Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Change in Urinary Glycosaminoglycan (GAG) Excretion
研究概览
简要总结
This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) treated with a prior allogeneic transplant >2 years previously
- •Persons currently receiving Naglazyme may be accepted into the study
- •Age > 2 years
- •>10% engrafted based on most recent testing
- •Willing to commit to traveling to the University of Minnesota every 6 months
- •Written informed consent with parent/guardian consent for children < 18 years of age or persons unable to consent with minor assent if appropriate
排除标准
- •History of cardiac or pulmonary insufficiency or those requiring continuous supplemental oxygen
- •Pregnant or breastfeeding
- •Any condition that, in the view of the investigator, places the patient at high risk of poor treatment compliance or of not completing the study
研究组 & 干预措施
Naglazyme®
weekly Naglazyme® infusion for 2 years
干预措施: Naglazyme® (Drug)
结局指标
主要结局
Change in Urinary Glycosaminoglycan (GAG) Excretion
时间窗: Baseline and 2 years
Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years
Change in Distance Traveled
时间窗: Baseline and 2 years
Change in distance traveled in 6 minute walk and standard tests of range of motion and mobility. Improvement in distance walked from the start to study to end of the study is reported
Change in Neurocognitive Ability
时间窗: Baseline and 2 years
Subjects will undergo full neuropsychological testing annually and abbreviated testing for attention and adaptive skills at the six-month interval visits
次要结局
- Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy(2 years)