Volunteering to trial new treatments, interventions or tests

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.
Bowel Cancer Australia Research-Clinical-Trials 770new

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 doctors and nurses, 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 doctor 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 ie 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.

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, speak to to your specialist.
Please see below a list of bowel cancer clinical  and treatment trials.  For a list of global bowel cancer clinical trials, please visit Clinicaltrials.gov website.  For a list of Australian bowel cancer clinical trials please visit Australian Clinical Trials website.

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). 

The purpose of this study is to determine the maximum tolerated dose (which will be the dose recommended for a Phase 2 study), safety, tolerability and pharmacokinetic profile (study of movement of the drug within the body, including absorption and distribution) of the study drug, BNC101 when administered intravenously as a single agent or in combination with chemotherapy in patients with metastatic colorectal cancer who have failed at least 1 or 2 lines of chemotherapy.

CheckMate 142
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 current study is an investigation into the use of HA-Irinotecan in a Phase II single arm trial of FOLF(HA)iri plus cetuximab in irinotecan-naïve second line patients with KRAS wild type metastatic colorectal cancer. The study objectives are to confirm the safety and efficacy of FOLF(HA)iri plus cetuximab as second-line therapy in irinotecan-naïve metastatic colorectal cancer patients. 

To evaluate and compare the efficacy and safety of regorafenib versus placebo in subjects with colorectal cancer (CRC) after curative resection of liver metastasis and completion of all planned chemotherapy.

This is a Phase III, multicenter, open-label, three-arm, randomized study in participants with unresectable locally advanced or metastatic colorectal cancer (CRC) who have received at least two prior regimens of cytotoxic chemotherapy for metastatic disease. The study compares regorafenib, a standard of care therapy in this setting, to cobimetinib plus atezolizumab and atezolizumab monotherapy.

This study is a randomized, multi-center study that will compare the efficacy and safety of selective internal radiation therapy (SIRT) using SIR-Spheres microspheres plus a standard chemotherapy regimen of FOLFOX6m versus FOLFOX6m alone as first-line therapy in patients with non-resectable liver metastases from primary colorectal carcinoma.

Health Education Materials With or Without a Physical Activity Program
This randomized 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.

Anal cancer is a relatively uncommon disease and there is currently no standard chemotherapy treatment for patients with inoperable locally recurrent or metastatic disease. The aim of this phase II study is compare two well known and largely used chemotherapy regimens - Cisplatin plus 5-fluorouracil vs Carboplatin plus Paclitaxel. The result of this study will set a standard of care for this disease and provide useful information for future Phase III trials.

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.

This is a Phase II multicenter, randomized, parallel arms, double-blind study of RO5520985 to evaluate the efficacy and safety of RO5520985 in combination with oxaliplatin, folinic acid, and 5-fluorouracil (mFOLFOX-6) versus bevacizumab (Avastin) plus mFOLFOX-6 in patients previously untreated metastatic colorectal cancer.

Nindetanib (BIBF 1120)
The objective of this Phase III study is to evaluate the efficacy of nintedanib in patients with metastatic colorectal cancer (mCRC) after failure of previous treatment with standard chemotherapy and biological agents.

The incidence of rectal cancers is at 15,000 new cases per year in France of which 10 to 15% are locally advanced (T4bNxM0) at the moment of diagnosis. The rate of invaded resection margins (R1) for these locally advanced and fixed rectal tumours varies from 10 to 20%. The invasion of the resection margins triples the risk of local recurrence. In the absence of surgical treatment, the 5-year survival rate for patients having had pelvic recurrence of rectal cancer is lower than 4% whereas it varies from 35 to 40% in cases of curative resection. The care and management of locally advanced and fixed rectal tumours and pelvic recurrence of rectal cancer constitutes, therefore, in the absence of recommendation, a difficult therapeutic problem with great variability in the methods of care and management around the world. These variations in practice can be explained by structural and organizational differences, as well as cultural dissimilarities. With regards to the organization of its healthcare system, Australia is shown to be a leader as regards the care and management of locally advanced and fixed rectal tumours and pelvic recurrence of rectal cancer.

The main purpose of this study is to evaluate the progression-free survival (PFS) in patients receiving S 95005 + bevacizumab (experimental arm) or capecitabine + bevacizumab (control arm) as first-line treatment for metastatic colorectal cancer in patients non-eligible for intensive therapy.

The purpose of this study is to determine safety and anti-tumor immune activation generated by TG02 and Granulocyte macrophage colony stimulating factor (GM-CSF), first as monotherapy (Part I), thereafter in combination with the checkpoint inhibitor pembrolizumab (Part II), in patients with locally recurrent rectal cancer scheduled to have surgery.
Part I and Part II will include approximately 10 patients each. Part I and Part II are separate and independent sequential components of the study. Patients will only be able to participate in either the Part I cohort or Part II cohort.
Main objective of the study is to investigate safety and immune response after TG02-treatment.

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