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Amiloride does not protect retinal nerve fibre layer thickness in optic neuritis in a phase 2 randomised controlled trial

Research output: Contribution to journalArticle

  • Justin B. McKee
  • Charles L. Cottriall
  • John Elston
  • Simon Epps
  • Nikos Evangelou
  • Stephen Gerry
  • Christopher Kennard
  • Yazhuo Kong
  • Abigail Koelewyn
  • Wilhelm Kueker
  • Maria Isabel Leite
  • Jacqueline Palace
  • Matthew Craner
Original languageEnglish
Pages (from-to)246-255
Number of pages10
JournalMultiple Sclerosis Journal
Issue number2
Early online date27 Nov 2017
DateAccepted/In press - 20 Oct 2017
DateE-pub ahead of print - 27 Nov 2017
DatePublished (current) - 1 Feb 2019


Background: Recent basic and clinical evidence suggests amiloride may be neuroprotective in multiple sclerosis (MS) through the blockade of the acid sensing ion channel (ASIC). Objective: To examine the neuroprotective efficacy of amiloride in acute optic neuritis (ON). Methods: A total of 48 patients were recruited to a phase 2, double blind, single site, randomised controlled trial. Scanning laser polarimetry (GDx) at 6 months was the primary outcome measure and optical coherence tomography (OCT) and visual and electrophysiological measures were secondary outcome measures. Participants aged 18–55 years, ≤28 days of onset of first episode unilateral ON, were randomised to amiloride (10 mg daily for 5 months) or placebo (, NCT 01802489). Results: Intention-to-treat (ITT) cohort consisted of 43 patients; 23 placebo and 20 amiloride. No significant drug-related adverse events occurred. No significant differences were found in GDx (p = 0.840). Visual evoked potentials (VEP) were significantly prolonged in the amiloride group compared to placebo (p = 0.004). All other secondary outcome measures showed no significant difference. Baseline analysis of OCT data demonstrated a significant pre-randomisation thinning of ganglion cell layer. Conclusion: Amiloride has not demonstrated any neuroprotective benefit within this trial paradigm, but future neuroprotective trials in ON should target the window of opportunity to maximise potential neuroprotective benefit.

    Research areas

  • axonal loss, Clinical trial, multiple sclerosis, outcome measurement



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