Fabry DiseasePhase 4NCT05054387
China Post-marketing Surveillance (PMS) Study of Fabrazyme®
A Phase 4 study testing Agalsidase beta in people with Fabry Disease, run by Genzyme, a Sanofi Company at 6 sites in China.
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. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Agalsidase beta
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 6 sitesChina
- Study dates
- 13 Oct 2021to 9 Mar 2023
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
China6 sites
Investigational Site Number :1560003
Beijing, 100034
Investigational Site Number :1560002
Beijing, 100730
Investigational Site Number :1560001
Shanghai, 200025
Investigational Site Number :1560004
Shanghai, 201102
Investigational Site Number :1560006
Taiyuan, 030001
Investigational Site Number :1560005
Wuhan, 016040
About this study
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
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.
Read the 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
- Conditions
- Fabry Disease
- Being tested
- Agalsidase beta
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Agalsidase betaDrug
Also called GZ419828 Fabrazyme
Powder for concentration into a solution Intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| Agalsidase beta | Experimental | Agalsidase beta treatment at approved dose and regimen, administered once every 2 weeks as an IV infusion Receives: Drug: Agalsidase beta |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline to week 50
Secondary outcomes (9)
The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)
Measured: from baseline to Week 6, Week 12, Week 24 and Week 48
The percent changes of plasma lyso-GL3
Measured: from baseline to Week 6, Week 12, Week 24 and Week 48
The absolute changes of plasma globotriaosylceramide (GL3)
Measured: from baseline to Week 6, Week 12, Week 24 and Week 48
The percent changes of plasma GL3
Measured: 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
Measured: at Week 6, Week 12, Week 24 and Week 48
The percentage of participants with abnormal plasma GL3 values per central lab reference range
Measured: at Week 6, Week 12, Week 24 and Week 48
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
Measured: 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)
Measured: 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)
Measured: from baseline to Week 12, Week 24, Week 36 and Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2021
- Study start13 Oct 2021
- Primary completion9 Mar 2023
- Study completion9 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05054387
- Sponsor's protocol ID
- LPS16583
- Other IDs
- U1111-1255-4881
- Status last verified
- Sep 2025
- First submitted
- 9 Sep 2021
- Last updated
- 11 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Publications
Ren H, Zhang W, Ouyang Y, Guo J, Xu H, Ma J, Luo X, Pan X, Yuan Y, Zhang W, Shen Q, Li B, Feng Q, Liu S, Chen N. A phase 4, open-label, multicenter study of the safety and efficacy of agalsidase beta in Chinese patients with Fabry disease. Orphanet J Rare Dis. 2025 Aug 4;20(1):401. doi: 10.1186/s13023-025-03950-7.
PubMed 40760696
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05054387
- Acronym / sponsor ID
- LPS16583
- Conditions
- Fabry Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 13 Oct 2021 (actual)
- Primary completion
- 9 Mar 2023 (actual)
- Study completion
- 9 Mar 2023 (actual)
- First posted
- 23 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Agalsidase beta | Experimental | Drug: Agalsidase betaAgalsidase beta treatment at approved dose and regimen, administered once every 2 weeks as an IV infusion |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Agalsidase betaAlso: GZ419828 FabrazymePowder for concentration into a solution Intravenous (IV) infusion | Drug | Agalsidase beta |
Primary outcome
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
9 secondary outcomes
The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)
Time frame: from baseline to Week 6, Week 12, Week 24 and Week 48
The percent changes of plasma lyso-GL3
Time frame: from baseline to Week 6, Week 12, Week 24 and Week 48
The absolute changes of plasma globotriaosylceramide (GL3)
Time frame: from baseline to Week 6, Week 12, Week 24 and Week 48
The percent changes of plasma GL3
Time frame: 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
Time frame: at Week 6, Week 12, Week 24 and Week 48
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
The change of Fabry disease symptoms
Time frame: 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)
Time frame: 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)
Time frame: from baseline to Week 12, Week 24, Week 36 and Week 48
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2025.
- Derived from this study: PubMed 40760696Ren H, Zhang W, Ouyang Y, Guo J, Xu H, Ma J, Luo X, Pan X, Yuan Y, Zhang W, Shen Q, Li B, Feng Q, Liu S, Chen N. A phase 4, open-label, multicenter study of the safety and efficacy of agalsidase beta in Chinese patients with Fabry disease. Orphanet J Rare Dis. 2025 Aug 4;20(1):401. doi: 10.1186/s13023-025-03950-7.
- LPS16583 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Agalsidase beta in people with Fabry Disease, run by Genzyme, a Sanofi Company at 6 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (AEs) (time frame: Baseline to week 50). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05054387, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Sep 2025.