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Trial record 1 of 1 for:    LPS17726
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A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (SHORTEN)

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ClinicalTrials.gov Identifier: NCT06019728
Recruitment Status : Recruiting
First Posted : August 31, 2023
Last Update Posted : March 6, 2024
Sponsor:
Information provided by (Responsible Party):
Sanofi

Study Description
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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.

Condition or disease Intervention/treatment Phase
Fabry's Disease Drug: AGALSIDASE BETA (GZ419828) Drug: Acetaminophen Drug: Diphenhydramine Drug: Dexamethasone Drug: Montelukast Phase 4

Detailed Description:
The total duration will be up to 6 months
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Study to Investigate Safety and Tolerability of Higher Infusion Rate to shORten the duraTion of FabrazymE Infusion
Actual Study Start Date : November 10, 2023
Estimated Primary Completion Date : October 6, 2025
Estimated Study Completion Date : October 6, 2025

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Arms and Interventions
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Arm Intervention/treatment
Experimental: agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
 Drug: AGALSIDASE BETA (GZ419828)
Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion
Other Name: Fabrazyme

Drug: Acetaminophen
Tablet or solution; Oral

Drug: Diphenhydramine
Tablet or solution; Oral

Drug: Dexamethasone
Tablet or solution; Oral

Drug: Montelukast
Tablet or chewable tablet or oral granules; Oral


Outcome Measures
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Primary Outcome Measures :
  1. Reduction of infusion duration from pretrial average of recent 3 infusions [ Time Frame: Baseline to month 4 ]
  2. Reduction of infusion duration from initial 120 minutes [ Time Frame: Baseline to month 4 ]
  3. Shortest infusion duration each participant tolerates [ Time Frame: Baseline to month 4 ]

Secondary Outcome Measures :
  1. Number of participants achieving the shortest planned duration of infusion time [ Time Frame: Baseline to month 4 ]
  2. Percentage of participants achieving the shortest planned duration of infusion time [ Time Frame: Baseline to month 4 ]
  3. Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR [ Time Frame: Baseline to month 4 ]
  4. Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR [ Time Frame: Baseline to month 4 ]
  5. Number of participants achieving infusion duration without experiencing any IAR [ Time Frame: Baseline to month 4 ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06019728


Contacts
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Contact: Trial Transparency email recommended (Toll free for US & Canada) 800-633-1610 ext option 6 Contact-US@sanofi.com

Locations
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United States, Michigan
Infusion Associates Site Number : 1001 Recruiting
Grand Rapids, Michigan, United States, 49525
United States, New York
Metropolitan Hospital Center Site Number : 1004 Recruiting
New York, New York, United States, 10021
United States, Virginia
Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 1002 Recruiting
Fairfax, Virginia, United States, 22030
Sponsors and Collaborators
Sanofi
Investigators
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Study Director: Clinical Sciences & Operations Sanofi
More Information
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Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT06019728    
Other Study ID Numbers: LPS17726
U1111-1287-8570 ( Registry Identifier: ICTRP )
First Posted: August 31, 2023    Key Record Dates
Last Update Posted: March 6, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Fabry Disease
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
Metabolism, Inborn Errors
 Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Acetaminophen
Diphenhydramine
Promethazine
Dexamethasone
Montelukast
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs