Acute intermittent porphyria

MONDO:0008294

Acute intermittent porphyria is the most frequent and the most severe form of the acute hepatic porphyrias. It is characterized by the occurrence of neuro-visceral attacks without cutaneous manifestations.

Also known as: acute intermittent porphyria, AIP, HMBS deficiency, PBGD deficiency, UPS deficiency, hydroxymethylbilane synthase deficiency, porphobilinogen deaminase deficiency, porphyria, Chester type

5 clinical trials for this condition and its sub-types, 5 tagged with Acute intermittent porphyria itself.

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Where it sits in the disease tree

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Part of

↑ Acute disease (4756) ↑ HMBS-related hepatic porphyria (5)

Including sub-types (5) Tagged with Acute intermittent porphyria (5)

Trials to join now! 3 Not yet finished but already full! 1 Terminated 1

Porphyria prevention study halted early – little data to go on

Prevention Stopped early

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 …

Phase 2 • Sponsor: The University of Texas Medical Branch, Galveston • Aim: Prevention

Last updated Jun 27, 2026 09:00 UTC

Acute intermittent porphyria

Where it sits in the disease tree

Porphyria prevention study halted early – little data to go on