What is a Clinical Trial?
A clinical trial is a research study designed to evaluate the success of a new treatment in an attempt to prevent, stabilise or reduce the effects of a disease. Clinical trials are particularly important when treatment options are limited, as is still the case for pulmonary fibrosis, and involve the voluntary participation of individuals. Each individual’s response to the treatment administered is monitored closely in a clinical trial. This research is then collected and used to further the development of these treatments, in turn, helping those affected by the disease now and in the future.
Here is a short video from the Australian Clinical Trials Alliance (ACTA) to explain more about clinical trials.
Overview of the Types and Phases of Clinical Trials
Types of Clinical Trials
- Treatment trials
Treatment trials test new:
- Medicines or combinations of medicines
- Medical devices
- Surgical techniques
- Other new medical and non-medical therapies.
- Diagnostic or screening trials
Diagnostic or screening trials evaluate tests or procedures to diagnose and detect diseases or conditions.
- Prevention trials
Prevention trials test new ways to prevent disease, including medicines, vaccines, vitamins, or changes to diet, lifestyle or behaviour.
Clinical Trial Phases
Phase 1
Phase 1 clinical trials:
- Test a new intervention for the first time
- Use a small number of people (between 20 and 80)
- Determine a safe dosage range
- Identify side effects.
Phase 2
Phase 2 clinical trials:
- Use a larger group of people, for example several hundred
- Determine efficacy; that is, whether it works as intended
- Further evaluate safety.
Phase 3
- Use large groups of people, from several hundred to several thousand
- Compare the intervention to other standard interventions, experimental interventions, standard care or a placebo
- Monitor adverse effects
- Collect information that will allow the intervention to be used safely.
Phase 4
- Happen after an intervention is in use
- Monitor the effectiveness of the approved intervention in the general population
- Collect information about any adverse effects associated with widespread use over longer periods of time
- May also be used to investigate potential use of the intervention in a different condition, or in combination with other therapies.
What is randomisation?
As described, there are different types of clinical trials, but one of the most common is the randomised controlled trial.
Randomisation in its simplest form is allocating a participant to a study group.
For further information about randomisation, please watch the following video produced by ACTA.
What is a Placebo and why is it important?
Most clinical trials include a placebo to help researchers more thoroughly determine the impacts of the experimental treatment. A placebo treatment is created to look identical to the research drug, but has no active ingredients. During a randomised clinical trial, some participants will be given the experimental treatment, while others will be provided with the placebo treatment. The participants and researchers will not know to which treatment ‘arm’ a person has been assigned. This eliminates the potential for bias. Having some participants on the placebo treatment and others on the experimental treatment helps researchers detect differences in any treatment effect, but also in side-effects, between the active and placebo.
Protecting the rights of Clinical Trial Participants
To ensure that the rights of individuals participating in clinical trials are protected, all trials are closely monitored and regulated by specialist committees such as Institutional Review Boards (IRBs) and Ethics Committees (ECs). These committees ensure that the rights and safety of participants are the utmost priority. As well as this, it is within a participant’s right to withdraw from a clinical trial at any point as it is a voluntary process.
How are Participants Selected?
Each clinical trial has strict eligibility criteria that details who can participate in the trial. Each group of participants will be chosen based on their shared characteristics. These characteristics or ‘inclusion criteria’ differ from trial to trial. Some eligibility criteria could include:
- Stage of the disease
- Medical history
- Age
- Lifestyle factors e.g. smoking/drinking
- Current medication
- Health status
Participant Responsibilities
When a patient is signed onto a clinical trial they have responsibilities that need to be respected. These will be clearly explained to the participant before the trial commences. These responsibilities may include:
- Disclosing any medication or lifestyle changes during the trial period
- Filling-out the necessary documents in a timely manner
- Limiting communication between participants so as not to interfere with the accuracy of the trial
- Attending each organised appointment