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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1287-8570 | Registry Identifier | ICTRP |
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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| agalsidase beta | Experimental | agalsidase beta 1 mg/kg infusion once every other week |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AGALSIDASE BETA (GZ419828) | Drug | Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 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. | Up to 16 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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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.
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.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA Medical Center Site Number : 1003 | Santa Monica | California | 90404 | United States | ||
| Emory University School of Medicine Site Number : 1005 |
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| Label | URL |
|---|---|
| LPS17726 Plain Language Results Summary | View source |
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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
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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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 9, 2024 | Oct 7, 2025 |
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| Acetaminophen | Drug | Tablet or solution; Oral |
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| Diphenhydramine | Drug | Tablet or solution; Oral |
|
| Dexamethasone | Drug | Tablet or solution; Oral |
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| Montelukast | Drug | Tablet or chewable tablet or oral granules; Oral |
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| Loratadine | Drug | Tablet or chewable tablet; Oral |
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| Cetirizine | Drug | Tablet or oral solution; Oral |
|
| Fexofenadine | Drug | Tablet or oral suspension; Oral |
|
| 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. | 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. | 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. | 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. | 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. | Up to 16 weeks |
| Atlanta |
| Georgia |
| 30322-1007 |
| United States |
| Infusion Associates Site Number : 1001 | Grand Rapids | Michigan | 49525 | United States |
| Metropolitan Hospital Center Site Number : 1004 | New York | New York | 10021 | United States |
| Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 1002 | Fairfax | Virginia | 22030 | United States |
| COMPLETED |
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| NOT COMPLETED |
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The Intent-to-treat (ITT) analysis set included all participants who were enrolled and allocated to study treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Fabrazyme® | All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions). |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | The ITT analysis set included all participants who were enrolled and allocated to study treatment. | Posted | Median | Inter-Quartile Range | percent | Up to 16 weeks |
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| |||||||||||||||||||||||||
| Primary | 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. | The ITT analysis set included all participants who were enrolled and allocated to study treatment. | Posted | Median | Inter-Quartile Range | minutes | Up to 16 weeks |
|
| ||||||||||||||||||||||||||
| Secondary | 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. | The ITT analysis set included all participants who were enrolled and allocated to study treatment. | Posted | Count of Participants | Participants | Up to 16 weeks |
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| Secondary | 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. | The ITT analysis set included all participants who were enrolled and allocated to study treatment. | Posted | Count of Participants | Participants | Up to 16 weeks |
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| Secondary | 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. | The ITT analysis set included all participants who were enrolled and allocated to study treatment. | Posted | Count of Participants | Participants | Up to 16 weeks |
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| Secondary | 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. | The ITT analysis set included all participants who were enrolled and allocated to study treatment. | Posted | Count of Participants | Participants | Up to 16 weeks |
|
| |||||||||||||||||||||||||||
| Secondary | 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. | 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. | Posted | Count of Participants | Participants | Up to 16 weeks |
|
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| Secondary | 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. | 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. | Posted | Count of Participants | Participants | Up to 16 weeks |
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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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Fabrazyme® | All participants received Fabrazyme® 1 mg/kg Q2W via IV infusion for up to 16 weeks (up to 9 infusions). | 0 | 8 | 0 | 8 | 2 | 8 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Influenza | Infections and infestations | MedDra 27.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDra 27.1 | Systematic Assessment |
| |
| Globotriaosylsphingosine Increased | Investigations | MedDra 27.1 | Systematic Assessment |
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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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi aventis recherche & développement | 800-633-1610 ext 6# | Contact-US@sanofi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 22, 2024 | Oct 7, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
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| ID | Term |
|---|---|
| C459420 | agalsidase beta |
| D000082 | Acetaminophen |
| D004155 | Diphenhydramine |
| D003907 | Dexamethasone |
| C093875 | montelukast |
| D017336 | Loratadine |
| D017332 | Cetirizine |
| C093230 | fexofenadine |
| ID | Term |
|---|---|
| D000083 | Acetanilides |
| D000813 | Anilides |
| D000577 | Amides |
| D009930 | Organic Chemicals |
| D000814 | Aniline Compounds |
| D000588 | Amines |
| D005021 | Ethylamines |
| D001559 | Benzhydryl Compounds |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |
| D003533 | Cyproheptadine |
| D003986 | Dibenzocycloheptenes |
| D001567 | Benzocycloheptenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D010880 | Piperidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D006919 | Hydroxyzine |
| D010879 | Piperazines |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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