PHASE I STUDY OF PERFENIDIONE IN NF1 AND PLEXIFORM NEUROFIBROMAS

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。1型神经纤维瘤病是一种常染色体显性进行性遗传病，临床表现多样。NF1患者发生中枢和周围神经系统肿瘤（包括丛状神经纤维瘤）的风险增加。这种生长可能导致严重的发病率和可能的死亡率。本研究是一项标准的i期研究，评估吡非尼酮的毒性，并根据药代动力学数据确定吡非尼酮的剂量与成人研究中使用的剂量相当。I期研究结束后，将立即开展II期研究，评估吡非尼酮对3岁和21岁进行性网状神经纤维瘤和NF1患儿的疗效。

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. Neurofibromatosis type 1 is an autosomal dominant progressive genetic disorder characterized by diverse clinical manifestations. Patients with NF1 have an increased risk of developing tumors of the central and peripheral nervous system including plexiform neurofibromas. Such growth may cause severe morbidity adn possible mortality. This study is a standard Phase 1 study evaluating the toxicity of Pirfenidone and determining, using pharacokinetic data, a dose of Pirfenidone equivalent to the dose being used in adult studies ofa the agent. It si planned that immediatly following the Phase I study, a Phase II study will be undertaken evaluating the efficacy of Pirfenidone in children 3 and 21 years of age with progressive plexiform neurofibromas and NF1.