US-based biopharmaceutical research and development firm CytRx has started an open-label, multi-centre Phase II clinical trial with aldoxorubicin to treat patients with unresectable glioblastoma multiforme (GBM), a deadly form of brain cancer.
The trial is designed to evaluate the preliminary efficacy and safety of aldoxorubicin in subjects with unresectable GBM whose tumours have progressed following prior treatment with surgery, radiation and temozolomide.
About 28 subjects will be enrolled in the trial, who will be given either 350mg/m2 (260mg/m2 doxorubicin equivalent) or 250mg/m2 (185 mg/m2 doxorubicin equivalent) of aldoxorubicin intravenously on day 1, and every 21 days thereafter until evidence of tumour progression, unacceptable toxicity or withdrawal of consent.
In the trial, which is being carried out at the John Wayne Cancer Center/Sarcoma Oncology Center and the Louisiana State University Health Sciences Center, tumour response will be monitored every six weeks by MRI until disease progression occurs.
Primary objective of the trial is to determine progression-free survival (PFS) and overall survival (OS), while the principal secondary objective is an assessment of the safety of aldoxorubicin in subjects.
The trial follows positive, confirmatory results from a preclinical study in which aldoxorubicin showed significant efficacy in the treatment of rapidly growing human brain (glioblastoma) cancer in the brains of animals.
In the preclinical trial, animals treated with aldoxorubicin had median survival of more than 63 days, compared with about 25 days for animals treated with doxorubicin or saline.
CytRx president and CEO Steven Kriegsman said: "We were highly encouraged by aldoxorubicin’s apparent ability to cross the blood-brain barrier, potentially creating a new approach to attacking brain tumours."
The company said it is on track with the development of aldoxorubicin for unresectable GBM, and is seeking to release preliminary results of the Phase II study in 2014.
If the results from the trial are positive, the company intends to submit breakthrough therapy designation with the US Food and Drug Administration (FDA) for expediting the marketing approval of the drug.
Image: Histopathological image of cerebral glioblastoma. Photo: courtesy of KGH.