A Phase 4 interventional study of AGALSIDASE BETA (GZ419828) and Acetaminophen in Fabry's Disease, sponsored by Sanofi. Completed at 5 sites in United States. Open to participants aged 2 Years to 65 Years. Per ClinicalTrials.gov, last updated 2025-10-21.
Sponsored by Sanofi · Phase 4, Interventional, and Treatment
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
242 studies on the registry are indexed under Fabry Disease; 54 are open to participants now.
This study's enrollment of 8 is below the median of 22 across 105 interventional studies indexed under Fabry Disease.
Browse Fabry Disease studies →Sanofi is the lead sponsor of 1,508 studies on the registry; 90 are open to participants now.
Of its 198 completed or terminated interventional studies of FDA-regulated products, 118 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
- 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.
Women of childbearing potential must use a highly effective method of contraception through the study.
Exclusion Criteria:
The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
agalsidase beta 1 mg/kg infusion once every other week
Drug: AGALSIDASE BETA (GZ419828) · Drug: Acetaminophen · Drug: Diphenhydramine · Drug: Dexamethasone · Drug: Montelukast · Drug: Loratadine · Drug: Cetirizine · Drug: Fexofenadine
Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion
Also known as: Fabrazyme
Tablet or solution; Oral
Tablet or solution; Oral
Tablet or solution; Oral
Tablet or chewable tablet or oral granules; Oral
Tablet or chewable tablet; Oral
Tablet or oral solution; Oral
Tablet or oral suspension; Oral
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
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.
Time frame: Up to 16 weeks
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 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.
Time frame: Up to 16 weeks
Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing a Second 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.
Time frame: Up to 16 weeks
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 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.
Time frame: Up to 16 weeks
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction
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.
Time frame: Up to 16 weeks
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.
| Milestone | Fabrazyme® |
|---|---|
| Started | 8 |
| Completed | 8 |
| Not completed | 0 |
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).
| percent | Fabrazyme® |
|---|---|
| Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions | 84.19 (80.56 to 86.32) |
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.
| minutes | Fabrazyme® |
|---|---|
| Shortest Actual Tolerated Infusion Duration | 20.00 (20.00 to 20.00) |
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.
| Participants | Fabrazyme® |
|---|---|
| Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing Any Infusion Associated Reaction | 8 |
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.
| Participants | Fabrazyme® |
|---|---|
| Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing a Second Infusion Associated Reaction | 8 |
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.
| Participants | Fabrazyme® |
|---|---|
| Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing Any Infusion Associated Reaction | 8 |
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.
| Participants | Fabrazyme® |
|---|---|
| Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing a Second Infusion Associated Reaction | 8 |
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.
| Participants | Fabrazyme® |
|---|---|
| 120 minutes | 6 |
| 90 minutes | 7 |
| 60 minutes | 8 |
| 40 minutes | 8 |
| 20 minutes | 8 |
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.
| Participants | Fabrazyme® |
|---|---|
| 120 minutes | 6 |
| 90 minutes | 7 |
| 60 minutes | 8 |
| 40 minutes | 8 |
| 20 minutes | 8 |
Collected over Adverse events and all-cause mortality were collected from the first dose of study treatment (Day 1) up to 16 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Fabrazyme® | 0/8 (0%) | 0/8 (0%) | 2/8 (25%) |
| Event | Fabrazyme® |
|---|---|
| InfluenzaInfections and infestations | 1/8 |
| NasopharyngitisInfections and infestations | 1/8 |
| Globotriaosylsphingosine IncreasedInvestigations | 1/8 |
The Intent-to-treat (ITT) analysis set included all participants who were enrolled and allocated to study treatment.
| Age, Continuous(years) | Fabrazyme® |
|---|---|
| Mean | 40.0 ± 17.40 |
| Sex: Female, Male(Participants) | Fabrazyme® |
|---|---|
| Female | 3 |
| Male | 5 |
| Race (NIH/OMB)(Participants) | Fabrazyme® |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 6 |
| More than one race | 0 |
| Unknown or Not Reported | 2 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
This study is completed, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Sanofi