New trial for people with brittle bone disease
- Published
X-ray of a baby with osteogenesis imperfecta
People with a rare bone condition that can cause them to have hundreds of fractures during their lifetime are being urged to trial a new treatment.
Edinburgh researchers have received £1.5m to conduct the study, which will involve 390 people with a condition called Osteogenesis Imperfecta (OI).
The six-year trial will test the combination of two therapies used to treat a different bone condition.
Researchers will track the participants for up to five years.
About half of the study's participants will be treated with a drug called teriparatide followed by treatment with another drug called zoledronic acid.
The other half will receive standard care.
OI is caused by genetic mutations that lead to abnormalities in a component of bone called collagen.
'Potentially game-changing'
People with the disease have extremely fragile bones that break easily, often from mild trauma or for no apparent cause.
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.
The study, led by the University of Edinburgh, will involve 25 hospitals in the UK and one in the Republic of Ireland.
It is being funded by The National Institute for Health Research Efficacy and Mechanism Evaluation (EME) Programme.
Prof Stuart Ralston, of the University of Edinburgh's centre for genomic and experimental medicine, said: "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.
"If the results are positive, it could herald a new dawn in the treatment of this rare but devastating condition."
Patricia Osborne, chief executive of the Brittle Bone Society, said "We have been supporting people with OI for 50 years and are pleased to see a potential new therapy being trialled that may improve the quality of peoples' lives".