Status:
COMPLETED
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
Lead Sponsor:
Takeda
Conditions:
Gaucher Disease
Eligibility:
All Genders
Up to 5 years
Brief 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. ...
Eligibility Criteria
Inclusion
- The participant's caregiver is able and willing to provide informed consent.
- The participant is male or female younger than or equal to 4 years 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 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
- 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.
Key Trial Info
Start Date :
January 8 2021
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
April 17 2023
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT04721366
Start Date
January 8 2021
End Date
April 17 2023
Last Update
March 29 2024
Active Locations (1)
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1
Lysosomal & Rare Disorders Research & Treatment Center
Fairfax, Virginia, United States, 22030