A Phase I, multi-centres, randomized, placebo-controlled, double-blind, single and repeat dose escalation of a drug to treat ALS
|Sponsor||Royal Free Hampstead NHS Trust|
|CI||Dr Richard Orrell|
|UK sites||London Queen square|
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