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

Sanofi

Status and phase: Completed; Phase 4

Conditions: Fabry's Disease

Treatments: Drug: Fexofenadine; Drug: Loratadine; Drug: AGALSIDASE BETA (GZ419828); Drug: Acetaminophen; Drug: Cetirizine; Drug: Diphenhydramine; Drug: Dexamethasone; Drug: Montelukast

Study type: Interventional

Funder types: Industry

Identifiers

NCT06019728

LPS17726

U1111-1287-8570 (Registry Identifier)

Details and patient eligibility

About

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.

Full description

The total duration will be up to 7 months

Enrollment: 8 patients

Sex: All

Ages: 2 to 65 years old

Volunteers: No 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.

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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8 participants in 1 patient group

agalsidase beta

Experimental group

Description:

agalsidase beta 1 mg/kg infusion once every other week

Treatment:

Drug: Montelukast

Drug: Dexamethasone

Drug: Cetirizine

Drug: Diphenhydramine

Drug: Acetaminophen

Drug: Loratadine

Drug: AGALSIDASE BETA (GZ419828)

Drug: Fexofenadine