Clinical trial NCT05054387: China Post-marketing Surveillance (PMS) Study of Fabrazyme®
Official title: A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replacement Therapy in Chinese Participants With Fabry Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Fabry Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2021-09-23
Enrollment: 22
Sex: ALL
Minimum age (years): 8
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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 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.

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (AEs)
timeFrame: Baseline to week 50
Secondary outcomes:
measure: The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)
timeFrame: from baseline to Week 6, Week 12, Week 24 and Week 48
measure: The percent changes of plasma lyso-GL3
timeFrame: from baseline to Week 6, Week 12, Week 24 and Week 48
measure: The absolute changes of plasma globotriaosylceramide (GL3)
timeFrame: from baseline to Week 6, Week 12, Week 24 and Week 48
measure: The percent changes of plasma GL3
timeFrame: from baseline to Week 6, Week 12, Week 24 and Week 48
measure: The number of participants with abnormal plasma GL3 values per central lab reference range
timeFrame: at Week 6, Week 12, Week 24 and Week 48
measure: The percentage of participants with abnormal plasma GL3 values per central lab reference range
timeFrame: at Week 6, Week 12, Week 24 and Week 48
measure: The change of Fabry disease symptoms
timeFrame: from baseline to Week 24 and Week 48
measure: The absolute change of estimated glomerular filtration rate (eGFR) by chronic kidney disease epidemiology collaboration (CKD-EPI) for adult (≥18 years)
timeFrame: from baseline to Week 12, Week 24, Week 36 and Week 48
measure: The absolute change of estimated glomerular filtration rate (eGFR) by Schwartz for children (8 ≤age <18 years)
timeFrame: from baseline to Week 12, Week 24, Week 36 and Week 48
Other outcomes:
Locations:
city: Beijing
country: China
facility: Investigational Site Number :1560003
city: Beijing
country: China
facility: Investigational Site Number :1560002
city: Shanghai
country: China
facility: Investigational Site Number :1560001
city: Shanghai
country: China
facility: Investigational Site Number :1560004
city: Taiyuan
country: China
facility: Investigational Site Number :1560006
city: Wuhan Shi
country: China
facility: Investigational Site Number :1560005
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: