Queen Square Centre for Neuromuscular Diseases


Nogo-A study (GSK1223249 in MND/ALS)

A Phase I, multi-centres, randomized, placebo-controlled, double-blind, single and repeat dose escalation of a drug to treat ALS

SponsorRoyal Free Hampstead NHS Trust
CIDr Richard Orrell
UK sitesLondon Queen square
Contact detailsr.orrell@ucl.ac.uk

GSK 1223249 is a new drug developed by GlaxoSmithKline. It targets a protein called Neurite Outgrowth Inhibitor (Nogo-A), which hinders neurone regeneration.

There is evidence of increased Nogo-A in muscle of people with MND. By blocking the effect of Nogo-A, the drug may be an effective treatment for the disease. The drug delays symptom onset and prolongs survival in a mouse model of MND.

The trial aimed to provide safety and tolerability information, together with biomarker and functional information. The study included:

  • an infusion of the drug or placebo
  • a muscle biopsy taken before and after the infusion
  • plus other monitoring assessments