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

Completed

• Conditions: Gaucher Disease
• Interventions: Other: Standard of Care

• Registration Number: NCT04721366
• Lead Sponsor: Takeda

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.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-01-08
• Study First Submitted: 2021-01-21
• Study First Submitted QC: 2021-01-21
• Study First Posted: 2021-01-22
• Primary Completion: 2023-04-17
• Study Completion: 2023-04-17
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-28
• Last Update Posted: 2024-03-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

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.

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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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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Standard of Care (SoC)	Standard of Care	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.

Primary Outcome Measures

Name	Time	Method
Percent Change From Baseline in Liver Volume	From start of ERT initiation up to 5 years of age	Percent change from baseline in liver volume will be assessed.
Percentage of Participants With Growth Normalization	From start of ERT initiation up to 5 years of age	Percentage of participants with growth normalization will be assessed.
Percent Change From Baseline in Platelet Count Increase	From start of ERT initiation up to 5 years of age	Percent change from baseline for platelet count increase will be assessed.
Change From Baseline in Hemoglobin (Hb) Level	From start of ERT initiation up to 5 years of age	Increase hemoglobin levels up to 11.0 gram per deciliter (g/dL) will be assessed.
Percent Change From Baseline for Spleen Volume	From start of ERT initiation up to 5 years of age	Percent change from baseline for spleen volume will be assessed.
Percentage of Participants With Improvement in Bone Disease	From start of ERT initiation up to 5 years of age	Percentage of participants with improvement in bone disease will be assessed.
Percentage of Participants With Improvement in Thrombocytopenia	From start of ERT initiation up to 5 years of age	Percentage of participants with improvement in thrombocytopenia will be assessed.

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	From start of ERT initiation up to 5 years of age	An adverse event (AE) is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. AEs include SAEs, non-serious AEs.

Trial Locations

Locations (1)

Lysosomal & Rare Disorders Research & Treatment Center; 🇺🇸 Fairfax, Virginia, United States