Safety and Efficacy of Panhematin™ for Prevention of Acute Attacks of Porphyria

Who is this study for? Adult patients with Acute Porphyria
What treatments are being studied? Hemin
Status: Recruiting
Location: See location...
Intervention Type: Biological, Other
Study Type: Interventional
Study Phase: Phase 2

The purpose of this study is to determine if Panhematin is safe and effective for prevention of acute attacks of porphyria. The study aims to provide high quality evidence for the use on hemin for prevention of acute attacks of porphyria. High quality studies have not been done previously for treating or preventing acute attacks with hemin. The lack of strong evidence for efficacy of hemin for treatment and prevention of attacks limits its availability for patients with acute porphyrias. Funding source: FDA Office of Orphan Product Development (FDA OOPD) FD-R-03720

Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: No

• Male or female aged 18 years

• Willing to provide written informed consent

• A diagnosis of acute intermittent porphyria, hereditary coproporphyria or variegate porphyria confirmed by the following criteria, which are based on the criteria for enrollment in the Longitudinal Study of the Porphyrias Consortium. For each type of porphyria, the inclusion criteria are based on 1) clinical features, 2) biochemical findings, as documented by laboratory reports (or copies) of porphyria-specific testing, and 3) molecular studies to identify a mutation in a porphyria-related gene. Equivocal biochemical measurements may require confirmatory testing. Testing for a disease-causing mutation must be attempted, but an identified mutation is not essential for enrollment, since it is known that a mutation cannot be found in a small fraction (<5%) of biochemically proven cases of porphyria. Subjects will (1.) have had frequent attacks in the past, with symptoms such as abdominal, back and/or limb pain and diagnosed after exclusion of other causes, and (2.) be on hemin prophylaxis for prevention of frequent attacks. It is expected that they will have had 6 or more attacks in one year before starting hemin prophylaxis.

United States
University of Texas Medical Branch
Contact Information
Karl E Anderson, MD
Csilla Hallberg, MD
Time Frame
Start Date: October 30, 2015
Estimated Completion Date: September 2023
Target number of participants: 20
Experimental: Hemin for injection
Double blind doses of Panhematin 4 mg/kg body weight reconstituted with 25% human albumin and infused over at least one hour.
Placebo Comparator: Placebo
A double blind dose of saline.
Leads: The University of Texas Medical Branch, Galveston

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