Analysis was performed on the safety analysis set which included all participants who received any amount of study drug (Fabrazyme®) during study treatment period.
Infections and infestations Nasopharyngitis	12.5% 1/8 • Number of events 1 • Adverse events and all-cause mortality were collected from the first dose of study treatment (Day 1) up to 16 weeks Analysis was performed on the safety analysis set which included all participants who received any amount of study drug (Fabrazyme®) during study treatment period.
Investigations Globotriaosylsphingosine Increased	12.5% 1/8 • Number of events 1 • Adverse events and all-cause mortality were collected from the first dose of study treatment (Day 1) up to 16 weeks Analysis was performed on the safety analysis set which included all participants who received any amount of study drug (Fabrazyme®) during study treatment period.

Additional Information

Trial Transparency Team

Sanofi aventis recherche & développement

Phone: 800-633-1610 ext 6#

Email: Contact-US@sanofi.com

Results disclosure agreements

• Principal investigator is a sponsor employee The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
• Publication restrictions are in place

Restriction type: OTHER