A Real-world Analysis of Pharmacodynamic Response to Velmanase Alfa (Lamzede®) Treatment in Patients With Alpha-Mannosidosis Less Than 3 Years of Age
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 2
- 主要终点
- Pharmacodynamic Response to velmanase alfa
研究概览
简要总结
The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis.
The main questions it aims to answer are:
- study the effect of velmanase alfa on a marker of the disease called GlcNAc(Man)2 after one year of therapy
- explore how the child's body reacts to velmanase alfa during the therapy The parents or legal guardians of participants will be asked to provide the results of analyses performed in the routine clinical setting related to the participant's general health and the administration of velmanase alfa.
Additional data will be extracted from other observational sponsored studies/registries, compassionate use programs, investigator-initiated studies (IIS), and published case reports (presented in the literature) if existing.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 年龄范围
- — 至 3 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects with the provision of informed consent from their legal guardians (LAR)
- •Have a confirmed diagnosis of alpha mannosidosis
- •Have initiated treatment with velmanase alfa between birth to at least six weeks before turning 3 years of age
- •Have information on the disease marker GlcNAc(Man)2 obtained:
- •before velmanase treatment initiation (ideally max 6 month before), and at least one post-treatment sample, collected following at least six weeks of treatment.
- •Participants treated with Lamzede, 1 mg/kg body weight, via weekly intravenous infusions.
排除标准
- •Participants who have undergone prior hematopoietic stem cell transplantation (HSCT) or other investigational therapies for treating alfa mannosidosis (supportive treatments acceptable).
研究组 & 干预措施
Paediatric patients with alpha-mannosidosis treated with Lamzede before 3 years of age
Paediatric patients with a confirmed diagnosis of alpha-mannosidosis with data for at least one pre- and one post-Lamzede treatment sample obtained when < 3 YOA.
干预措施: Velmanase Alfa (Drug)
结局指标
主要结局
Pharmacodynamic Response to velmanase alfa
时间窗: 52 weeks of treatment
Change (absolute and relative) of GlcNAc(Man)2 level from pre-velmanase alfa treatment baseline in blood
次要结局
未报告次要终点