About Acute Intermittent Porphyria
Acute intermittent porphyria (AIP) is the most common acute hepatic porphyria, caused by mutations in the HMBS gene encoding hydroxymethylbilane synthase, resulting in haem biosynthesis dysfunction. Patients experience attacks of severe abdominal pain, neurological dysfunction, and autonomic instability triggered by factors including certain drugs, fasting, hormonal changes, and stress. Givosiran (Givlaari), an RNA interference therapy targeting ALAS1, is approved for recurrent attacks.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Acute Intermittent Porphyria trials.
Attack frequency in the prior 12 months (typically 2 or more) is the standard inclusion criterion for acute porphyria trials
Urinary aminolevulinic acid (ALA) and porphobilinogen (PBG) levels are required biomarkers — collect during both attacks and between attacks
Givosiran (Givlaari) is approved — trials may study alternative RNAi therapies, hemin formulations, or combination approaches
Drug interaction lists are critical — many common medications are porphyrinogenic and must be reviewed with trial coordinators
Patient Resources
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