Porphyria prevention study halted early – little data to go on
NCT ID NCT02922413
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This small phase 2 trial tested whether the drug Panhematin could prevent acute attacks in people with certain types of porphyria. Only 13 participants were enrolled before the study was terminated early. Researchers tracked attacks and serious side effects, but the limited data makes it hard to draw firm conclusions.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Hemin (Panhematin) given intravenously
- What this could lead to
- If it worked, this could point toward a way to prevent painful acute attacks in people with certain porphyrias.
- What could go wrong
- The trial was terminated early and only enrolled 13 people, so results are very limited. It is unclear if Panhematin is truly effective or safe for prevention.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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13 people
The number who actually took part.
- Started
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Oct 2015
- Finished
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Dec 2023
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Male or female aged 18 years 2. Willing to provide written informed consent 3. 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. Exclusion Criteria: 1. Symptoms such as abdominal, back or limb pain are explained by another condition, as judged by the investigator 2. Known or suspected allergy to Panhematin™ or related products 3. A known or suspected allergy to human albumin 4. Any disease or condition that the investigator judges would lead to an unacceptable risk to the patient or interfere with the successful collection of data for the trial 5. Previous randomization in this trial
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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University of Texas Medical Branch
Galveston, Texas, 77555, United States
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