IIH:DT Trial


The IIH Drug Trial, or IIH:DT, is a randomised controlled trial of the drug AZD4017 vs placebo for treatment of Idiopathic Intracranial Hypertension. The trial recruited at the Queen Elizabeth Hospital, Birmingham, Walton Centre, Liverpool and Southern General Hospital, Glasgow.

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The IIH Drug Trial results have now been published!

Markey M, Mitchell J, Botfield H, Ottridge RS, Matthews T, Krishnan A, Woolley R, Westgate C, Yiangou A, Alimajstorovic Z, Shah P, Rick C, Ives N, Taylor A, C Gilligan L, Jenkinson C, Arlt W, Scotton W, Fairclough R, Singhal R Stewart P, Tomlinson J, Lavery G, Mollan S, Sinclair A. 11β-Hydroxysteroid Dehydrogenase Type 1 Inhibition in Idiopathic Intracranial Hypertension: A Double-Blind Randomized Controlled Trial Brain Communications, fcz050, https://doi.org/10.1093/braincomms/fcz050

The aim of the IIH:DT trial is to assess if the 11β-hydroxysteroid dehydrogenase type 1 inhibitor AZD4017 is an effective treatment for IIH, with reduction of ICP, visual symptoms and headaches.


IIH:DT was a double blind placebo controlled randomised (1:1) phase II trial to assess the efficacy, safety, and tolerability of AZD4017 400mg twice daily for 12 weeks in the treatment of IIH. Thirty one participants (against a target of 15 to each arm) were randomised.

Aim of the Study

The aim of the IIH:DT trial is to assess if AZD4017 is an effective and safe treatment for IIH, with reduction of ICP, visual symptoms and headaches.


Suitable patients were identified and randomised at NHS hospitals across the United Kingdom.

Target Population

Women aged 18-55 with active (papilloedema [Frisen grade ≥ 1], ICP >25 cmH2O) IIH and normal brain imaging.


Participants were randomised to AZD4017 400mg twice daily for 12 weeks or placebo.

Measurement of outcomes

The primary outcome measure is the change in intracranial pressure (ICP) between baseline and 12 weeks.

There are a battery of secondary and exploratory outcome measures that we will analyse to further investigate the safety and efficacy of AZD4017. Participants were followed up for 16 weeks.