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NCT Number: NCT04721366

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

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.

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Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

Up to 5 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Lysosomal & Rare Disorders Research & Treatment Center

Fairfax, Virginia, 22030, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Standard of care

Other

Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC.

Other names: SOC

Primary outcomes

  1. Change From Baseline in Hemoglobin (Hb) Level

    Time frame: 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.

  2. Percent Change From Baseline in Platelet Count Increase

    Time frame: From start of ERT initiation up to 5 years of age

    Percent change from baseline for platelet count increase will be assessed.

  3. Percent Change From Baseline in Liver Volume

    Time frame: From start of ERT initiation up to 5 years of age

    Percent change from baseline in liver volume will be assessed.

  4. Percent Change From Baseline for Spleen Volume

    Time frame: From start of ERT initiation up to 5 years of age

    Percent change from baseline for spleen volume will be assessed.

  5. Percentage of Participants With Growth Normalization

    Time frame: From start of ERT initiation up to 5 years of age

    Percentage of participants with growth normalization will be assessed.

  6. Percentage of Participants With Improvement in Bone Disease

    Time frame: From start of ERT initiation up to 5 years of age

    Percentage of participants with improvement in bone disease will be assessed.

  7. Percentage of Participants With Improvement in Thrombocytopenia

    Time frame: From start of ERT initiation up to 5 years of age

    Percentage of participants with improvement in thrombocytopenia will be assessed.

Secondary outcomes

  1. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

Gaucher Disease During Infancy and Early Childhood and Experience With Enzyme Replacement Therapy (ERT) Using Velaglucerase Alfa (VPRIV): A Combined Retrospective and Prospective Cohort Study

Acronym: PEDS

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Jan 22, 2021
Registry last updated
Mar 29, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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