Clinical trial NCT06019728: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Fabry's Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2023-08-31
Enrollment: 8
Sex: ALL
Minimum age (years): 2
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 7 months

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.

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.

Primary outcomes:
measure: Reduction of infusion duration from pretrial average of recent 3 infusions
timeFrame: Baseline to month 4
measure: Reduction of infusion duration from initial 120 minutes
timeFrame: Baseline to month 4
measure: Shortest infusion duration each participant tolerates
timeFrame: Baseline to month 4
Secondary outcomes:
measure: Number of participants achieving the shortest planned duration of infusion time
timeFrame: Baseline to month 4
measure: Percentage of participants achieving the shortest planned duration of infusion time
timeFrame: Baseline to month 4
measure: Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR
timeFrame: Baseline to month 4
measure: Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR
timeFrame: Baseline to month 4
measure: Number of participants achieving infusion duration without experiencing any IAR
timeFrame: Baseline to month 4
Other outcomes:
Locations:
city: Santa Monica
country: United States
facility: UCLA Medical Center Site Number : 1003
state: California
city: Atlanta
country: United States
facility: Emory University School of Medicine Site Number : 1005
state: Georgia
city: Grand Rapids
country: United States
facility: Infusion Associates Site Number : 1001
state: Michigan
city: New York
country: United States
facility: Metropolitan Hospital Center Site Number : 1004
state: New York
city: Fairfax
country: United States
facility: Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 1002
state: Virginia
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: