A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease (PEDS)
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|ClinicalTrials.gov Identifier: NCT04721366|
Recruitment Status : Recruiting
First Posted : January 22, 2021
Last Update Posted : January 9, 2023
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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.
|Condition or disease||Intervention/treatment|
|Gaucher Disease||Other: Standard of Care|
|Study Type :||Observational|
|Estimated Enrollment :||20 participants|
|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|
|Actual Study Start Date :||January 8, 2021|
|Estimated Primary Completion Date :||January 2, 2025|
|Estimated Study Completion Date :||January 2, 2025|
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
Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC.
Other Name: SOC
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||up to 5 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Non-Probability Sample|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04721366
|Contact: Takeda Contact||+1 866 842 5335||ClinicalTransparency@takeda.com|
|United States, Virginia|
|Lysosomal & Rare Disorders Research & Treatment Center||Recruiting|
|Fairfax, Virginia, United States, 22030|
|Contact: Site Contact 703-261-6220 firstname.lastname@example.org|
|Principal Investigator: Ozlem Goker-Alpan|
|Study Director:||Study Director||Takeda|
|Other Study ID Numbers:||
TAK-669-4019 ( Other Identifier: Takeda )
|First Posted:||January 22, 2021 Key Record Dates|
|Last Update Posted:||January 9, 2023|
|Last Verified:||January 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
|Plan Description:||Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.|
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
|Access Criteria:||IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lipid Metabolism Disorders