A nationwide trial led by nephrologist Professor Jonathan Craig of Flinders University will seek to compared to best approach to use of steroids in children being treated for nephrotic syndrome.
The OPEN (Optimising Prednisolone dosE in childhood Nephrotic syndrome) trial is being run by paediatric nephrology units across Australia and aims to compared intensive versus less intensive doses of steroid for relapses of nephrotic syndrome.
As chief investigator Professor Craig says the clinical trial will help improve the management of the condition that affects 1 in 50,000 Australian children annually.
“Corticosteroid therapy has been the gold standard of care for nephrotic syndrome since the 1960s, and while this does save lives, it comes with significant side effects including weight gain, behaviour changes, infections and impaired growth,” he said.
“Additionally, relapses are also very common, with children then requiring multiple doses of steroids. These doses essentially begin to accumulate overtime, further exacerbating these side-effects. While a cure is not possible, there is an urgent need to ensure we can optimise steroid dosages to effectively manage the disease, while minimising the burden to children and their families.”
“Recent clinical trials have confirmed what we give to children the first time they are diagnosed is the optimal dose, however what we don’t know yet is if there is the possibility of treating relapses with half the amount of steroids we currently use,” said Professor Craig.
“Using a randomised trial, we will compare the efficacy of a lower intensity corticosteroid treatment to the current standard with the hope this still leads to remission, and maintains remission.
“This approach has already shown promise in observational and small trial data, which demonstrates we could potentially halve the cumulative steroid dose and alleviate the associated side effects.”
The project ‘A randomised trial of intensive vs less intensive corticosteroids for children with nephrotic syndrome (OPEN trial)’ Backed by recently announced grant funding of almost $2 million from the Australian Government’s Medical Research Future Fund (MRFF), the clinical trial partners include Perth Children’s Hospital, University of Sydney, University of Queensland, The Royal Children’s Hospital Melbourne and Adelaide’s Women’s and Children’s Hospital.
The trial will also incorporate economic evaluations and patient-reported outcomes, co-designed with patient partners, to provide a comprehensive assessment of the treatment’s impact.
“The results of the OPEN trial will answer a critical and unanswered question and has the potential to transform care and outcomes for children with this rare and neglected disease worldwide,” said Professor Craig.