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

Genzyme

Status and phase

Completed

Phase 4

Conditions

Fabry Disease

Treatments

Drug: Agalsidase beta

Study type

Interventional

Funder types

Industry

Identifiers

NCT05054387

LPS16583

U1111-1255-4881 (Registry Identifier)

Details and patient eligibility

About

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.

Full description

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

Enrollment

22 patients

Sex

All

Ages

8+ years old

Volunteers

No Healthy Volunteers

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.