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.

该主题是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子弹和调查员（PI）可能已经从其他NIH来源获得了主要资金，因此可以在其他清晰的条目中代表。列出的机构是针对该中心的，这不是调查人员的机构。急性间米特斑岩是由Pophobilinogen脱氨酶（PBGD）的遗传缺陷引起的，这是生物合成途径中的第三个酶，导致Heme。这种新方法是通过现代分子生物学方法在生物实验室中进行的，是该酶，RHPBGD和Porphozym¿的准备。该试验的目的是将卟啉的效率和安全性与安慰剂的效率和安全性进行比较，以作为对急性发作受试者的AIP的治疗。安慰剂是一种与接受测试的药物相同的不活动材料。丹麦/瑞典公司Hemebiotech A/S将提供卟啉和安慰剂。在急性发作并签署研究同意书的情况下来医院后，您将接受卟啉或安慰剂的治疗。您将根据随机确定的预定顺序（例如翻转硬币）将您分配给治疗。因此，您将有50％的机会接受新疗法。您的医生和您都不会知道您将获得哪种治疗方法。在48小时的时间内将进行治疗中的血管（静脉）。您将受到关注，直到您出院。治疗结束后的14天28天，您将来门诊诊所进行后续访问。如果您在最后一次访问之前没有遇到新的攻击，那么您将遵循您的下一次攻击或6个月（以先到者为准）。