SHORTEN: A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
Study Details
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 4 |
Detailed Description
The total duration will be up to 6 months
Study Design
Arms and Interventions
| 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 Names:
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
Primary Outcome Measures
- Reduction of infusion duration from pretrial average of recent 3 infusions [Baseline to month 4]
- Reduction of infusion duration from initial 120 minutes [Baseline to month 4]
- Shortest infusion duration each participant tolerates [Baseline to month 4]
Secondary Outcome Measures
- Number of participants achieving the shortest planned duration of infusion time [Baseline to month 4]
- Percentage of participants achieving the shortest planned duration of infusion time [Baseline to month 4]
- Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR [Baseline to month 4]
- Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR [Baseline to month 4]
- Number of participants achieving infusion duration without experiencing any IAR [Baseline to month 4]
Eligibility Criteria
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
Locations
No locations specified.Sponsors and Collaborators
- Sanofi
Investigators
- Study Director: Clinical Sciences & Operations, Sanofi
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- LPS17726
- U1111-1287-8570