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

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

Tracking Information

• First Submitted Date: August 25, 2023
• First Posted Date: August 31, 2023
• Last Update Posted Date: March 6, 2024
• Actual Study Start Date: November 10, 2023
• Estimated Primary Completion Date: October 6, 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 30, 2023)

• Reduction of infusion duration from pretrial average of recent 3 infusions [ Time Frame: Baseline to month 4 ]
• Reduction of infusion duration from initial 120 minutes [ Time Frame: Baseline to month 4 ]
• Shortest infusion duration each participant tolerates [ Time Frame: Baseline to month 4 ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: August 30, 2023)

• Number of participants achieving the shortest planned duration of infusion time [ Time Frame: Baseline to month 4 ]
• Percentage of participants achieving the shortest planned duration of infusion time [ Time Frame: Baseline to month 4 ]
• Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR [ Time Frame: Baseline to month 4 ]
• Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR [ Time Frame: Baseline to month 4 ]
• Number of participants achieving infusion duration without experiencing any IAR [ Time Frame: Baseline to month 4 ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
• Official Title: A Study to Investigate Safety and Tolerability of Higher Infusion Rate to shORten the duraTion of FabrazymE Infusion
• 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
• Study Type: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Fabry's Disease

Intervention

• 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

Study Arms

Experimental: agalsidase beta

agalsidase beta 1 mg/kg infusion once every other week

Interventions:

• Drug: AGALSIDASE BETA (GZ419828)
• Drug: Acetaminophen
• Drug: Diphenhydramine
• Drug: Dexamethasone
• Drug: Montelukast

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: August 30, 2023): 18
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: October 6, 2025
• Estimated Primary Completion Date: October 6, 2025 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years to 65 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

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

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT06019728
• Other Study ID Numbers: LPS17726; U1111-1287-8570 ( Registry Identifier: ICTRP )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

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

• Current Responsible Party: Sanofi
• Original Responsible Party: Same as current
• Current Study Sponsor: Sanofi
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Clinical Sciences & Operations; Sanofi

• PRS Account: Sanofi
• Verification Date: March 2024