Genesis of a clinical trial

Dr Khasraw and Luke Garrick

To establish a clinical trial and successfully receive funding for academic research takes a considerable amount of effort and time. It requires collaboration across multi-disciplinary teams made up of experts in the fields of medical oncology, surgery, radiation oncology, biological research, quality of life research, statistics, study coordination and consumer involvement.

Here’s a step-by-step description of the genesis of a clinical trial – from identification of the problem to obtaining funding – in this case, for AGITG’s NABNEC trial.

Step 1: Which treatment for a rare GI cancer?

Dr Mustafa Khasraw had a patient with an unusual and rare histology that was difficult to interpret. It was not clear how to treat his patient. In the hope of receiving advice, he approached a number of experts, including some from overseas. No one had a clear answer.

Step 2: Literature review  

A search for information to guide the treatment management of his patients found a gap in knowledge.

Step 3: Publish in a journal

An article was written and published about the gap in treatment for patients with Neuroendocrine Carcinomas (NEC) in the ‘Expert Review of Anticancer Therapy’.

Step 4: Notification from the AGITG for Best New Concepts Symposium at the Annual Scientific Meeting

Dr Khasraw presented a concept at the AGITG Annual Scientific Meeting in 2013 for a single arm trial using Abraxane and Carboplatin to treat patients with NEC and to measure the response rate and other data. The audience responded positively to the concept; the overwhelming suggestion was to develop a randomised clinical trial.

Step 5: Further discussion

AGITG members went on to discuss the feasibility and design of a randomised clinical trial.

Step 6: Teleconference with the AGITG Upper GI Working Party

The AGITG’s Upper GI and Lower GI Working Parties are the working parties of the Scientific Advisory Committee. The working parties include medical oncologists, surgeons, radiation oncologists, statisticians, translational scientists, consumers and allied health practitioners.

The Upper GI Working Party focuses on cancers of the oesophagus, gallbladder, pancreas, stomach and liver. Dr Khasraw discussed with the Upper GI Working Party a randomised clinical trial. Based on the verbal feedback from this multidisciplinary group of peers, Dr Khasraw revised the concept to present to the AGITG Scientific Advisory Committee. The Working Party also recommended that, while applying for grant funding, Dr Khasraw undertake the single arm pilot study.

Step 7: Presentation to the AGITG Scientific Advisory Committee (SAC)

The SAC is the focal point for widespread discussion of research ideas. Its members, who are drawn from the AGITG and the NHMRC Clinical Trials Centre, are experts in the fields of medical oncology, surgery, radiation oncology, biological research, quality of life research, statistics, study coordination
and consumer involvement. Dr Khasraw presented to 20 members; questions were asked and a vote took place that encouraged Dr Khasraw to continue.

Step 8: A grant is written and submitted to the Victorian Cancer Agency

Unfortunately, the grant request is unsuccessful.

Step 9: A pilot study is started

Dr Khasraw commences a small single arm pilot study funded by the Unicorn Foundation, supported by Specialised Therapeutics Australia and a grant from Barwon Health Hospital. The research team’s objective is to establish a network of investigators, assess the capacity to do translational research, maintain enthusiasm and interest in the research and make a stronger case for an NHMRC grant application.

Step 10: Application for NHMRC grant submitted  

One year later the NABNEC application is successful and receives NHMRC funding and support from Specialised Therapeutics Australia.

Dr Khasraw is pictured above (right) receiving the Award for Best of New Concepts Symposium, 2013 from Luke Garrick, Specialised Therapeutics Australia.