Efficacy and safety of 11b-HSD1 inhibitor in IIH

• Research type

  Research Study

• Full title

  Assessing the therapeutic efficacy and safety of an 11ß-hydroxysteroid dehydrogenase type 1 inhibitor (AZD4017) in idiopathic intracranial hypertension (IIH).

• IRAS ID

  138910

• Contact name

  Alexandra Sinclair

• Contact email

  a.b.sinclair@bham.ac.uk

• Eudract number

  2013-003643-31

• ISRCTN Number

  N/A

• Research summary

  Assessing the safety and effectiveness of a 11β-hydroxysteroid dehydrogenase type 1 inhibitor (AZD4017), in a placebo controlled trial, in acute idiopathic intracranial hypertension (IIH).
  IIH is a condition of young, overweight women with characteristic raised intracranial pressure (pressure around the brain) leading to papilloedema (swelling of the nerve supplying the eye), visual loss and headaches. Medical literature (Cochrane review) demonstrates there is little evidence for the current treatments used for IIH. Weight control appears the most effective method of improving symptoms but weight loss is difficult to maintain. 11β-hydroxysteroid dehydrogenase type 1 (11β-HSD1) is an enzyme which regulates local steroid levels and our previous research suggests it may influence the production of brain fluid(cerebrospinal fluid or CSF). 11β-HSD1 levels fall with weight loss and this is associated with with decreased intracranial pressure.
  Our primary outcome is to determine whether AZD4017, an inhibitor of 11β-HSD1, will reduce the pressure in the brain and as a consequence improve IIH. Patients are eligible to enter the study if they are between 18-55 years old with acute (<3 months) IIH, signs of active disease (papilloedema and raised CSF pressure (>25 cmH20)), no other major illnesses and have no plans for pregnancy during the study period.
  This is an MRC funded single centre, phase II, double-blinded, randomised control drug trial. It will be conducted at the University Hospital Birmingham and the University of Birmingham will act as Sponsor.
  Eligible participants will be randomly assigned to AZD4017 or a placebo (’dummy’ with no active drug) for 3 months with a follow up a month later. Investigations during the study will include bloods, urine samples, pregnancy tests, lumbar punctures, DXA scans and small fat/skin biopsies. Participants will benefit from increased monitoring and a potential improvement in their condition

• REC name

  Yorkshire & The Humber - Leeds West Research Ethics Committee

• REC reference

  13/YH/0366

• Date of REC Opinion

  19 Nov 2013

• REC opinion

  Favourable Opinion