A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
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- STATUS
- Recruiting
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- End date
- Jan 2, 2023
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- participants needed
- 20
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- sponsor
- Takeda
Summary
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants under 5 years old 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.
Details
| Condition | Gaucher Disease, gaucher's disease |
|---|---|
| Treatment | Standard of Care |
| Clinical Study Identifier | NCT04721366 |
| Sponsor | Takeda |
| Last Modified on | 20 July 2021 |
How to participate?
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
Learn moreIf you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Learn moreComplete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.
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