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

Sanofi

Status and phase

Enrolling

Phase 4

Conditions

Fabry's Disease

Treatments

Drug: AGALSIDASE BETA (GZ419828)

Drug: Acetaminophen

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 6 months

Enrollment

18 estimated 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.
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.