Trials / Withdrawn
WithdrawnNCT04143958
To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease
A Randomized, Open-label, Active Comparator, 2-arm, Prospective Study to Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease
- Status
- Withdrawn
- Phase
- Phase 4
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- Sanofi · Industry
- Sex
- Male
- Age
- 16 Years – 45 Years
- Healthy volunteers
- Not accepted
Summary
Primary Objective: To assess reduction of plasma lyso-GL3 level after switch to agalsidase beta from agalsidase alfa Secondary Objectives: * To assess reduction of kidney podocyte GL3 content after switch to agalsidase beta from agalsidase alfa * To assess reduction of GL3 content in endothelial skin cells after switch to agalsidase beta from agalsidase alfa * To assess change in renal function after switch to agalsidase beta from agalsidase alfa * To assess disease severity and clinical changes after switch to agalsidase beta from agalsidase alfa * To assess improvement in symptoms of Fabry disease after switch to agalsidase beta from agalsidase alfa
Detailed description
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).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | agalsidase beta (GZ419828) | Pharmaceutical form:Powder for concentrate for solution for infusion Route of administration: Intravenous (IV) infusion, |
| DRUG | agalsidase alfa | Pharmaceutical form:concentrate for solution for infusion Route of administration: Intravenous (IV) infusion |
Timeline
- Start date
- 2020-09-01
- Primary completion
- 2023-11-01
- Completion
- 2023-11-01
- First posted
- 2019-10-30
- Last updated
- 2023-04-07
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04143958. Inclusion in this directory is not an endorsement.