A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replacement Therapy in Chinese Participants With Fabry Disease

Genzyme, a Sanofi Company6 sites in 1 country22 target enrollmentOctober 13, 2021

ConditionsFabry Disease

InterventionsAgalsidase beta

DrugsAgalsidase beta

Overview

Phase: Phase 4
Intervention: Agalsidase beta
Conditions: Fabry Disease
Sponsor: Genzyme, a Sanofi Company
Enrollment: 22
Locations: 6
Primary Endpoint: Incidence of treatment-emergent adverse events (AEs)
Status: Completed
Last Updated: 7 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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.

Detailed 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

Registry

clinicaltrials.gov

Start Date

October 13, 2021

End Date

March 9, 2023

Last Updated

7 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Genzyme, a Sanofi Company

Responsible Party

Sponsor

Eligibility Criteria

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 m
•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.

Arms & Interventions

Agalsidase beta

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

Intervention: Agalsidase beta

Outcomes

Primary Outcomes

Incidence of treatment-emergent adverse events (AEs)

Time Frame: Baseline to week 50

Including TEAE, SAEs, and adverse events of special interest (AESIs) including infusion associated reactions (IARs) and change of clinical laboratory, vital signs and ECG

Secondary Outcomes

The percentage of participants with abnormal plasma GL3 values per central lab reference range(at Week 6, Week 12, Week 24 and Week 48)
The percent changes of plasma GL3(from baseline to Week 6, Week 12, Week 24 and Week 48)
The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)(from baseline to Week 6, Week 12, Week 24 and Week 48)
The change of Fabry disease symptoms(from baseline to Week 24 and Week 48)
The absolute change of estimated glomerular filtration rate (eGFR) by chronic kidney disease epidemiology collaboration (CKD-EPI) for adult (≥18 years)(from baseline to Week 12, Week 24, Week 36 and Week 48)
The absolute change of estimated glomerular filtration rate (eGFR) by Schwartz for children (8 ≤age <18 years)(from baseline to Week 12, Week 24, Week 36 and Week 48)
The percent changes of plasma lyso-GL3(from baseline to Week 6, Week 12, Week 24 and Week 48)
The absolute changes of plasma globotriaosylceramide (GL3)(from baseline to Week 6, Week 12, Week 24 and Week 48)
The number of participants with abnormal plasma GL3 values per central lab reference range(at Week 6, Week 12, Week 24 and Week 48)