What is a clinical trial? 
Clinical trials are carefully controlled research studies designed to look at very specific aspects of a single treatment, or to compare the effectiveness of several different treatments, to establish what is effective in treating a particular disease. They might be testing new medicines or treatment, or they might be looking at new ways of using current or older treatments to make them work better, or for different types of problems.

Are there different types of trials?

Diagnosing trials look at new technologies or ways of improving the way diseases are diagnosed in humans.

Prevention trials ask questions about how disease and illness can be prevented, and invite participants to do something that will prove or disprove the question being asked. For example, it might be an 'action' study: 'Does exercising three times a week reduce your risk of cancer?' Or it might ask a question about how effective an agent is: 'Does taking a certain vitamin reduce your risk of cancer?''.

Quality of life trials can measure an individual's sense of well-being and how your quality of life is affected by any given treatment. Screening trials test new methods of screening for cancer which may lead to more cases being diagnosed at an earlier stage.

Treatment trials look at new ways of treating a specific illness or symptom more effectively.

Clinical Treatment and Trials

Why are clinical trials needed in cancer? 

Clinical trials help to provide the proof that the medicines or therapies being used to treat patients are effective and safe. As well as testing new medicines, new ways of taking existing medicines or even different combinations of treatment, trials can also look at the consequences of treatments for people. They can assess and reduce the risk of unwanted side-effects of treatment by changing dosages or timing of the treatments.

Clinical trials will also consider the direct patient benefit, which arm of the trial (see below) showed the most benefit and also how the treatments affected quality of life both in a positive and negative way.

What are the different phases of treatment trials? 

Treatment trials go through a series of phases to test whether they are safe and if they work. All new cancer drugs are tested in the laboratory before they are given to people.

Phase 1 trials involve a very small number of people in a specialist research unit. They aim to discover what the most appropriate human dose might be, and what the side-effects are. If the drug is effective and a safe dose is found after a number of phase 1 trials, it will progress to phase 2.

Phase 2 trials still involving a small number of patients, aim to discover which types of cancer the treatment is going to be most effective for. They may also check the best way of giving the treatment, the optimum dose for effective treatment, and the side-effects and consequences of this pattern of treatment.

Phase 3 trials compare the effectiveness of the new treatment with current, standard treatment. This phase often involves much bigger groups of patients from many different hospitals, often in different countries. They can gather much more information on the effectiveness and side-effects of new treatments, and often last a year or more. They always involve 'randomisation'.

Phase 4 trials are carried out after a drug has been given a licence which proves it to be safe and effective for patients. These studies usually investigate the long term risks / side-effects of treatment in standard use. Phase 4 studies are not required for every medicine.

What is randomisation?

Randomisation is a way of allocating people to a certain treatment pathway (arm) within the trial including the new treatment or the standard 'best currently available' treatment. Occasionally, if there is no current treatment, a new treatment may need to be compared with a placebo (dummy treatment). Randomisation is done by a computer in order to avoid bias. Treatment arms are allocated according to various criteria i.e. gender, age, stage of disease to ensure that the different arms of the trial are as similar as possible. Each patient has an equal chance of being given the new treatment; it does mean you may not get access to the treatment being tested.

Should you take part in a clinical trial? 

There are a number of reasons why you may wish to take part in a clinical trial. They offer you the chance to:
  • access new treatments before they become widely available;
  • contribute to current medical knowledge and cancer research;
  • receive healthcare provided by leading clinicians in the field of cancer research;
  • have your health closely monitored during the trial.

If you are considering entering a trial, you should discuss it not only with your specialist(s), but with your family and friends. They may also have worries and concerns and it is helpful to have a different point of view when you are making such a big decision. You can leave the trial at any time, without giving a reason, if you change your mind.

However, if you have been receiving a new treatment it is unlikely that you would be able to continue with this. It is essential that you discuss this with your specialist before making a final decision.

It is important to be aware that if you enter a clinical trial you may be allocated to any of the arms. You therefore need to feel comfortable about receiving any of the treatments offered within the trial - i.e. the standard treatment or the new treatment.

What is informed consent?

Informed consent means being given all the information you need about the trial and the treatments being offered, before you decide whether or not to take part.
This is usually done by the dedicated research nurse or specialist who will be looking after you during the whole of the trial period. They will tell you about different aspects of the trial, including:
  • why the trial is taking place;
  • why the trial may be suitable for you;
  • what will happen to you during the trial;
  • whether or not it is a randomised trial and that you may or may not get the new drug or treatment being tested;
  • the standard treatment available if you do not want to enter the trial;
  • information on other treatment options available;
  • the possible risks, side-effects and benefits of the treatment;
  • how your progress will be monitored, whatever you decide.

Participation in research and clinical trials is entirely voluntary and you will be advised that you can withdraw at any time. You will be asked to complete and sign a consent form for the trial after you have had time to read the patient information sheet and ask questions.

You may also be asked to agree to additional tests being carried out on existing tissue samples that have been stored in the lab since they were taken during your original operation or biopsy. These samples will help scientists to better understand cancer and develop new treatments. In future, this will help to develop more successful and personalised treatments for bowel cancer patients.

You should only agree to take part in a trial if you are completely happy with what you are being asked to do. Your medical care and treatment will not be jeopardised in any way should you decide not to take part in a trial.

Placebo controlled trials 

A trial may use a placebo (dummy treatment) if it is trying to establish how effective a new treatment is when there is no comparative treatment available for any particular disease or symptom.

If you were taking part in a trial using a placebo, you would have an equal chance of being randomised between the new treatment arm and the placebo, which will look like the treatment being tested, but will not contain any active medicines. This is done on a 'blind protocol' - no-one in your specialist team will know which treatment you are having, so that they cannot unintentionally influence you or how you are feeling in any way. These kinds of trials could be looking to see if a particular treatment helps to prevent cancer coming back, for example.

Occasionally, a trial which is randomised to the standard treatment plus a new treatment may also use a placebo, where it is ethical to do so. An example might be a study to find out if some of the symptoms or complications of treatment could be prevented or treated more effectively by adding in a new medicine. In this case, you could be randomised to the standard treatment as a minimum, PLUS the new drug or the placebo.

Placebo controlled trials attempt to further remove any bias within the trial, to prove - or disprove - that there is a benefit, or any other difference, between the two types of treatment by comparing the results from both. By doing this with lots of different people, it may be possible to conclude that this difference is due to the new drug rather than other factors such as the power of positive thinking etc.

Are there any risks in taking part? 

Clinical trials involve a long and careful research process, but they do still carry potential risks, including:
  • side-effects or risks that have not been noticed previously;
  • the treatment may be less effective than standard treatment;
  • the new treatment may not work for you.

Your responsibilities 

If you are asked to fill in any questionnaires asking about how the treatment is affecting your quality of life, it is very important that you are able to do this. Your answers to the questions will help the researchers to understand if there are side-effects associated with the treatment which need to be considered when decisions are made about making the treatment more widely available.

Follow up 

Taking part in a clinical trial is not only about having the active treatment, but also to find out what happens to you in the months and years after the treatment has been completed. For this reason, it is likely that you will be asked to attend the hospital at more regular intervals. It is also possible that your tumour or biopsy samples may be used for some additional tests and investigations that you might not otherwise have had, or you may continue to have follow up appointments and assessments of your symptoms and quality of life for a much longer period of time than for a standard treatment pathway.

Clinical Trials
How do you get involved in a clinical trial?

Some people are invited to take part in a clinical trial by the medical team treating them. Other people actively seek to enter a clinical trial to potentially get access to treatment that is not currently available on the Pharmaceutical Benefits Scheme (PBS). If you want to find out more, talk to to your specialist.

Below is a list of bowel cancer clinical and treatment trials with study locations throughout Australia. A list of global bowel cancer clinical and treatment trials can be found on the clinicaltrials.gov website.

A Phase 1/1b, open-label, dose-escalation, and dose-expansion study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical activity of AB928 in combination with mFOLFOX in participants with advanced metastatic gastroesophageal Cancer (GEC) or bowel cancer.
Avelumab With Chemoradiation in Locally Advanced Rectal Cancer
This trial is investigating the inclusion of avelumab post long-course chemo-radiotherapy in patients with resectable locally advanced rectal cancer.

A phase III study in at least 440 patients with advanced bowel cancer to compare the efficacy of treatment with arfolitixorin versus Leucovorin in combination with 5-fluorouracil, oxaliplatin, and bevacizumab.
To assess the effectiveness of Aspirin against placebo control in patients with dukes C or high risk dukes B colorectal cancer in terms of Disease Free Survival (DFS) and Overall Survival (OS).
To evaluate the safety, tolerability, and pharmacokinetics of BLYG8824A and make a preliminary assessment of the anti-tumor activity of BLYG8824A in patients with locally advanced or metastatic bowel cancer.
A phase Ib, open-label platform study of select drug combinations chosen in order to characterise safety and tolerability of each treatment arm tested and to identify recommended doses and regimens for future studies.

To evaluate whether encorafenib (Braftovi) plus cetuximab (Erbitux), alone or in combination with chemotherapy, can improve clinical outcomes relative to current standard of care chemotherapy in participants with previously untreated BRAF V600E-mutant metastatic bowel cancer.

This trial aims to see whether the Nous-209 vaccine is safe to give to patients with Lynch syndrome, whether people are able to take the Nous-209 vaccine without becoming too uncomfortable, and how the immune system of patients with Lynch syndrome respond to the Nous-209 vaccine. This trial may help researchers determine whether receiving Nous-209 have an effect on the development of polyps or tumors in the colon.
This randomised phase III trial is studying a physical activity program given together with health education materials to see how well it works compared with giving health education materials alone for patients who have undergone treatment for high-risk stage II or stage III colon cancer.

The purpose of this study is to examine if Nivolumab alone or in combination with Ipilimumab will demonstrate a meaningful objective response rate in patients with recurrent and metastatic colon cancer who also have a specific biomarker in their tumors.
The main purpose of this study is to compare the clinical benefit, as measured by Progression-Free Survival (PFS), Objective Response Rate (ORR), and Overall Survival (OS), achieved by nivolumab in combination with ipilimumab or by nivolumab monotherapy in patients with Microsatellite Instability High (MSI-H) or Mismatch Repair Deficient (dMMR) metastatic bowel cancer.

The purpose of this study is to investigate treatment with Nivolumab in combination with Trametinib with or without Ipilimumab in patients with previously treated cancer of the colon or rectum that has spread. 

This prospective, cross-sectional, multi-centre, blinded clinical trial evaluates the performance of the ColoSTAT as an in vitro diagnostic test (IVD) for the early detection of bowel cancer.

A study to evaluate the safety and efficacy of standard of care (FOLFOX plus bevacizumab) alone and in combination with novel oncology therapies in first-line metastatic microsatellite-stable bowel cancer (MSS-CRC)
This study will evaluate the efficacy, safety, and pharmacokinetics of Trastuzumab deruxtecan (T-DXd) in participants with human epidermal growth factor 2 (HER2)-overexpressing locally advanced, unresectable, or metastatic bowel cancer (mCRC).

This study will determine the effect of the use of circulating tumour DNA (ctDNA) to guide adjuvant chemotherapy on recurrence-free survival in stage II colon or rectal cancer patients.
The aim of this study is to compare treatment informed by ctDNA results to standard care in patients with stage III colon cancer.

A study of efficacy and safety of Fruquintinib (HMPL-013) in patients with metastatic bowel cancer (FRESCO-2).
The purpose of this study is to determine if a commonly used radiological solution (Gastrografin) is a suitable option to help with the detection of anastomotic leakage, a serious complication of intestinal surgery.

A Phase I dose-escalation and dose-expansion study that will evaluate the safety, pharmacokinetics (PK), and preliminary activity of GDC-6036 in patients with advanced or metastatic bowel cancers with a KRAS G12C mutation. This study will determine the pharmacodynamically-active dose of gevokizumab and the tolerable dose and preliminary efficacy of gevokizumab in combination with the standard of care anti-cancer therapy in patients with metastatic bowel cancer, metastatic gastroesophageal cancer and metastatic renal cell carcinoma.

This is a phase Ib/II open label study. The escalation part will characterise the safety and tolerability of JDQ443 single agent and JDQ443 in combination with the other study treatments (TNO155 and spartalizumab) in advanced solid tumor patients. After the determination of the maximum tolerated dose / recommended dose for a particular treatment arm, dose expansion will assess the anti-tumor activity and further assess the safety, tolerability, and PK/PD of each regimen at the maximum tolerated dose / recommended dose.
In this study, participants with MSI-H or dMMR advanced colorectal carcinoma will be randomly assigned to receive either pembrolizumab or the Investigator's choice of 1 of 6 standard of care (SOC) chemotherapy regimens for the treatment of advanced colorectal carcinoma. The primary study hypothesis is that pembrolizumab will prolong progression-free survival (PFS) compared to current SOC chemotherapy.
The purpose of this study is to assess the safety and efficacy of lenvatinib (MK-7902/E7080) in combination with pembrolizumab (MK-3475) in participants with metastatic bowel cancer. The study will also compare lenvatinib plus pembrolizumab with the standard of care treatment of regorafenib and TAS-102 (trifluridine and tipiracil hydrochloride). The primary study hypothesis is that lenvatinib plus pembrolizumab is superior to standard of care with respect to overall survival.

The aim of this study is to assess the efficacy of delivering chemotherapy treatment through the arteries directly to the liver, bypassing the main blood supply throughout the body.

The purpose of this study is to determine the safety of an extracellular signal regulated kinase (ERK1/2) inhibitor LY3214996 administered alone or in combination with other agents in participants with advanced cancer (including bowel cancer).
The reason for this study is to see if the study drug LY3499446 is safe and effective in participants with solid tumors with KRAS G12C mutation.
The purpose of this study is to assess the safety and efficacy of coformulated favezelimab/pembrolizumab (MK-4280A) in participants with metastatic bowel cancer. The study will also compare MK-4280A with the standard of care treatment of regorafenib and TAS-102 (trifluridine and tipiracil).

A Phase II research project will test the efficacy, safety, and tolerability of an experimental drug combination: either nivolumab and BBI608 or nivolumab and BNC105 in patients with metastatic bowel cancer who have previously failed standard of care treatment.

A randomised phase II study of panitumumab monotherapy and panitumumab plus 5 fluorouracil as first line therapy for RAS and BRAF wild type metastatic bowel cancer. 
A phase Ib/II, open-label, multicentre, randomised study designed to assess the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of immunotherapy-based treatment combinations in patients with metastatic bowel cancer (mCRC) who experienced disease progression during or following two lines of treatment.
This study will determine the safety and tolerability and establish a preliminary recommended Phase 2 dose of V941(mRNA-5671/V941) as a monotherapy and in combination with pembrolizumab infusion.

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics and preliminary antitumor activity of MVC-101.
This is an international multi-center, prospective, open-label, randomised phase 3 trial of the cancer stem cell pathway inhibitor napabucasin plus standard bi-weekly FOLFIRI versus standard bi-weekly FOLFIRI in patients with previously treated metastatic colorectal cancer (CRC). 
Navarixin (MK-7123) in combination with Pembrolizumab (MK-3475)
The purpose of this study is to assess the efficacy and safety of navarixin (MK-7123) in combination with pembrolizumab (MK-3475) in adults with microsatellite stable (MSS) bowel cancer. 
This is an efficacy and safety study of olaparib alone or in combination with bevacizumab being compared to bevacizumab with Fluorouracil (5-FU) in participants with unresectable or metastatic bowel cancer (mCRC) who have not progressed following first-line induction of FOLFOX with bevacizumab.

A phase II randomised study to assess if oxaliplatin neurotoxicity be reduced with ibudilast in people with metastatic bowel cancer.

This study aims to determine the maximum tolerated dose (MTD) and assess the efficacy of panitumumab, irinotecan and trifluridine/tipiracil when given in combination for participants with RAS/BRAF wild-type (WT) metastatic bowel cancer.
Phase 1/2 Study of LOXO-292 in Patients With Advanced Solid Tumors (including colon cancer)
This is a Phase 1/2, open-label, first-in-human study designed to evaluate the safety, tolerability, pharmacokinetics (PK) and preliminary anti-tumor activity of selpercatinib (also known as LOXO-292) administered orally to patients with advanced solid tumors (including colon cancer).

This study is a Phase Ib, multi-center, open-label study of TNO155 in combination with spartalizumab or ribociclib with a dose escalation part followed by a dose expansion part in adults with advanced solid tumors. These two treatment arms will enroll people in parallel to characterise the safety, tolerability, PK, PD and preliminary antitumor activity. The study treatment will be administered until the subject experiences unacceptable toxicity, progressive disease, and/or has treatment discontinued at the discretion of the Investigator or the subject, or due to withdrawal of consent.

The aim of this study is to compare the use of robotic surgery with laparoscopic surgery for the surgical treatment of right sided colon cancer.
A randomized, placebo-controlled phase II trial of Simvastatin in addition to standard chemotherapy and radiation in preoperative treatment for rectal cancer.
Phase III study in first-line treatment of patients with metastatic bowel cancer who are not candidate for intensive therapy. The main purpose of this study is to demonstrate the superiority of S95005 in combination with bevacizumab over capecitabine in combination with bevacizumab.

This study is an open-label, multi-centre, dose-escalation, dose expansion study in adults with advanced solid tumors. The study will evaluate the safety, tolerability, PK, and preliminary anti-tumor efficacy of SM08502 administered orally, once daily, following a 5 days on 2 days off treatment schedule in combination with chemotherapy or hormonal therapy.

An open-label, multicenter, global Phase 2 basket study of entrectinib (RXDX-101) for the treatment of patients with solid tumors that harbor an NTRK1/2/3, ROS1, or ALK gene fusion. Patients will be assigned to different baskets according to tumor type and gene fusion.
In this study, participants with multiple types of advanced (unresectable and/or metastatic) solid tumors who have progressed on standard of care therapy will be treated with pembrolizumab (MK-3475).
The goal of this study is to evaluate the safety of intrahepatic injection (directly into the liver) of talimogene laherparepvec in combination with intravenously administered atezolizumab in patients with bowel cancer with liver metastases.

This study aims to compare the quality of life and quality adjusted life years of surgical versus non-surgical recurrent rectal cancer patients.

The purpose of this study is to collect additional safety and efficacy data during treatment with trifluridine / tipiracil in patients with a pretreated metastatic bowel; cancer (mCRC). Eligible patients may receive an early access to trifluridine / tipiracil through this clinical study until progression of disease, unacceptable toxicity, investigator decision, patient refusal or until market authorisation or reimbursement has been granted by the relevant Authority of the country where that patient is treated or until trifluridine / tipiracil is available by a doctor's prescription or can be accessed from another source or Sponsor decision. 

Related Information

Share your colonoscopy experience

My Colonoscopy Experience

Bowel Care Nurses

Bowel Care Nurses

Order a Screening Test

BowelScreen Australia