Clinical trial NCT04721366: A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
Official 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Gaucher Disease
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2021-01-22
Enrollment: 11
Sex: ALL
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change From Baseline in Hemoglobin (Hb) Level
timeFrame: From start of ERT initiation up to 5 years of age
measure: Percent Change From Baseline in Platelet Count Increase
timeFrame: From start of ERT initiation up to 5 years of age
measure: Percent Change From Baseline in Liver Volume
timeFrame: From start of ERT initiation up to 5 years of age
measure: Percent Change From Baseline for Spleen Volume
timeFrame: From start of ERT initiation up to 5 years of age
measure: Percentage of Participants With Growth Normalization
timeFrame: From start of ERT initiation up to 5 years of age
measure: Percentage of Participants With Improvement in Bone Disease
timeFrame: From start of ERT initiation up to 5 years of age
measure: Percentage of Participants With Improvement in Thrombocytopenia
timeFrame: From start of ERT initiation up to 5 years of age
Secondary outcomes:
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From start of ERT initiation up to 5 years of age
Other outcomes:
Locations:
city: Fairfax
country: United States
facility: Lysosomal & Rare Disorders Research & Treatment Center
state: Virginia
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: