An Open-Label Phase 3 Study of the Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Percent change of IGF-1 z-score from baseline to end of study (12 month visit).
研究概览
简要总结
Background:
For children with gigantism, too much growth hormone (GH) in the body causes abnormal growth and many other problems. Current treatments often don t work; no medical treatment is approved by FDA. Researchers want to see if the drug pegvisomant can help.
Objective:
To test the role of pegvisomant in children and adolescents with gigantism.
Eligibility:
People ages 2-18 with GH excess for whom usual treatments have not worked or who are not eliginle for them
Design:
Participants will be screened with a medical history.
The study will last 60 weeks and include at least 3 visits: baseline, 6-month, and 12-month visits. For the baseline visit, participants will stay a few nights for testing. They may stay overnight for the other visits.
All visits will include:
Medical history
Physical exam
Questionnaires
Heart and liver tests
Participants may be photographed in their underwear if they agree.
Blood tests: Participants will get a catheter: A small plastic tube will be placed in an arm vein. For some tests, the blood may be drawn every 30 minutes over 3 hours. For other tests, blood will be drawn every 20 minutes over 12 hours. Only clinically necessary tests will be done in each patient.
At the baseline visit, participants will have the study drug injected under the skin. They will learn to take the injection at home. They will take the injection daily during the study.
The baseline and 12-month visits will include:
MRI: Participants will have a dye injected into a vein. They will lie in a machine that takes pictures of the body.
Hand X-ray
Participants must get their height and weight at their local doctor s office monthly.
Participants must have blood and urine tests at their local lab monthly for the first 6 months then every 3 months until the study ends.
...
详细描述
Study Description:
GH excess is a rare and potentially lethal condition associated with hypersecretion of GH, usually by a pituitary tumor or hyperplasia. When it occurs prior to the complete fusion of growth plates, it leads to pathological tall stature, and it is called gigantism. After the fusion of the growth plates, it is called acromegaly. It may be associated with debilitating cardiovascular disease and/or diabetes. Children and adolescents with gigantism are currently treated with surgery, radiation therapy, and medications, such as octreotide, to reduce hypersecretion of GH; however, these treatments may lack efficacy and have significant side effects. Pegvisomant is a genetically engineered GH-receptor (GHR) antagonist that blocks the action of GH. In adults with acromegaly, pegvisomant has been shown to effectively reduce serum insulin-like growth factor type 1 (IGF-1) concentrations and lead to clinical improvement. However, experience in children and adolescents is limited to a small number of case series.,We propose the initiation of a new protocol at the NICHD, NIH, to treat children and adolescents with GH excess that is refractory to surgical therapy and/or radiation therapy, or in children and adolescents where the above therapies are contraindicated.
Objectives:
PRIMARY OUTCOMES:
- Percent change of IGF-1 z-score from baseline to end of study (12 month visit).
- Determine the safety and tolerability of pegvisomant in children and adolescents with GH excess.
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
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
1
Intervention arm, Patient received pegvisomant
干预措施: Pegvisomant (Drug)
结局指标
主要结局
Percent change of IGF-1 z-score from baseline to end of study (12 month visit).
时间窗: 1 year
The primary endpoint is decrease in IGF-1 z-score \>50% from baseline. This criterion will be used to determine efficacy.
Determine the safety and tolerability of pegvisomant in children with GH excess
时间窗: During 1 year
Safety will be determined by the periodical description of vital signs, laboratory and imaging studies, and other reported side effects.
次要结局
- Normalization of IGF-1 for age and sex from baseline to end of study (12 month visit)(1 year)
- Normalization of growth velocity(1 year)
- Improvement in signs and symptoms of GH excess and quality of life from baseline to end of study (12 month visit)(1 year)
- Left ventricular ejection fraction change on echocardiogram from baseline to end of study (12 month visit).(1 year)
- Reduction of the left ventricular mass index (LVMi) on echocardiogram from baseline to end of study (12 month visit)(1 year)