NCT03581591已完成3 期
An Open Label Trial to Assess the Safety and Efficacy of Burosumab (KRN23), an Investigational Antibody to FGF23, in a Single Pediatric Patient With Epidermal Nevus Syndrome(ENS) and Associated Hypophosphatemic Rickets
Redwood Dermatology Sciences2 个研究点 分布在 1 个国家目标入组 1 人开始时间: 2018年1月31日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 1
- 试验地点
- 2
- 主要终点
- The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets
研究概览
简要总结
A 52 week, open label trial to assess the safety and efficacy of KRN23, an investigational antibody to FGF23, in a single pediatric patient with Epidermal Nevus Syndrome(ENS) and associated hypophosphatemic rickets
A 26 weeks extension to original study to monitor patient lab results for her safety.
详细描述
1.1 Primary Objective
The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets
1.2 Secondary Objectives
- The PD profile of KRN23 as assessed by changes from baseline over time 1,25(OH)2D, iPTH, Serum Calcium, TRP and TmP/GFR (the ratio of renal tubular maximum phosphate reabsorption rate to glomerular filtration rate)
- Changes in underlying skeletal disease/rickets as assessed by standard radiographs utilizing the Radiographic Global Impression of Change (RGI-C) rating scales
- Effects of KRN23 on biochemical markers of bone turnover that reflect rickets severity, alkaline phosphatase (ALP)
- Walking ability as assessed by 6-Minute Walk Test (6MWT)
- Patient/parent-Reported Outcomes as assessed by PROMIS and FPS-R rating scales
1.3 Exploratory Objective
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
- — 至 18 Years（Child, Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Patient has confirmed ENS by physician diagnosis
- •Patient has confirmed FGF23 elevations in the context of low serum phosphorous < 4.1 mg/dL
- •Patient able to tolerate KRN23 treatment
- •Have a corrected serum calcium level < 10.8mg/dL
- •Have an eGFR >60 ml/min
- •Must be willing in the opinion of the investigator, to comply with study procedures and schedule
- •Provide written informed consent by a parent after the study has been explained and prior to any research related procedures begin
排除标准
- •Concomitant use of active vitamin D (i.e calcitriol) and/or exogenous phosphate supplementation. Patient will be allowed OTC Vitamin D should levels drop below <20 ng/ml
- •The use or enrollment in studies using other investigational therapies including other monoclonal antibodies
- •Subject and their parent not willing or not able to give written informed consent
- •In the Investigators opinion, the patient may not be able to meet all the requirements for study participation
- •Patient has a history of hypersensitivity to KRN23 excipients that in the opinion of the investigator, places the patient at an increased risk of adverse effects
- •Patient has a condition that in the opinion of the investigator could present a concern for subject safety or data interpretation.
结局指标
主要结局
The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets
时间窗: 18 months
次要结局
未报告次要终点
研究者
研究点 (2)
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