PEGA-0435-005

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。肢端肥大症是一种极为罕见的由脑垂体肿瘤引起的疾病。垂体瘤引起生长激素分泌增加。这反过来又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IGF-1升高的结果。因此，肢端肥大症治疗的一个目标是控制IGF-1的水平。本研究的目的是更多地了解两种最有效的治疗肢端肥大症的药物：Pegvisomant和Sandostatin。在我们最近的肢端肥大症研究中，以往的经验表明，一些患者需要同时使用山多他汀和Pegvisomant来充分控制他们的全部症状。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。参与的患者必须是确诊为肢端肥大症的成年人。参与者必须之前接受过放射或手术治疗，没有完全控制他们的症状。孕妇也不宜参与。一些患者将单独接受批准的药物Somavert (Pegvisomant)，而另一些患者将同时接受Pegvisomant和批准的药物Sandostatin LAR Depot。第三组患者，已证明对桑多司他汀LAR治疗有良好反应，将继续服用桑多司他汀LAR，并作为安全对照组。治疗将随机分配，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。该研究还将测量每组患者的生活质量和对其体征和症状的感知，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