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
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Percent Change From Baseline in Liver Volume
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
结局指标
主要结局
Percent Change From Baseline in Liver Volume
时间窗: From start of ERT initiation up to 5 years of age
Percent change from baseline in liver volume will be assessed.
Percentage of Participants With Growth Normalization
时间窗: From start of ERT initiation up to 5 years of age
Percentage of participants with growth normalization will be assessed.
Percent Change From Baseline in Platelet Count Increase
时间窗: From start of ERT initiation up to 5 years of age
Percent change from baseline for platelet count increase will be assessed.
Change From Baseline in Hemoglobin (Hb) Level
时间窗: 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 for Spleen Volume
时间窗: 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 Improvement in Bone Disease
时间窗: 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
时间窗: From start of ERT initiation up to 5 years of age
Percentage of participants with improvement in thrombocytopenia will be assessed.
次要结局
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(From start of ERT initiation up to 5 years of age)