ACUTE INTERMITTENT PORPHOZYM

急性间歇性卟啉病

• 批准号: 7377337
• 负责人: HERBERT L BONKOVSKY
• 金额: $ 0.43万
• 依托单位: UNIVERSITY OF CONNECTICUT SCH OF MED/DNT
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2007-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7377337
• 关键词: ACUTE; INTERMITTENT; PORPHOZYM

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.

该子项目是利用 NIH/NCRR 资助的中心拨款提供的资源的众多研究子项目之一。子项目和研究者 (PI) 可能已从另一个 NIH 来源获得主要资金，因此可以在其他 CRISP 条目中得到体现。列出的机构是中心的机构，不一定是研究者的机构。急性间歇性卟啉症是由胆色素原脱氨酶 (PBGD) 的遗传缺陷引起的，PBGD 是血红素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通过现代分子生物学方法进行的，是这种酶 rhPBGD（Porphozym）的制剂。该试验的目的是比较 Porphozym 与安慰剂治疗 AIP 急性发作的疗效和安全性。安慰剂是一种非活性材料，其外观与正在测试的药物相同。丹麦/瑞典公司 HemeBiotech A/S 将提供 Porphozym¿ 和安慰剂。因急性发作而前往医院并签署研究同意书后，您将接受 Porphozym 或安慰剂的治疗。您将被分配到基于随机决定的预定顺序的治疗（如掷硬币）。因此，您将有 50% 的机会接受新的治疗。您的医生和您都不知道您将接受哪种治疗。将在 48 小时内对血管（静脉）进行治疗。我们将跟踪您直至您出院。治疗结束后14天和28天您将到门诊进行随访。如果您在最后一次访问之前未经历新的攻击，我们将跟踪您直至下一次攻击或 6 个月（以先到者为准）。