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

NCT06019728 | Completed Phase 4 Results FDA Drug

• 2 other identifiers: LPS17726U1111-1287-8570
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Fabry's Disease

Outcome Measures

Primary Outcomes (2)

• 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

Secondary Outcomes (6)

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

  Up to 16 weeks

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

  Up to 16 weeks

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

  Up to 16 weeks

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

  Up to 16 weeks

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

  Up to 16 weeks

Study Arms (1)

agalsidase beta

EXPERIMENTAL

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

Interventions

AGALSIDASE BETA (GZ419828) DRUG

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

Also known as: Fabrazyme

agalsidase beta

Acetaminophen DRUG

Tablet or solution; Oral

agalsidase beta

Diphenhydramine DRUG

Tablet or solution; Oral

agalsidase beta

Dexamethasone DRUG

Tablet or solution; Oral

agalsidase beta

Montelukast DRUG

Tablet or chewable tablet or oral granules; Oral

agalsidase beta

Loratadine DRUG

Tablet or chewable tablet; Oral

agalsidase beta

Cetirizine DRUG

Tablet or oral solution; Oral

agalsidase beta

Fexofenadine DRUG

Tablet or oral suspension; Oral

agalsidase beta

Eligibility Criteria

• Age: 2 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \- 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (5)

UCLA Medical Center Site Number : 1003

Santa Monica, California, 90404, United States

Location

Emory University School of Medicine Site Number : 1005

Atlanta, Georgia, 30322-1007, United States

Location

Infusion Associates Site Number : 1001

Grand Rapids, Michigan, 49525, United States

Location

Metropolitan Hospital Center Site Number : 1004

New York, New York, 10021, United States

Location

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

Fairfax, Virginia, 22030, United States

Location

Related Links

• LPS17726 Plain Language Results Summary

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase beta; Acetaminophen; Diphenhydramine; Dexamethasone; montelukast; Loratadine; Cetirizine; fexofenadine

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Acetanilides; Anilides; Amides; Organic Chemicals; Aniline Compounds; Amines; Ethylamines; Benzhydryl Compounds; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated; Cyproheptadine; Dibenzocycloheptenes; Benzocycloheptenes; Polycyclic Aromatic Hydrocarbons; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Hydroxyzine; Piperazines

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi aventis recherche & développement

Contact-US@sanofi.com

800-633-1610 ext 6#

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 25, 2023
• First Posted: August 31, 2023
• Study Start: November 10, 2023
• Primary Completion: October 25, 2024
• Study Completion: October 25, 2024
• Last Updated: October 21, 2025
• Results First Posted: October 21, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

US (5)