Clinical Trial: Panhematin for Prevention of Acute Attacks of Porphyria Panhematin for Prevention of Acute Attacks of Porphyria

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Safety and Efficacy of Panhematin™ for Prevention of Acute Attacks of Porphyria

Brief Summary:

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. A separate study is in progress to evaluate the use of Panhematin for treatment of acute attacks of porphyria. Such 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.


Detailed Summary:

This is a double-blind, randomized, placebo-controlled, parallel group trial investigating the efficacy and safety of Panhematin™ for preventing acute attacks in at least 20 patients with well-documented acute porphyria (acute intermittent porphyria, hereditary coproporphyria or variegate porphyria). These patients 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 patients will have had 6 or more attacks in one year before starting hemin prophylaxis. This would be considered justification for a preventive regimen of hemin on clinical grounds. A single double blind dose of Panhematin™ or placebo will be given. An interim analysis will be carried out after completion of 10 patients to assess progress and possibly adjust the sample size. The trial consists of the following:

  • A screening visit to determine eligibility and obtain informed consent
  • A treatment visit for administration of a double blind prophylactic dose of Panhematin™ or placebo
  • Follow up visit at 1, 2, 3, and 4 weeks to assess response to the infusion of Panhematin™ or placebo. These visits will be in person or by telephone.
  • Additional visits may be scheduled if needed, for example for treatment of symptoms.
  • Follow-up visits 3 and 6 months after the end of treatment either in person or by telephone Patients will have laboratory documentation of one of the acute porphyrias. Molecular documentation is also expected, although rarely a causative mutation cannot be detected. Upon entry into the study they will be given in a blinded fashio
    Sponsor: The University of Texas Medical Branch, Galveston

    Current Primary Outcome:

    • Occurance of an acute attack of porphyria after treatment [ Time Frame: 1-4 weeks ]
      To evaluate in 20 patients who are on a Panhematin™ prophylactic regimen whether a blinded dose of Panhematin™ is more effective than placebo in preventing an attack within the next 1-4 weeks. The emphasis will be placed on prevention of attacks in the next week.
    • Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 1-4 weeks ]
      To evaluate in 20 patients whether hemin reconstituted with 25% human albumin is as safe and well tolerated as placebo when administered in a blinded fashion. Safety parameters will include the frequency and severity of phlebitis, nausea, vomiting and coagulation abnormalities.


    Original Primary Outcome: Same as current

    Current Secondary Outcome: Effects on levels of porphobilinogen [ Time Frame: 1-4 weeks ]

    To evaluate the biochemical effects of Panhematin™ in patients treated with Panhematin™ to prevent attacks of acute porphyria by measuring urinary porphobilinogen and serum porphobilinogen. This will determine whether biochemical measurements are predictive of efficacy in preventing an attack.


    Original Secondary Outcome: Same as current

    Information By: The University of Texas Medical Branch, Galveston

    Dates:
    Date Received: January 1, 2016
    Date Started: August 2015
    Date Completion: September 2020
    Last Updated: September 29, 2016
    Last Verified: September 2016