ACUTE INTERMITTENT PORPHOZYM

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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.