ACUTE INTERMITTENT PORPHOZYM

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。急性间歇性卟啉酶是由胆色素原脱氨酶（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。这种新的治疗方法是在生物实验室中通过现代分子生物学方法进行的，是这种酶rhPBGD，Porphozym？的制备。该试验的目的是比较Porphozym？与安慰剂治疗急性发作受试者AIP的疗效和安全性。安慰剂是一种外观与试验药物相同的非活性物质。丹麦/瑞典公司HemeBiotech A/S将提供Porphozym和安慰剂。在急性发作来到医院并签署研究知情同意书后，您将接受Porphozym？或安慰剂治疗。您将被随机分配到一个预先确定的治疗顺序（如抛硬币）。因此，您将有50%的机会接受新的治疗。你和你的医生都不知道你会接受哪种治疗。将在48小时内对血管（静脉）进行治疗。您将被随访至出院。治疗结束后14天和28天，您将前往门诊进行随访。如果您在最后一次访视前未发生新的发作，则将对您进行随访，直至您下次发作或随访6个月，以先发生者为准。

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.