NCT02456038已完成2 期
A Multicenter Study of the Safety and Efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Patients With Hypophosphatasia (HPP)
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan12 个研究点 分布在 1 个国家目标入组 13 人开始时间: 2014年8月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 13
- 试验地点
- 12
- 主要终点
- Number of subjects with Adverse Events as an assessment of the Safety of repeated subcutaneous (SC) injections of asfotase alfa
研究概览
简要总结
The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."
- •Patient who has been already treated with Asfotae Alfa (ALXN1215) out of this clinical trial
- •Patient who has been diagnosed as HPP
- •Documented diagnosis of HPP as indicated by:
- •Total serum alkaline phosphatase below the lower limit of normal for age
- •Ultrasonographic features of prenatal, characterized by:
- •severe short extremities (femur length <-4SD in second and third trimesters) 2) extending into the metaphysis (femur metaphysis length or femur length >0.33) 3) craniotabes 4) Hypoplastic thorax (Thoracic or abdominal circumference <0.6) (3) Computed tomographic findings of prenatal, characterized by:
- •Generalized decreased ossification
- •Extreme shortening of tubular bones
- •Hypoplastic thorax (4) Radiographic evidence of HPP, characterized by:
- •Flared and frayed metaphyses 2) Severe, generalized osteopenia 3) Widened growth plates 4) Areas of radiolucency or sclerosis (5) Two or more of the following HPP-related findings:
- •History or presence of:
- •Nontraumatic post-natal fracture
- •Delayed fracture healing
- •Nephrocalcinosis or history of elevated serum calcium
- •Functional craniosynostosis
- •Respiratory compromise or rachitic chest deformity
- •Vitamin B6 dependent seizures
- •Failure to thrive
- •Premature tooth loss (6) Patient who have the mutation of tissue non-specific ALP gene
- •Parent or legal guardian(s) must provide written informed consent prior to any study procedures being performed and must be willing to comply with all study-required procedures
排除标准
- •Current evidence of treatable form of rickets
- •Serum calcium or phosphate levels below the normal range
- •Pregnant women and nursing mothers
- •Patient who cannot enforce suitable contraceptive measures during the clinical trial
- •Prior treatment with bisphosphonates
- •Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
- •Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
- •Clinically significant disease that precludes study participation, in the opinion of the Investigator
研究组 & 干预措施
Asfotase Alfa (ALXN1215)
Experimental
Asfotase Alfa (ALXN1215) is administered 6mg/kg in total per week, divided into 3 times.
干预措施: Asfotase Alfa (ALXN1215) (Drug)
结局指标
主要结局
Number of subjects with Adverse Events as an assessment of the Safety of repeated subcutaneous (SC) injections of asfotase alfa
时间窗: Up to 50 months or until regulatory approval
Safety of repeated subcutaneous (SC) injections of asfotase alfa for all treated patients
次要结局
- Profile of asfotase alfa treatment on respiratory function(Up to 50 months or until regulatory approval)
- Profile of asfotase alfa treatment on physical growth(Up to 50 months or until regulatory approval)
- Effect of asfotase alfa treatment on development(Up to 50 months or until regulatory approval)
- Effect of asfotase alfa treatment on skeletal manifestations of HPP as measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale(Up to 50 months or until regulatory approval)
- Overall survival(Up to 50 months or until regulatory approval)
- Effect of asfotase alfa treatment on ventilator-free survival: (percentage of patients who are alive and ventilator-free after receiving asfotase alfa)(Up to 50 months or until regulatory approval)
研究者
研究点 (12)
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