Race Oncology (ASX:RAC) has received human ethics approval for its open-label clinical trial of Zantrene (bisantrene dihydrochloride) in patients with extramedullary Acute Myeloid Leukaemia (AML) or high-risk Myelodysplastic Syndrome (MDS).
This company said the open-label Phase 1 trial with a dose-expansion Phase 2 stage will recruit up to 60 patients with extramedullary AML or MDS.
AML occurs when leukaemia spreads from the bone marrow and forms solid tumours in tissues such as the skin, breast, kidney, brain and others. A 2020 prospective positron imaging trial identified that up to 22 per cent of AML patients have the extramedullary form. Extramedullary AML patients have no clinically approved treatments and limited experimental treatment options, with many clinical trials explicitly excluding this difficult to treat form of AML.
MDS is a group of blood cancers that affect the production of normal blood cells in the bone marrow.
The trial primary endpoint will be a complete response (CR) and complete response with incomplete haematological recovery (CRi), with the clinical aim of bridging the patient to an allogeneic hematopoietic stem cell transplant, and safety and tolerability of the decitabine/Zantrene regimen.
Key secondary endpoints include safety and tolerability of Zantrene, overall and event-free survival, and the correlation of FTO expression or other biomarkers with response to treatment.
The trial is expected to take 36 to 40 months to complete with full patient recruitment over approximately 18 months.
Race chief medical officer Dr David Fuller said, “We are pleased to receive this first human ethics approval and expect to expand the study to more approved Australian sites in the coming months. In addition, based on positive feedback from European clinical key opinion leaders and supported by the proceeds of our recent share purchase plan, we look forward to adding additional trial sites in Europe.”