ACUTE INTERMITTENT PORPHOZYM

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。急性间歇性胰腺炎是由胆红素原脱氨酶(PBGD)的遗传缺陷引起的，PBGD是导致血红素的生物合成途径中的第三种酶。这种新的处理方法是在生物实验室用现代分子生物学方法进行的，是一种酶的制剂，即重组人PBGD，PorPhozym。这项试验的目的是比较Porphy zym和安慰剂作为急性发作患者治疗AIP的有效性和安全性。安慰剂是一种非活性物质，在外观上与接受测试的药物相同。丹麦/瑞典公司HepheBiotech A/S将同时供应PorPhozym和安慰剂。在急性发作来到医院并签署研究同意书后，您将接受PorPhozym？或安慰剂的治疗。你将被分配到一个基于随机决定的预定顺序的治疗(就像抛硬币一样)。因此，你将有50%的机会接受新的治疗。你的医生和你都不知道你将接受哪种治疗。治疗将在48小时内进入血管(静脉)。你会一直被跟踪，直到你出院。在治疗结束后14天和28天，您将来到门诊进行随访。如果您在这些访问的最后一次之前没有经历新的攻击，您将被跟踪到您的下一次攻击或6个月，以最先发生的为准。

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. Acute Intermittemt Porphozym is caused by an inherited defect of the enzyme porphobilinogen deaminase (PBGD), the third enzyme in the biosynthetic pathway leading to heme. The new treatment, which has been made in a biological laboratory by modern methods of molecular biology, is a preparation of this enzyme, rhPBGD, Porphozym¿. The purpose of the trial is to compare the efficacy and safety of Porphozym¿ with that of placebo as a treatment of AIP in subjects with acute attacks. The placebo is an inactive material identical in appearance to the drug undergoing testing. The Danish/Swedish company, HemeBiotech A/S, will supply both Porphozym¿ and placebo. After coming to the hospital with an acute attack, and signing the study consent form, you will be given treatment with either Porphozym¿ or placebo. You will be assigned to a treatment based on a pre-determined order decided at random (like flipping a coin). Therefore you will have a 50% chance of receiving the new treatment. Neither your doctor nor you will know which treatment you will receive. Treatment will be given into a blood vessel (a vein) over a period of 48 hours. You will be followed until you are discharged from the hospital. 14 days and 28 days after end of treatment you will come to the outpatient clinic for a follow-up visit. If you do not experience a new attack before the last of these visits, you will be followed until your next attack or for 6 months, whichever comes first.