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

Primary Purpose

Fabry's Disease

Status
Not yet recruiting
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
AGALSIDASE BETA (GZ419828)
Acetaminophen
Diphenhydramine
Dexamethasone
Montelukast
Sponsored by
Sanofi
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry's Disease

Eligibility Criteria

2 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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. Cohort 5: ERT-naïve participants. 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, 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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    agalsidase beta

    Arm Description

    agalsidase beta 1 mg/kg infusion once every other week

    Outcomes

    Primary Outcome Measures

    Reduction of infusion duration from pretrial average of recent 3 infusions
    Reduction of infusion duration from initial 120 minutes
    Shortest infusion duration each participant tolerates

    Secondary Outcome Measures

    Number of participants achieving the shortest planned duration of infusion time
    Percentage of participants achieving the shortest planned duration of infusion time
    Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR
    Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR
    Number of participants achieving infusion duration without experiencing any IAR

    Full Information

    First Posted
    August 25, 2023
    Last Updated
    September 14, 2023
    Sponsor
    Sanofi
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    1. Study Identification

    Unique Protocol Identification Number
    NCT06019728
    Brief Title
    A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
    Acronym
    SHORTEN
    Official Title
    A Study to Investigate Safety and Tolerability of Higher Infusion Rate to shORten the duraTion of FabrazymE Infusion
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    September 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    September 29, 2023 (Anticipated)
    Primary Completion Date
    September 4, 2025 (Anticipated)
    Study Completion Date
    September 4, 2025 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Sanofi

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

    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.
    Detailed Description
    The total duration will be up to 6 months

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Fabry's Disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 4
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    18 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    agalsidase beta
    Arm Type
    Experimental
    Arm Description
    agalsidase beta 1 mg/kg infusion once every other week
    Intervention Type
    Drug
    Intervention Name(s)
    AGALSIDASE BETA (GZ419828)
    Other Intervention Name(s)
    Fabrazyme
    Intervention Description
    Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion
    Intervention Type
    Drug
    Intervention Name(s)
    Acetaminophen
    Intervention Description
    Tablet or solution; Oral
    Intervention Type
    Drug
    Intervention Name(s)
    Diphenhydramine
    Intervention Description
    Tablet or solution; Oral
    Intervention Type
    Drug
    Intervention Name(s)
    Dexamethasone
    Intervention Description
    Tablet or solution; Oral
    Intervention Type
    Drug
    Intervention Name(s)
    Montelukast
    Intervention Description
    Tablet or chewable tablet or oral granules; Oral
    Primary Outcome Measure Information:
    Title
    Reduction of infusion duration from pretrial average of recent 3 infusions
    Time Frame
    Baseline to month 4
    Title
    Reduction of infusion duration from initial 120 minutes
    Time Frame
    Baseline to month 4
    Title
    Shortest infusion duration each participant tolerates
    Time Frame
    Baseline to month 4
    Secondary Outcome Measure Information:
    Title
    Number of participants achieving the shortest planned duration of infusion time
    Time Frame
    Baseline to month 4
    Title
    Percentage of participants achieving the shortest planned duration of infusion time
    Time Frame
    Baseline to month 4
    Title
    Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR
    Time Frame
    Baseline to month 4
    Title
    Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR
    Time Frame
    Baseline to month 4
    Title
    Number of participants achieving infusion duration without experiencing any IAR
    Time Frame
    Baseline to month 4

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Maximum Age & Unit of Time
    65 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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. Cohort 5: ERT-naïve participants. 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, 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.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Trial Transparency email recommended (Toll free for US & Canada)
    Phone
    800-633-1610
    Ext
    option 6
    Email
    Contact-US@sanofi.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Clinical Sciences & Operations
    Organizational Affiliation
    Sanofi
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Plan to Share IPD
    Yes
    IPD Sharing Plan Description
    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 Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

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