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

  • End date
    Jan 2, 2023
  • participants needed
  • sponsor
Updated on 20 July 2021
velaglucerase alfa


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.

Condition Gaucher Disease, gaucher's disease
Treatment Standard of Care
Clinical Study IdentifierNCT04721366
Last Modified on20 July 2021


Yes No Not Sure

Inclusion Criteria

The participant's caregiver is able and willing to provide informed consent
The participant is male or female younger than or equal to 4 year of age at treatment initiation
The participant has received and confirmed a current diagnosis of GD type 1 or type 3 (biochemically and/or genetically)
The participant has been receiving intravenous (IV) Velaglucerase alfa 60 units per kilogram (U/kg) every other week (EOW) ERT treatment for GD
In the opinion of the investigator, the participant's caregiver is capable of understanding and complying with protocol requirements
The participant's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures

Exclusion Criteria

The participant is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in conduct of this study (e.g., child, sibling) or may consent under duress
The participant is judged by the investigator as being ineligible for any other reason
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How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If 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.

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Complete 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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