China Post-marketing Surveillance (PMS) Study of Fabrazyme®

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

22 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-10-13

Study Completion Date

2023-03-09

Brief Summary

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This is a 54-week Phase 4, open label, single arm study to evaluate the safety and the efficacy of Fabrazyme (agalsidase beta) as enzyme replacement therapy (ERT) in Chinese participants with Fabry Disease.

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Detailed Description

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Study participation for each patient will be total of 54 weeks which will include 4 weeks of screening, 48 weeks of treatment period and 2 weeks of post study treatment observation

Conditions

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Fabry 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 treatment at approved dose and regimen, administered once every 2 weeks as an IV infusion

Group Type EXPERIMENTAL

Agalsidase beta

Intervention Type DRUG

Powder for concentration into a solution Intravenous (IV) infusion

Interventions

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Agalsidase beta

Powder for concentration into a solution Intravenous (IV) infusion

Intervention Type DRUG

Other Intervention Names

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GZ419828 Fabrazyme

Eligibility Criteria

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Inclusion Criteria

* Participant must be 8 years of age or older, at the time of signing the informed consent
* Participants naive to agalsidase beta and agalsidase alpha
* Chinese participants diagnosed with Fabry disease and with documented plasma or leukocyte αGAL activity deficient below laboratory's reference range, and/or documented diagnosis by genotyping
* Participants must have one or more symptoms and signs consistent with manifestations of Fabry disease (not limited to neuropathic pain, chronic kidney disease, hypertrophic cardiomyopathy, cardiac rhythm disturbances, cerebrovascular involvement, cornea verticillata, angiokeratoma, gastrointestinal symptoms, hypo- or anhydrosis)
* A female participant is eligible to participate if she is not pregnant or breastfeeding and use an acceptable contraceptive method
* Participants and/or participant's legal representative capable of giving signed informed consent.

Exclusion Criteria

* The participant has undergone kidney transplantation.
* The participant has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) in the opinion of the Investigator, would preclude participation in the trial.
* Received an investigational drug, or device, other than Fabrazyme, within 30 days of anticipated IMPs administration or 5 half-lives of the previous investigational drug, whichever is longer.
* The patient has current evidence of kidney failure or renal insufficiency, as defined by eGFR \<30 mL/min/1.73 m2.
* Individuals who have life threatening hypersensitivity (anaphylactic reaction) to the active substance or any of the excipients included.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Minimum Eligible Age

8 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No