The National Institute for Health Research Efficacy and Mechanism Evaluation (EME) Programme is funding a new £1.5 million trial testing a combination of therapies to prevent rapture in patients with brittle bone disease.
The six year study, led by the University of Edinburgh and involving 25 hospitals in the UK and one in the Republic of Ireland, will assess a regimen of teriparatide followed zoledronic acid versus placebo in 390 people with osteogenesis imperfecta.
The rare bone condition is caused by genetic mutations that lead to abnormalities in collagen, leaving people with extremely fragile bones that break easily.
Both teriparatide and zoledronic acid are established treatments for the bone-thinning disease osteoporosis but this is the first time they have been tested in combination as therapies for OI.
"This is potentially a game-changing trial since it is the first study that had been specifically designed to investigate whether any treatment can prevent fractures in osteogenesis imperfecta," noted Professor Stuart Ralston, of the University of Edinburgh's Centre for Genomic and Experimental Medicine. "If the results are positive, it could herald a new dawn in the treatment of this rare but devastating condition."