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

• Sponsor: Genzyme, a Sanofi Company (Industry)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT05054387
• Collaborator: (none)
• Enrollment: 22
• Locations: 6
• Arm: 1
• Anticipated Duration (Months): 18.9
• Patients Per Site: 3.7
• Patients Per Site Per Month: 0.2

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Fabry's Disease	Drug: Agalsidase beta	Phase 4

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

• Study Type: Interventional
• Actual Enrollment: 22 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: Oct 13, 2021
• Anticipated Primary Completion Date: May 11, 2023
• Anticipated Study Completion Date: May 11, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Agalsidase beta Agalsidase beta treatment at approved dose and regimen, administered once every 2 weeks as an IV infusion	Drug: Agalsidase beta; Powder for concentration into a solution Intravenous (IV) infusion; Other Names: GZ419828 Fabrazyme

Outcome Measures

Primary Outcome Measures

1. Incidence of treatment-emergent adverse events (AEs) [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 Outcome Measures

1. The absolute changes of plasma globotriaosylsphingosine (lyso-GL3) [from baseline to Week 6, Week 12, Week 24 and Week 48]

2. The percent changes of plasma lyso-GL3 [from baseline to Week 6, Week 12, Week 24 and Week 48]

3. The absolute changes of plasma globotriaosylceramide (GL3) [from baseline to Week 6, Week 12, Week 24 and Week 48]

4. The percent changes of plasma GL3 [from baseline to Week 6, Week 12, Week 24 and Week 48]

5. The number of participants with abnormal plasma GL3 values per central lab reference range [at Week 6, Week 12, Week 24 and Week 48]

6. The percentage of participants with abnormal plasma GL3 values per central lab reference range [at Week 6, Week 12, Week 24 and Week 48]

7. The change of Fabry disease symptoms [from baseline to Week 24 and Week 48]

   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

8. 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]

9. 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]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 8 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigational Site Number :1560003	Beijing		China	100034
2	Investigational Site Number :1560002	Beijing		China	100730
3	Investigational Site Number :1560001	Shanghai		China	200025
4	Investigational Site Number :1560004	Shanghai		China	201102
5	Investigational Site Number :1560006	Taiyuan		China	030001
6	Investigational Site Number :1560005	Wuhan Shi		China	016040

Sponsors and Collaborators

• Genzyme, a Sanofi Company

Investigators

• Study Director: Clinical Sciences & Operations, Sanofi

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Genzyme, a Sanofi Company
• ClinicalTrials.gov Identifier: NCT05054387
• Other Study ID Numbers: LPS16583; U1111-1255-4881
• First Posted: Sep 23, 2021
• Last Update Posted: May 9, 2022
• Last Verified: May 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• 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
• Additional relevant MeSH terms: Fabry Disease

Study Results

No Results Posted as of May 9, 2022