PEGA-0435-005

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。肢端肥大症是由脑下垂体肿瘤引起的一种极其罕见的疾病。脑下垂体瘤导致生长激素分泌增加。这反过来会导致胰岛素样生长因子1(IGF-1)水平升高。研究表明，肢端肥大症的大多数症状和健康问题都是由于IGF-1增加所致。因此，肢端肥大症的治疗目标之一是控制IGF-1的水平。这项研究的目的是了解更多关于治疗肢端肥大症的两种最有效的批准药物：聚乙二醇胺和善得定。在我们最近的Acromegaly研究中，先前的经验表明，一些患者需要联合使用Sandostatin和Pegvisomant来充分控制他们的所有症状。这两种药物的作用方式不同，但对一些肢端肥大症患者都有效。这项研究将有助于确定联合治疗的安全性和耐受性。要参与，患者必须是被诊断为肢端肥大症的成年人。参与者必须以前接受过放射治疗或手术，但他们的症状没有完全控制。孕妇也不应参与。一些患者将单独接受批准的药物Somvert(Pegvisomant)，另一些患者将同时接受批准的药物Sandostatin LAR Depot的Pegvisomant。第三组患者对Sandostatin LAR治疗显示出良好的反应，将继续服用Sandostatin LAR，并作为安全对照组。治疗将被随机分配，就像抛硬币一样。在为期40周的研究中，参与者将每月抽血，研究将检查每种方案的安全性和耐受性。这将通过检查每组不良事件的数量和类型来衡量。这项研究还将测量每组患者的生活质量和对他们的体征和症状的感知，以及控制IGF-1的有效性

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Acromegaly is an extremely rare disease caused by a tumor in the pituitary. The pituitary tumor causes increased secretion of Growth Hormone. This in turn leads to elevated levels of Insulin-Like Growth Factor 1 (IGF-1). Research indicates that most of the symptoms and health problems experienced in Acromegaly are a result of increased IGF-1. Thus, one goal of acromegaly treatment is to control levels of IGF-1. The purpose of this study is to learn more about the two most effective approved medications for treatment of acromegaly: Pegvisomant and Sandostatin. Previous experience in our most recent Acromegaly study showed that some patients needed a combination of both Sandostatin and Pegvisomant to adequately control their full range of symptoms. The two medications work in different ways, but both are effective for some acromegaly patients. This study will help determine the safety and tolerability of combination therapy. To participate, patients must be adults diagnosed with acromegaly. Participants must have been previously treated with radiation or surgery, without full control of their symptoms. Pregnant patients should also not participate. Some patients will receive the approved medication Somavert (Pegvisomant) alone, and others will receive both Pegvisomant the approved medication Sandostatin LAR Depot. A third group of patients, who have demonstrated a good response to Sandostatin LAR therapy, will continue taking their Sandostatin LAR and serve as a safety control group. Treatment will be assigned randomly, like flipping a coin. Participants will have monthly blood draws during the 40-week study, and the study will examine the safety and tolerability of each regimen. This will be measured by examining the number and type of adverse events in each group. The study will also measure quality of life and patients' perceptions of their signs and symptoms in each group, as well as effectiveness in controlling IGF-1