NCT00739505已完成1 期
A Multicenter, Open-Label, Dose Escalating Study of the Safety, Tolerability and Pharmacology of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 6
- 主要终点
- To determine the safety and tolerability of Asfotase Alfa given intravenously and given subcutaneously.
研究概览
简要总结
This clinical trial studies the safety, tolerability, and pharmacology of asfotase alfa when given to adults with HPP.
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 no approved disease-modifying treatments for patients with this disease. There is also 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
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •In order to qualify for participation, patients must meet all of the following criteria:
- •Patients must provide written informed consent, including privacy authorization, prior to participation.
- •Women of childbearing potential must sign the Women of Childbearing Potential Addendum and must be using an acceptable method of birth control. Women considered not of childbearing potential must be surgically sterile (total hysterectomy, bilateral salpingo-oophorectomy, or tubal ligation) or post-menopausal, which is defined as a complete cessation of menstruation for at least one year after the age of 45 years. All women must have a serum pregnancy test conducted at Screening prior to enrollment and the results must be negative.
- •Be between 18 and 80 years of age at the time of consent
- •Patients must be medically stable in the opinion of the Investigator.
- •Patients must be willing to comply with study procedures and the visit schedule.
- •Pre-established clinical diagnosis of HPP as indicated by:
- •a. Serum alkaline phosphatase at least 3 SD below the mean for age
- •b. Radiologic evidence of osteopenia or osteomalacia
- •c. Two or more HPP-related findings:
- •i. Plasma pyridoxal 5'-phosphate at least 2.5 SD above the mean (no vitamin B6 administered for at least 1 week prior to determination
- •ii. History of rickets
- •iii. History of premature loss of deciduous teeth
- •iv. Bone deformity consistent with osteomalacia or past history of rickets
- •v. History of any one of the following:
- •Non-traumatic fracture
- •Pseudofracture
- •Non-healing fracture
排除标准
- •In order to qualify for participation, patients must not meet any of the following criteria:
- •Women who are pregnant or lactating.
- •History of sensitivity to any of the constituents of the study drug.
- •Low levels of serum calcium, magnesium or phosphate.
- •Serum 25(OH) vitamin D level below 9.2 ng/mL.
- •Elevated serum creatinine or parathyroid hormone level.
- •Known cause of hypophosphatasemia other than HPP.
- •Current or prior clinically significant cardiac, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal condition and/or other major disease which, in the opinion of the investigator, precludes study participation.
- •Treatment with a bisphosphonate or parathyroid hormone (PTH) within 6 months prior to the start of Asfotase Alfa administration.
- •Participation in an interventional or investigational drug study within 30 days prior to study participation.
结局指标
主要结局
To determine the safety and tolerability of Asfotase Alfa given intravenously and given subcutaneously.
时间窗: Within the first 2 months (8 weeks).
次要结局
- To assess the pharmacokinetics (PK) of Asfotase Alfa given intravenously and subcutaneously(Within the first 2 months (8 weeks))
- To assess the bioavailability of the subcutaneous Asfotase Alfa(Within the first 2 months (8 weeks))
研究者
研究点 (6)
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