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Clinical trial information

What is a clinical trial?

Clinical trials are medical research studies used to compare two or more treatments. They are typically set up to determine which of the two treatments is more safe and effective.

A clinical trial is typically conducted by dividing a group of participants into two groups, one of which is treated one way and the other group is treated in another way. A process of ‘randomisation’ is used to determine who is assigned to each group (see below).

After entry to the trial each participant is followed up in an identical fashion to allow the researchers to measure the results of each treatment.

Why are clinical trials important

Clinical trials provide the most convincing evidence that a treatment is safe and effective. Over the last 50 years medical science has progressed to a stage where clinical trials are regarded as essential for this purpose.

The reason that clinical trials are important is because they are virtually the only way to identify an effect of treatment from amongst the large amount of variability occurring in both the nature of their illness and their response to a treatment.

Most illnesses affecting human health progress in an unpredictable manner. The same condition may cause minor inconvenience in one person but progress rapidly and become incapacitating in another.

Similarly, a person’s health may worsen or improve for no apparent reason in the absence of any treatment.

Different people also respond differently to a treatment. Even a placebo (inactive/inert treatment), if promoted enthusiastically by a doctor, may influence people to report fewer symptoms and feel better.

This variation makes it difficult to measure the effect of a treatment. If any single individual is given a treatment and appears to recover it is typically impossible to be certain that the treatment was responsible for the improvement.

Examples of interventions that have been approved for use following clinical trials:

  • Cochlear implant (Bionic ear)
  • Stents for blocked coronary arteries
  • Human papilloma virus vaccine for prevention of cervical cancer

What are the benefits of participating in clinical trials?

  • You may help others by contributing to medical research
  • You may benefit from receiving the intervention if it is effective
  • You will obtain medical care
  • Taking an active role in your health

What are the potential harms of participating in clinical trials?

  • No medication, even those as simple as aspirin is without adverse effects
  • The intervention may not be effective
  • You may need to have more doctor visits than normal
  • You may be randomized to the placebo group

What type of clinical trial is STAREE?

STAREE is a large clinical trial conducted in the traditional manner of all large scale international trials. Amongst medical researchers it is referred to as a ‘randomised, double-blind, placebo-controlled, morbidity-mortality trial’.

Its essential features are:

  • RANDOMISATION: this means that the decision about whether any participant will receive the statin or the placebo tablets is decided by a process similar to rolling a dice. There is no way that anyone in the trial can choose whether to be in the statin or the placebo group. This is the best way to ensure that the group of participants taking the statin medication and the group taking the placebo medication are as nearly identical as possible.
  • DOUBLE BLINDING: this means that neither the participant nor the researchers know whether the participant is receiving statin or the placebo. Both are manufactured to look exactly the same in size, shape, taste and markings. Double blinding prevents preconceived ideas about a drug influencing the results.
  • PLACEBO CONTROLLED: when it is not known whether a treatment works at all, an inactive substance called a ‘placebo’ may be used as the comparison. Typically this is a tablet that appears identical to the treatment but contains lactose or another inactive ingredient.
  • MORBIDITY-MORTALITY: this means that the trial will study whether the treatment affects a person’s survival and their experience of major illnesses such as heart attacks, cancer and stroke.

What is a protocol?

A protocol is a document that sets out the details of the clinical trial and the methods to be followed. It includes information about the background and aims of the trial, the procedures for recruitment of participants and assessment of patient events, details about the interventions being investigated, the possible risks and benefits of the intervention, and data analysis. The protocol also specifies how the researchers will monitor and manage adverse patient events.

What is informed consent?

Informed consent is a process that involves all clinical trial participants. Potential participants are provided with details about the trial and what will happen to them during the trial. The aim of informed consent is to ensure that potential participants understand all of the likely benefits and risks so they can make a fully informed decision about participating. Participants are required to give written consent before joining the trial but they can withdraw their consent at any time during the trial if they wish to.

Can I leave a clinical trial after it has begun?

Participation in the trial is voluntary and you may choose to withdraw at any time.

Who funds or sponsors clinical trials?

Clinical trials may be funded by numerous organisations such as government granting bodies (eg. NHMRC, NIH), charities (eg. National Heart Foundation, Arthritis Foundation), pharmaceutical companies, universities and research institutions, and philanthropic organisations.

Who manages clinical trials?

Clinical trials are managed by large teams of scientists and doctors who work in universities and hospitals. All clinical trials have a Principal Investigator who oversees the trial. Nurses, data managers and other health care professionals also play a part in clinical trials.

What if there are side effects from participating in a clinical trial?

If the participant experiences illness or possible side-effects while in the trial they should seek advice from their usual doctor.

What is the data and safety monitoring board and how does it ensure participant safety?

The data and safety monitoring board (DSMB) is a group of doctors, statisticians and researchers who meet regularly to review the progress of a clinical trial. These people are experts in the conduct of clinical trials but they are independent of the organisations that are managing the clinical trial for which they are DSMB members. Their role is to monitor the safety of participants and the quality of the data. As described below, the DSMB can decide to stop a trial early in certain circumstances.

Why might a trial be stopped early

Sometimes a clinical trial may be stopped early. There are a number of reasons that this can happen:

  • If participants experience unanticipated and serious side effects
  • If evidence shows that the risks are outweighed by the benefits.
  • If evidence has shown that the intervention being tested is effective
  • If it is not possible to recruit enough participants
  • If results from other trials that answer the same research question become available

Who monitors the conduct of protocols?

Auditors

What are my rights as a research participant?

Before you agree to participate you have the right to information about what will happen during the trial. This includes all details about the intervention, examinations and tests that you will undergo, and what the possible risks and benefits of participating are. You also have the right to discuss the trial with your doctor or the trial researchers.

Once a participant has agreed to take part in the trial and signed the informed consent they still have the right to decline any tests or examinations. They are also still able to leave the trial at any time.

The trial has been approved by an ethics committee. What is that?

An ethics committee is an independent group of people who ensure that research is conducted according to the guidelines on Good Clinical Practice and the ethical principles of the Declaration of Helsinki. Ethics committees are comprised of medical researchers, health professionals, lay people, lawyers and pastoral care workers. In Australia all clinical trials must be approved by an ethics committee before they begin to recruit participants. STAREE has been approved by a number of different ethics committees whose contact details are listed below.

How do I contact the ethics committee?

You can contact the ethics committee if you have any questions or a complaint about the conduct of the trial. Any of the ethics officers below can be contacted by telephone:

Monash University Human Research Ethics Committee (MUHREC)Executive Officer,
Human Research Ethics,
Building 3E, Research Office
Monash University
Victoria 3800
Tel: 03) 9905 2052

RACGP Ethics CommitteeExecutive Officer, National Research and Evaluation Ethics Committee
Royal Australian College of General Practitioners, College House
1 Palmerston Crescent
SOUTH MELBOURNE, VIC 3205
Tel: (03) 8699 0481
E-mail: ethics@racgp.org.au

What happens when a clinical trial is over?

The results of any clinical trial are carefully analysed and reported in a number of ways including publication in the scientific and medical literature, presentation at conferences, and incorporation into guidelines for clinical practice.

Will my health information remain confidential and private?

The individual data collected for each participant will only be accessible to the researchers analysing the results of the trial. Identifying information will be removed before analysis. Results for individual participants are confidential and are not made available or reported. Furthermore, it is not possible to identify individual participants through the published results.

What should I consider before agreeing to take part?

There are many questions a person may like to consider before they agree to participate in a clinical trial. Some of these have been answered in this section, but they may also like to ask their doctor about the following things:

  • What is the aim of the trial?
  • Do I have the time and ability to commit to the trial?
  • What tests and examinations will I need to undergo?
  • How will this trial affect my daily life?
  • How long will the study last?
  • Will I be reimbursed for any expenses?
  • What happens if I suffer any adverse events during the study?
  • What happens to my information if I decide to leave the trial?
  • Will I be told about my own results?
  • Will I receive the results of the trial?
  • What are the possible benefits of being involved?
  • What are the possible risks of being involved?