Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

This is an interventional treatment trial for Acute Porphyrias focused on measuring Acute porphyria, Hemin Read More

Inclusion Criteria:

• Male or female aged 18 years
• Willing to provide written informed consent
• Acute symptoms (7 days duration or less to time of enrollment) such as abdominal, back and/or limb pain, diagnosed by the investigator as caused by porphyria after initial evaluation has excluded other causes.
• Diagnosis of acute porphyria documented by a substantial increase in urinary or serum porphobilinogen (PBG).
• Type of acute porphyria confirmed by additional testing (in addition to increased PBG), which may be completed before or after treatment begins using pretreatment samples:
• For acute intermittent porphyria (AIP): Normal or only slight increases in plasma and fecal porphyrins. Most (~90 percent) will have deficient activity of erythrocyte porphobilinogen deaminase (PBGD), and almost all (>95 percent) will have a demonstrable disease-causing PBGD mutation.
• For hereditary coproporphyria (HCP): Substantial increases in fecal porphyrins (almost entirely coproporphyrin III). In the absence of skin photosensitivity, most will have normal or only slight increases in plasma porphyrins. Almost all (>95 percent) will have a demonstrable disease-causing coproporphyrinogen oxidase (CPO) mutation.
• For variegate porphyria (VP): Substantial increases in fecal porphyrins (mostly coproporphyrin III and protoporphyrin), increased plasma total porphyrins and a fluorescence emission maximum of diluted plasma at neutral pH near 626 nm. Almost all (~95 percent) will have a demonstrable disease-causing protoporphyrinogen oxidase (PPO) mutation.

Exclusion Criteria:

• Symptoms such as abdominal, back or limb pain are explained by another condition, as judged by the investigator
• Therapy with hemin within 7 days prior to enrollment in this study
• Known or suspected allergy to Panhematin™ or related products
• Preexisting coagulation defect or concurrent treatment with an anticoagulant
• Previously documented renal impairment defined as a serum creatinine above 1.7 mg/dL or 150 mmol/L.
• A diagnosis of diabetes mellitus, which might increase the risk of glucose infusion.
• Heart failure, significant chronic anemia or any disease or condition that the investigator judges would lead to an unacceptable risk to the patient or interfere with the successful collection of date for the trial
• Previous randomization in this trial