Clinical trials are important for the testing of new treatments in disease. Clinical trials also can be used to collect patient samples to understand why treatments work or why they do not work. The RESELECT trial will identify and apply novel drug and cellular therapies to benefit patients with the high-risk Bone Marrow Failure Syndrome of relapsed Aplastic Anaemia or bone marrow failure after transplantation (Poor Graft Function – PGF). An initial trial of N-acetylcysteine and atorvastatin has been undertaken with 11 patients enrolled to date and samples collected from all patients. The trial continues to accrue at the anticipated rate, remains of high scientific value and is an important platform for international collaboration. Analysis of the samples will proceed once accrual has been fulfilled.
2020-2025 (Grant): The Australian Bone Marrow Failure Biobank. Professor Melissa Southey, Biobanking Victoria, Monash University. The establishment of the Australian Marrow Failure Biobank, led by Maddie Riewoldt’s Vision, is a collaboration ...
Read more2020-2023 (Grant): Evaluating Multidisciplinary Bone Marrow Failure Syndrome Care – a prospective observational clinical trial offering comprehensive diagnostic genomic evaluation, multidisciplinary case review and multidisciplinary clinical ...
Read more2023- 2025 (Fellowship): The inaugural Captain Courageous Fellowship. A preclinical trial of next generation gene editing for the prevention of bone marrow failure in Fanconi Anaemia. Dr Astrid Glaser at the Genome Stability Unit of St Vincent’s ...
Read more2018-2020 (Grant-in-Aid): Predicting malignant transformation of Bone Marrow Failure Syndromes using longitudinal targeted sequencing of peripheral blood and cell-free DNA (cfDNA). Associate Professor Piers Blombery, Peter MacCallum Cancer ...
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