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Clinical trials are major contributors to cystic fibrosis research and treatment development. There are lots of opportunities to take part, but what should you consider before you get involved? 


We explore what a clinical trial is, how they work and who can take part. We look at the potential benefits and risk and where you can find more information on clinical trials.


What is a clinical trial? 

The World Health Organisation (WHO) defines a clinical trial as: 

‘any research study that prospectively assigns human participants or groups of humans to one or more health-related interventions to evaluate the effects on health outcomes’

A clinical trial is a form of research that uses volunteers to test new treatments, intervention or tests that may be used to treat, manage, detect, or even prevent illness. These treatments or interventions could be new drugs, vaccines, medical devices, diets and more. Some clinical trials may look at a new drug and its side effects, others make look at existing treatments and others may investigate how new treatments compare with existing.  

Participants can be people without chronic illness or disease, or they can be people who live with chronic illness.  

Illustrated outline of a doctor talking to a patient.

Are there different types of clinical trials?  

According to Australian Clinical Trials, there are three types of clinical trials:  

  • treatment trials to test new treatments, new medicines or combinations of medicines; or other new therapies such as surgery, the use of new medical devices or new approaches to surgery 
  • diagnostic or screening trials to evaluate tests or procedures to diagnose and detect diseases or conditions 
  • prevention trials to test new ways to prevent disease including medicines, vaccines, vitamins, or changes to diet, lifestyle or behaviour 

Clinical trials of new drug treatments generally fall into one of three phases. According to Health Direct, the three phases are:  

  • Phase I trials involve giving a group of 20 to 80 healthy volunteers the drug to check that it doesn’t harm them. 
  • Phase II trials involve giving a few hundred people the new medicine to check that it is safe and that it might work for more people. 
  • Phase III trials involve giving a few hundred or a few thousand people the new medicine to see if it really does work and to see what side effects it has.  


How do clinical trials work? 

Clinical trials are not the first step in the research of a new treatment. Often, before engaging human participants, researchers will test new treatments or interventions via laboratory studies or sometimes on animals.  

If laboratory tests suggest a treatment or intervention could have a positive impact on the treatment of disease or illness, the next step may be a clinical trial where more information can be gathered about the safety and effectiveness of the treatments or intervention.  

Clinical trials are strictly regulated. In Australia, those regulations include ethics guidelines and codes of conduct.  

Every trial will have its own protocol which is the plan or set of rules that must be followed to ensure the trial is safe and that the results will be of substance. 

Things covered in the protocol include:  

  • How long the trial runs for 
  • What data or information will be collected 
  • Who is eligible to participate 
  • How the treatment or intervention being tested will be delivered 

There are several parts of clinical trials that will look different in every trial, including the use of control groups or placebos, randomisation, and whether a trial is single or double blind.  

Placebos are substances that have no effect that are used in clinical trials to help determine the effectiveness of the treatment being tested.  

Randomisation refers to the ways in which participants in clinical trials are randomly assigned to separate groups or treatments. In a randomised trial, neither the researcher nor the people taking part in the research know if they will receive the treatment being tested or the placebo. 

A single blind clinical trial is a type of trial in which the researcher knows which treatment the participant is receiving, but the participant does not know. A double blind clinical trial is a type of trial where neither the researcher nor the participant knows if they are taking the treatment being trialed or a placebo.  


Who can be take part in clinical trials? 

Clinical trials can involve lots of different people, old and young, and can include people with and without chronic illness or disease. Often, researchers need people who live with a particular chronic illness, like CF, to see how effective new treatments of interventions may be.  

Participation is voluntary, so it’s completely up to you and you can change your mind at any time if you decide that you no longer want to take part.  

Participation in clinical trials is limited to those who can give informed consent. To give informed consent, you must understand all the facts about the clinical trial, including any potential risks. 

There are benefits to taking part in a clinical trial, they include:  

  • Helping scientists and researchers build knowledge of CF 
  • Getting access to new treatments and interventions 

But there are risks too. They may include:  

  • Side effects of treatments or interventions 
  • The new treatment or intervention failing 


Where to find more information about clinical trials  

You can find more information via Australian Clinical Trials, a joint initiative between the National Health and Medical Research Council and the Department of Industry, Innovation and Science which provides information and resources about clinical trials. 


Where to find a clinical trial 

Cystic Fibrosis Australia has a clinical trial finder which features the latest open clinical trials. You can sign up to receive email alerts when CF-related trials are open and looking for people to participate. If you are interested in taking part in a clinical trial, speak to your clinic team about what may be the best option for you and things you should consider before getting involved.

This story was published in May 2022. If you would like to share your story, please contact us at We’d love to hear from you and so would the CF community.