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

Takeda

Status: Completed

Conditions: Gaucher Disease

Treatments: Other: Standard of Care

Study type: Observational

Funder types: Industry

Identifiers

NCT04721366

TAK-669-4019 (Other Identifier)

MACS-2020-052801

Details and patient eligibility

About

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.

Enrollment: 11 patients

Sex: All

Ages: Under 5 years old

Volunteers: No Healthy Volunteers

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 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 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.

Trial design

11 participants in 1 patient group

Standard of Care (SoC)

Description:

Neonatal and pediatric participants who has been on ERT (VPRIV) will be followed up for 36 months from the time of treatment initiation as per SOC.

Treatment:

Other: Standard of Care