| Summary: | <p><strong>Background:</strong> Recent basic and clinical evidence suggests amiloride may be neuroprotective in multiple sclerosis (MS) through the blockade of the acid sensing ion channel.</p> <p><strong>Objective:</strong> To examine the neuroprotective efficacy of amiloride in acute optic neuritis (ON).</p> <p><strong>Methods:</strong> 48 patients were recruited to a phase 2, double blind, single site, randomised controlled trial with scanning laser polarimetry (GDx) at 6 month as a primary outcome measure, optical coherence tomography (OCT), visual and electrophysiological secondary outcome measures. Participants aged 1855 years, ≤ 28 days of onset of first episode unilateral ON, were randomised to amiloride (10mg daily for 5 months) or placebo. (clinicaltrials.gov, NCT 01802489)</p> <p><strong>Results:</strong> ITT cohort consisted of 43 patients; 23 placebo, and 20 amiloride. No significant drug related adverse events occurred.<br/>No significant differences were found in GDx (p=0.840). Visual evoked potentials 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.</p> <p><strong>Conclusion:</strong> Amiloride has not demonstrated any neuroprotective benefit within this trial paradigm but future neuroprotective trials in ON should target the window of opportunity to maximize potential neuroprotective benefit.</p>
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