An observational study in Gaucher Disease, sponsored by Takeda. Completed at 1 site in United States. Open to participants aged Up to 5 Years. Per ClinicalTrials.gov, last updated 2024-03-29.
Sponsored by Takeda · Observational
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.
171 studies on the registry are indexed under Gaucher Disease; 37 are open to participants now.
This study's enrollment of 11 is below the median of 60 across 63 observational studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →Takeda is the lead sponsor of 1,002 studies on the registry; 92 are open to participants now.
Of its 173 completed or terminated interventional studies of FDA-regulated products, 149 (86%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Exclusion Criteria:
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
Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC.
Also known as: SOC
Change From Baseline in Hemoglobin (Hb) Level
Increase hemoglobin levels up to 11.0 gram per deciliter (g/dL) will be assessed.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: From start of ERT initiation up to 5 years of age
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
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.
Time frame: From start of ERT initiation up to 5 years of age
Plan to share: No — De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).
This study is completed, as verified in Mar 2024. You cannot join it, but the record below documents what was studied.
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