search
Back to results

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

Primary Purpose

Fabry Disease

Status
Completed
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Agalsidase beta
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease

Eligibility Criteria

8 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

  • Investigational Site Number :1560003
  • Investigational Site Number :1560002
  • Investigational Site Number :1560001
  • Investigational Site Number :1560004
  • Investigational Site Number :1560006
  • Investigational Site Number :1560005

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Agalsidase beta

Arm Description

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)
The percent changes of plasma lyso-GL3
The absolute changes of plasma globotriaosylceramide (GL3)
The percent changes of plasma GL3
The number of participants with abnormal plasma GL3 values per central lab reference range
The percentage of participants with abnormal plasma GL3 values per central lab reference range
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
The absolute change of estimated glomerular filtration rate (eGFR) by chronic kidney disease epidemiology collaboration (CKD-EPI) for adult (≥18 years)
The absolute change of estimated glomerular filtration rate (eGFR) by Schwartz for children (8 ≤age <18 years)

Full Information

First Posted
September 9, 2021
Last Updated
September 26, 2023
Sponsor
Genzyme, a Sanofi Company
search

1. Study Identification

Unique Protocol Identification Number
NCT05054387
Brief Title
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

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
October 13, 2021 (Actual)
Primary Completion Date
March 9, 2023 (Actual)
Study Completion Date
March 9, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
22 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Agalsidase beta
Arm Type
Experimental
Arm Description
Agalsidase beta treatment at approved dose and regimen, administered once every 2 weeks as an IV infusion
Intervention Type
Drug
Intervention Name(s)
Agalsidase beta
Other Intervention Name(s)
GZ419828 Fabrazyme
Intervention Description
Powder for concentration into a solution Intravenous (IV) infusion
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events (AEs)
Description
Including TEAE, SAEs, and adverse events of special interest (AESIs) including infusion associated reactions (IARs) and change of clinical laboratory, vital signs and ECG
Time Frame
Baseline to week 50
Secondary Outcome Measure Information:
Title
The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)
Time Frame
from baseline to Week 6, Week 12, Week 24 and Week 48
Title
The percent changes of plasma lyso-GL3
Time Frame
from baseline to Week 6, Week 12, Week 24 and Week 48
Title
The absolute changes of plasma globotriaosylceramide (GL3)
Time Frame
from baseline to Week 6, Week 12, Week 24 and Week 48
Title
The percent changes of plasma GL3
Time Frame
from baseline to Week 6, Week 12, Week 24 and Week 48
Title
The number of participants with abnormal plasma GL3 values per central lab reference range
Time Frame
at Week 6, Week 12, Week 24 and Week 48
Title
The percentage of participants with abnormal plasma GL3 values per central lab reference range
Time Frame
at Week 6, Week 12, Week 24 and Week 48
Title
The change of Fabry disease symptoms
Description
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
Time Frame
from baseline to Week 24 and Week 48
Title
The absolute change of estimated glomerular filtration rate (eGFR) by chronic kidney disease epidemiology collaboration (CKD-EPI) for adult (≥18 years)
Time Frame
from baseline to Week 12, Week 24, Week 36 and Week 48
Title
The absolute change of estimated glomerular filtration rate (eGFR) by Schwartz for children (8 ≤age <18 years)
Time Frame
from baseline to Week 12, Week 24, Week 36 and Week 48

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
Investigational Site Number :1560003
City
Beijing
ZIP/Postal Code
100034
Country
China
Facility Name
Investigational Site Number :1560002
City
Beijing
ZIP/Postal Code
100730
Country
China
Facility Name
Investigational Site Number :1560001
City
Shanghai
ZIP/Postal Code
200025
Country
China
Facility Name
Investigational Site Number :1560004
City
Shanghai
ZIP/Postal Code
201102
Country
China
Facility Name
Investigational Site Number :1560006
City
Taiyuan
ZIP/Postal Code
030001
Country
China
Facility Name
Investigational Site Number :1560005
City
Wuhan Shi
ZIP/Postal Code
016040
Country
China

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Learn more about this trial

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

We'll reach out to this number within 24 hrs