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

NCT04721366 | Completed

• 2 other identifiers: MACS-2020-052801TAK-669-4019
• Study Type: observational
• Target: 11
• Locations: 1 country
• Sites: 1

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.

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (7)

• Change From Baseline in Hemoglobin (Hb) Level

  Increase hemoglobin levels up to 11.0 gram per deciliter (g/dL) will be assessed.

  From start of ERT initiation up to 5 years of age

• Percent Change From Baseline in Platelet Count Increase

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

  From start of ERT initiation up to 5 years of age

• Percent Change From Baseline in Liver Volume

  Percent change from baseline in liver volume will be assessed.

  From start of ERT initiation up to 5 years of age

• Percent Change From Baseline for Spleen Volume

  Percent change from baseline for spleen volume will be assessed.

  From start of ERT initiation up to 5 years of age

• Percentage of Participants With Growth Normalization

  Percentage of participants with growth normalization will be assessed.

  From start of ERT initiation up to 5 years of age

• Percentage of Participants With Improvement in Bone Disease

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

  From start of ERT initiation up to 5 years of age

• Percentage of Participants With Improvement in Thrombocytopenia

  Percentage of participants with improvement in thrombocytopenia will be assessed.

  From start of ERT initiation up to 5 years of age

Secondary Outcomes (1)

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

  From start of ERT initiation up to 5 years of age

Study Arms (1)

Standard of Care (SoC)

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.

Other: Standard of Care

Interventions

Standard of Care OTHER

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

Also known as: SOC

Standard of Care (SoC)

Eligibility Criteria

• Age: Up to 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Neonatal and pediatric participants included have a diagnosis of GD type I and III and are currently being treated with total ERT (VPRIV) for less than or equal to (\<=) 36 months from the time of treatment initiation.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (1)

Lysosomal & Rare Disorders Research & Treatment Center

Fairfax, Virginia, 22030, United States

Location

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Gaucher Disease

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 21, 2021
• First Posted: January 22, 2021
• Study Start: January 8, 2021
• Primary Completion: April 17, 2023
• Study Completion: April 17, 2023
• Last Updated: March 29, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)