A Phase 4 interventional study of agalsidase beta (GZ419828) and agalsidase alfa in Fabry Disease, sponsored by Sanofi. Withdrawn. Open to male participants aged 16 Years to 45 Years. Per ClinicalTrials.gov, last updated 2023-04-07.
Sponsored by Sanofi · Phase 4, Interventional, and Treatment
Primary Objective:
To assess reduction of plasma lyso-GL3 level after switch to agalsidase beta from agalsidase alfa
Secondary Objectives:
The study will have a screening period of up to 9 weeks. Eligible participants will be randomized to switch to agalsidase beta or to continue agalsidase alfa in a 1:1 ratio for a period of 12 months (52 weeks).
242 studies on the registry are indexed under Fabry Disease; 54 are open to participants now.
Browse Fabry Disease studies →Sanofi is the lead sponsor of 1,508 studies on the registry; 90 are open to participants now.
Of its 198 completed or terminated interventional studies of FDA-regulated products, 118 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion criteria:
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Commercially available agalsidase beta treatment at approved dose and regimen;administered once every 2 weeks as an IV infusion
Drug: agalsidase beta (GZ419828)
Commercially available agalsidase alfa treatment at approved dose and regimen; administered once every 2 weeks as an IV infusion
Drug: agalsidase alfa
Pharmaceutical form:Powder for concentrate for solution for infusion Route of administration: Intravenous (IV) infusion,
Pharmaceutical form:concentrate for solution for infusion Route of administration: Intravenous (IV) infusion
Change in Plasma globotriaosylsphingosine (lyso-GL3) level
Change from baseline to 12 months (week 52) for plasma lyso-GL3 level
Time frame: Baseline, 12 months (week 52)
Change in GL3 content in podocytes
Change from baseline to 12 months (week 52) for GL3 content in podocytes
Time frame: Baseline, 12 months (week 52)
Change in GL3 content in endothelial skin cells
Change from baseline to 12 months (Week 52) for GL3 content in endothelial skin cells
Time frame: Baseline, 12 months (week 52)
Change in measured glomerular filtration rate (mGFR)
Change from baseline to 12 months (Week 52) for measured glomerular filtration rate (mGFR) (measured by iohexol clearance)
Time frame: Baseline, 12 months (week 52)
Change in estimated glomerular filtration rate (eGFR) calculated
Change from baseline to 12 months (Week 52) for estimated glomerular filtration rate (eGFR) calculated using age appropriate formula \[Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)/ Bedside-Schwartz\]
Time frame: Baseline, 12 months (week 52)
Change in Mainz Severity Score Index (MSSI) total score
Change from baseline to 12 months (Week 52) for Mainz Severity Score Index (MSSI), based on MSSI total score
Time frame: Baseline, 12 months (week 52)
Change in Fabry Disease Patient Reported Outcomes (FD-PRO) total symptom score
Change from baseline to 12 months (Week 52) in Fabry Disease Patient Reported Outcomes (FD-PRO) score, based on FD-PRO total symptom score
Time frame: Baseline, 12 months (week 52)
No study locations are listed for this record.
Plan to share: 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
No publications or documents are linked to this record.
This study is withdrawn, as verified in Apr 2023. You cannot join it, but the record below documents what was studied.
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