Extension Study of Protocol ENB-006-09 Evaluating the Long-term Safety and Efficacy of Asfotase Alfa (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 4
- 主要终点
- Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09.
研究概览
简要总结
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
详细描述
Asfotase alfa was formerly referred to as ENB-0040
Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.
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
- 5 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Compliant and satisfactory completion of Enobia-sponsored clinical trial ENB-006-09
- •Written informed consent by parent or other legal guardian prior to any study procedures being performed
- •Parent or other legal guardian willing to comply with study requirements
排除标准
- •Clinically significant disease that precludes study participation, in the Investigator's opinion
- •Treatment with an investigational drug other than asfotase alfa
- •Enrollment in any study (other than ENB-006-09) involving an investigational drug, device, or treatment for HPP
- •Prior treatment with bisphosphonates
结局指标
主要结局
Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09.
时间窗: At least 72 months of treatment with asfotase alfa
Evaluation of radiographic change in rickets severity (as assessed by skeletal radiographs of the hands/wrists and knees) from the Baseline of Study ENB-006-09 (NCT00952484) to the End of Study (EOS) visit in Study ENB-008-10 using an ordinal RGI-C scale score. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP associated rickets) to +3 (indicative of complete or near complete healing of HPP associated rickets). The time points will be pre-treatment (Baseline from Study ENB-006-09) to the last radiographic assessment in Study ENB-008-10, which represents at least 72 months of treatment.
次要结局
未报告次要终点