EUCTR2010-019850-42-DE进行中（未招募）1 期
An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase alfa (human recombinant tissue nonspecific alkaline phosphatase fusion protein) in Infants and Children =5 Years of Age with Hypophosphatasia (HPP)
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 100
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Patients must meet all of the following criteria for enrollment in this study:
- •- 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-related procedures. If the minor is in a position to comprehend the nature, significance, and implications of the clinical trial and to form a rational intention in the light of these facts, then he/she must provide his/her assent prior to any study procedures being performed.
- •- Documented diagnosis of HPP as indicated by:
- •o Total serum alkaline phosphatase (ALP) below the lower limit of normal for age
- •o Plasma PLP above the upper limit of normal (unless patient is receiving pyridoxine for seizures)
- •o Radiographic evidence of HPP at screening, characterized by:
- •* Flared and frayed metaphyses and
- •* Severe, generalized osteopenia and
- •* Widened growth plates and
- •* Areas of radiolucency or sclerosis
- •o 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
- •- Onset of symptoms prior to 6 months of age
- •- Chronological age of = 5 years or adjusted age for premature infants born = 37 weeks gestation
- •- Otherwise medically stable in the opinion of the Investigator and/or Sponsor
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 100
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients will be excluded from enrollment in this study if they meet any of the following exclusion criteria:
- •- Clinically significant disease, such as, but not limited to, hepatitis C virus (HCV) / human immunodeficiency virus (HIV) / hepatitis B virus (HBV), that precludes study participation, in the opinion of the Investigator and/or Sponsor
- •- Serum calcium or phosphate levels below the normal range
- •- Serum 25-hydroxy (25[OH]) vitamin D below 20 ng/mL
- •- Current evidence of treatable form of rickets
- •- 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)
- •- Intolerance to the study drug or any of its excipients
- •- Previous participation in the same study
- •- Close relation to the Investigator
研究者
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