NCT04721366

A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

Gaucher Disease During Infancy and Early Childhood and Experience With Enzyme Replacement Therapy (ERT) Using Velaglucerase Alfa (VPRIV): A Combined Retrospective and Prospective Cohort Study

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 11 (actual)
• Sponsor: Takeda · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests. This study is about collecting data available in the participant's medical record as well as data from each participant's ongoing treatment. No study medicines will be provided to participants in this study. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. When the participants start the study, they will visit the study clinic every 6 months after their first visit.

Conditions

• Gaucher Disease

Interventions

Type	Name	Description
OTHER	Standard of Care	Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC.

Timeline

• Start date: 2021-01-08
• Primary completion: 2023-04-17
• Completion: 2023-04-17
• First posted: 2021-01-22
• Last updated: 2024-03-29

Locations

1 site across 1 country: United States