Increlex Treatment of Children With Chronic Liver Disease and Short Stature
A major consequence of chronic liver disease in childhood is growth failure. This is because a chemical essential for growth called growth factor is created in the liver. Lack of response to growth hormone in people with chronic liver disease is characterized by high levels of growth hormone and low levels of growth factors. This growth hormone resistance is reflected in a variety of factors including insulin resistance and low nutritional intake. Unfortunately, growth hormone therapy has no effect for children with liver disease. In addition, failure of normal growth or malnutrition makes liver disease even worse in children, and growth hormone therapy is not likely to reverse this. A lack of proper nutrition is associated with hospitalizations and frequent complications. Poor growth is a predictor of poor outcomes after liver transplantation. Thus the management of children with liver disease remains a challenge. Children who have successful orthotopic liver transplants (OLT) show much improvement in some aspects of growth, including skin fold thickness, mid-arm circumference, and normalization of growth factor levels. However, some studies have recently reported that the growth of 15-20% of children remains poor even after a liver transplant. This can be explained by persistent abnormalities in growth factors after transplant.
Growth factor was found to be a good tool for prognosis in patients with chronic liver disease. Studies showed that patients with liver cirrhosis and growth factor levels below normal values showed lower long-term survival rates compared with patients who had above normal values. This suggests that growth factor can be a good predictor of survival and early marker of poor liver function. In this case, aggressive feeding may modestly improve growth factor levels leading to improved growth but it is unlikely that effects will be optimal. The investigators propose that growth factor administration may have a positive effect that leads to better growth which is a major predictor of good outcome. To date, no reports study the use of growth factor in children with chronic liver disease. This study proposes to examine the effect of growth factor therapy in childhood chronic liver disease.
研究概览
研究类型
阶段
- 不适用
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
Pre liver transplant patients with:
- Chronic liver disease
- Short stature (< 5%)
- Low IGF-1 (<-1SDS for age)
- Chronologic age 4-18 and bone age < 14 for boys and < 12 for girls (pre-pubertal)
Exclusion Criteria:
- Status post transplant
- Evidence of malignancy
- Diabetes mellitus
- Participation in other clinical trials involving investigational products
- Treatment with growth hormone within 3 months
- Pregnancy
- Significant abnormality in clinical results
- Hypoglycemic at baseline
- Allergic to benzyl alcohol
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
---|---|
其他:All patients will be treated with IGF-1 factors
Patients will serve as their own control.
|
Increlex therapy will begin at 40 micrograms/kg/day twice a day.
The dose will be escalated by 20 mcg twice a day every other week up to 100mcg/kg/week.
|
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
---|---|---|
Growth velocity is the primary outcome. Improved height SDS
大体时间:One year of therapy
|
Improved growth velocity with improved height standard deviation scores (SDS) is the primary expected result.
|
One year of therapy
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
---|---|---|
Improved BMI
大体时间:12 months
|
An improved body mass index is a secondary expected result of this study.
|
12 months
|
Improved quality of life
大体时间:12 months
|
An improved quality of life as assessed by the Pediatric Quality of Life Inventory forms is another expected result.
|
12 months
|
合作者和调查者
研究记录日期
研究主要日期
学习开始
初级完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计)
研究记录更新
最后更新发布 (估计)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他研究编号
- 07-03-018
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Increlex的临床试验
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Boston Children's HospitalAutism Speaks; International Rett Syndrome Foundation完全的
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Ipsen招聘中IGF1缺陷西班牙, 美国, 意大利, 法国, 英国, 德国, 奥地利, 法属圭亚那, 波兰, 瑞典, 马提尼克岛
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Children's Hospital Medical Center, CincinnatiIpsen; Charley's Fund完全的