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

NCT04721366 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 11

Last updated 2024-03-29

No results posted yet for this study

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

OTHER

Standard of Care

Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC.

Sponsors & Collaborators

Principal Investigators

  • Study Director · Takeda

Eligibility

Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-01-08
Primary Completion
2023-04-17
Completion
2023-04-17

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04721366 on ClinicalTrials.gov