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A double-blind, randomized, placebo-controlled, parallel group trial on the efficacy and safety of Panhematin(TM) in the treatment of acute attacks of porphyria

For Diseases:

  • Acute intermittent porphyria (AIP)
  • Hereditary coproporphyria (HCP)
  • Variegate porphyria (VP)

Background

How common are certain indicators of the severity of acute porphyria, including excess ammonia in the blood, developmental disabilities, and long-term kidney and liver effects?

What is the relationship between biological markers (for example certain lab tests) and the severity of acute porphyria?

Both glucose and hemin given by an infusion through the veins and are considered standard treatments for attacks of porphyria. Hemin has been on the market since 1983, and based on experience over the past 30+ years is believed to be the most effective treatment. But a good study to prove that it is effective has not been done, and this has impaired its acceptance by doctors and its availability to patients. This study is designed to provide the needed evidence that has not been available before.

The research aims are:

  1. To evaluate the effectiveness of glucose and Panhematin™ compared to glucose alone treatment for acute attacks of porphyria
  2. To evaluate the safety of glucose and Panhematin™, compared to glucose alone for acute attacks of porphyria

This is a clinical trial, which means its purpose is to study an intervention or treatment. It is also a randomized, controlled clinical trial, which means there is a treatment group given active drug and a control group given an inactive placebo. In this study, both groups are also treated with glucose, which is what the Food and Drug Administration (FDA) currently recommends before treatment with Panhematin™. The study will occur in the hospital, with planned follow up visits in the outpatient clinic or by telephone.

Patients are asked come to the hospital as soon as possible after symptoms of an attack begin, because treatment is more effective if started early. Some patients who have attacks that are regular and predictable may come even before symptoms begin. The treatment the patient is given will be hidden (blinded) by using drapes and opaque containers so that participants and most of the staff do not know which treatment they are receiving. Participants will also be asked to wear a blindfold during the infusion. At least one pharmacist and one study nurse will know what treatment the participant is receiving, so we can be sure the treatment is properly prepared and safely administered.

Before treatment is started patients are asked to rate their pain and other symptoms on a rating scale (zero to 10). These assessments are repeated many times during the trial. The amount of drugs needed for pain, nausea and other symptoms, and whether or not rescue treatment is needed will also be recorded.

Follow-up visits or phone interviews will be scheduled after you leave the hospital at 7-10 days, 6 months and 1 year.

Targeted Enrollment

To be eligible to participate, you must:

  • Be an individual with one on the following, documented by lab reports:
    • Acute intermittent porphyria (AIP)
    • Hereditary coproporphyria (HCP)
    • Variegate porphyria (VP)
  • Be having an acute attack of porphyria:

You are not eligible to participate if you have:

  • Your symptoms are explained by another condition (not porphyria), as judged by the doctors
  • You have been treated with Panhematin™ within the past 7 days
  • You have a known or suspected allergy to Panhematin™ or related products, or to albumin
  • You have another medical condition that the doctors think might be made worse by human albumin
  • You have another disease or condition that the study doctors judge would interfere with the study
  • You were in this trial previously and were randomized to treatment

In order to participate in a study, you must personally contact the study coordinator of the participating institution closest to you by phone or e-mail. Please use the information below to inquire about participation.

Texas

University of Texas Medical Branch, Galveston
Csilla Hallberg, MD, Coordinator
409-772-4661
E-mail: porphyria.center@utmb.edu