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

Fabry Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05054387
• Other study ID #: LPS16583
• Secondary ID: U1111-1255-4881
• Status: Completed
• Phase: Phase 4
• Start date: October 13, 2021
• Est. completion date: March 9, 2023

Study information

• Verified date: September 2023
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: March 9, 2023
• Est. primary completion date: March 9, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years and older

Eligibility

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 aGAL 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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• Agalsidase beta
Powder for concentration into a solution Intravenous (IV) infusion

Locations

Country	Name	City	State
China	Investigational Site Number :1560002	Beijing
China	Investigational Site Number :1560003	Beijing
China	Investigational Site Number :1560001	Shanghai
China	Investigational Site Number :1560004	Shanghai
China	Investigational Site Number :1560006	Taiyuan
China	Investigational Site Number :1560005	Wuhan Shi

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events (AEs)	Including TEAE, SAEs, and adverse events of special interest (AESIs) including infusion associated reactions (IARs) and change of clinical laboratory, vital signs and ECG	Baseline to week 50
Secondary	The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)		from baseline to Week 6, Week 12, Week 24 and Week 48
Secondary	The percent changes of plasma lyso-GL3		from baseline to Week 6, Week 12, Week 24 and Week 48
Secondary	The absolute changes of plasma globotriaosylceramide (GL3)		from baseline to Week 6, Week 12, Week 24 and Week 48
Secondary	The percent changes of plasma GL3		from baseline to Week 6, Week 12, Week 24 and Week 48
Secondary	The number of participants with abnormal plasma GL3 values per central lab reference range		at Week 6, Week 12, Week 24 and Week 48
Secondary	The percentage of participants with abnormal plasma GL3 values per central lab reference range		at Week 6, Week 12, Week 24 and Week 48
Secondary	The change of Fabry disease symptoms	The change of Fabry disease symptoms assessment (improved, worsen or same): angiokeratoma, sweating, chronic abdominal pain, level of activity, exercise tolerance and heat tolerance, headache, tinnitus	from baseline to Week 24 and Week 48
Secondary	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
Secondary	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