Advocates from Save Our Sons Duchenne Foundation (SOS), in partnership with Parent Project Muscular Dystrophy (PPMD), have launched Australia’s first patient-led registry to increase clinical trial recruitment in Duchenne muscular dystrophy (DMD).
The initiative was inspired by PPMD’s DuchenneConnect patient registry, which since 2007 has provided data for planning clinical trials and research studies, and has helped recruit DMD patients. At present, 15 Duchenne treatments are being tested in U.S. clinical studies, with more drugs in preclinical development.
In Australia, where three such drugs are in the development stage, SOS advocates hope their new patient registry will make the country a more feasible place to conduct such trials. While DuchenneConnect allows anyone worldwide to join, the Australian portal will be specific to that region.
Both organizations encourage interested families to register in DuchenneConnect Australia in order to receive news and updates related to Australia and their local communities. The new “all-in-one” registry allows Duchenne-affected families to securely access their medical history anytime, anywhere — which is of particular importance in emergency situations.
To register, Duchenne patients or their caregivers can enter their medical information by completing a questionnaire online. The information will be safely stored; pharmaceutical firms, research institutions, policymakers and regulators can then apply to access that data.
SOS will manage and curate the registry under the guidance of a Registry Advisory Committee, consisting of key clinicians and Duchenne patient representatives. Australia’s Bellberry Human Research Ethics Committee has reviewed and approved the registry in accordance with the National Statement on Ethical Conduct in Human Research.
“Duchenne-specific data collected by the registry will help us to make big strides forward in improving Australian access to Duchenne clinical trials and emerging treatments, as well as improve care standards across the nation,” Kristina Elvidge, research affairs manager at SOS, said in a press release. “We are well aware that inequalities exist in local access to ‘gold standard’ care for Duchenne, and the registry will allow us to find gaps and take the appropriate steps to fill them.”
Some 30 boys are diagnosed with Duchenne each year in Australia, and between 600 and 900 Australian families live with the condition. Children diagnosed with Duchenne typically require a wheelchair by their early teens, and most do not live past their 20s.
Pat Furlong, PPMD’s founding president and CEO, says that the U.S. registry has proven an invaluable resource during the past 10 years, greatly assisting drug companies in planning trials and recruiting participants for these trials. “We hope that the launch of DuchenneConnect Australia will help make Australia a sought-after place to conduct Duchenne clinical trials,” she said.
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