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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-11-10

Study Completion Date

2024-10-25

Brief Summary

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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.

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Detailed Description

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The total duration will be up to 7 months

Conditions

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Fabry's Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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agalsidase beta

agalsidase beta 1 mg/kg infusion once every other week

Group Type EXPERIMENTAL

AGALSIDASE BETA (GZ419828)

Intervention Type DRUG

Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion

Acetaminophen

Intervention Type DRUG

Tablet or solution; Oral

Diphenhydramine

Intervention Type DRUG

Tablet or solution; Oral

Dexamethasone

Intervention Type DRUG

Tablet or solution; Oral

Montelukast

Intervention Type DRUG

Tablet or chewable tablet or oral granules; Oral

Loratadine

Intervention Type DRUG

Tablet or chewable tablet; Oral

Cetirizine

Intervention Type DRUG

Tablet or oral solution; Oral

Fexofenadine

Intervention Type DRUG

Tablet or oral suspension; Oral

Interventions

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AGALSIDASE BETA (GZ419828)

Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion

Intervention Type DRUG

Acetaminophen

Tablet or solution; Oral

Intervention Type DRUG

Diphenhydramine

Tablet or solution; Oral

Intervention Type DRUG

Dexamethasone

Tablet or solution; Oral

Intervention Type DRUG

Montelukast

Tablet or chewable tablet or oral granules; Oral

Intervention Type DRUG

Loratadine

Tablet or chewable tablet; Oral

Intervention Type DRUG

Cetirizine

Tablet or oral solution; Oral

Intervention Type DRUG

Fexofenadine

Tablet or oral suspension; Oral

Intervention Type DRUG

Other Intervention Names

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Fabrazyme

Eligibility Criteria

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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.

Minimum Eligible Age

2 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No