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

Last updated: March 28, 2024
Sponsor: Takeda
Overall Status: Completed

Phase

N/A

Condition

Gaucher Disease

Treatment

Standard of Care

Clinical Study ID

NCT04721366
MACS-2020-052801
TAK-669-4019
  • Ages < 5
  • All Genders

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.

Eligibility Criteria

Inclusion

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 years of age at treatmentinitiation.
  • 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 forGD.
  • In the opinion of the investigator, the participant's caregiver is capable ofunderstanding 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 initiationof any study procedures.

Exclusion

Exclusion Criteria:

  • The participant is an immediate family member, study site employee, or is in adependent relationship with a study site employee who is involved in conduct of thisstudy (e.g., child, sibling) or may consent under duress.
  • The participant is judged by the investigator as being ineligible for any otherreason.

Study Design

Total Participants: 11
Treatment Group(s): 1
Primary Treatment: Standard of Care
Phase:
Study Start date:
January 08, 2021
Estimated Completion Date:
April 17, 2023

Connect with a study center

  • Lysosomal & Rare Disorders Research & Treatment Center

    Fairfax, Virginia 22030
    United States

    Site Not Available

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