Skip to main content
OpenTrials
Completed

NCT Number: NCT06019728

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

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

Age range

2 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

UCLA Medical Center Site Number : 1003, Santa Monica, California, United States

Loading trial locations.

About this study

The total duration will be up to 7 months

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.
  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

AGALSIDASE BETA (GZ419828)

Drug

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

Other names: Fabrazyme

Acetaminophen

Drug

Tablet or solution; Oral

diphenhydramine

Drug

Tablet or solution; Oral

Dexamethasone

Drug

Tablet or solution; Oral

Montelukast

Drug

Tablet or chewable tablet or oral granules; Oral

Loratadine

Drug

Tablet or chewable tablet; Oral

Cetirizine

Drug

Tablet or oral solution; Oral

fexofenadine

Drug

Tablet or oral suspension; Oral

Primary outcomes

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

    Time frame: Up to 16 weeks

    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).

  2. Shortest Actual Tolerated Infusion Duration

    Time frame: Up to 16 weeks

    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.

Secondary outcomes

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

    Time frame: Up to 16 weeks

    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.

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

    Time frame: Up to 16 weeks

    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.

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

    Time frame: Up to 16 weeks

    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.

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

    Time frame: Up to 16 weeks

    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.

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

    Time frame: Up to 16 weeks

    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.

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

    Time frame: Up to 16 weeks

    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.

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study title

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

Acronym: SHORTEN

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
Aug 31, 2023
Registry last updated
Oct 21, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.