What is a clinical trial?
The World Health Organization’ (WHO) definition for a clinical trial is:
“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”.
The Australian Government clinical trials web site defines a clinical trial as:
“Clinical trials are research investigations in which people volunteer to test new treatments, interventions or tests as a means to prevent, detect, treat or manage various diseases or medical conditions. Some investigations look at how people respond to a new intervention* and what side effects might occur. This helps to determine if a new intervention works, if it is safe, and if it is better than the interventions that are already available.
Clinical trials might also compare existing interventions, test new ways to use or combine existing interventions or observe how people respond to other factors that might affect their health, such as dietary changes”
See Australian Government information on Australian Clinical trials.
What is the process of a clinical trial?
Drug development is a highly regulated and ordered process which involves a series of trial steps called “phases”.
Each phase is designed to address a different research question.
All new drugs must proceed through all phases in the order laid down.
The aim is to determine using a small number of people, a tolerated drug dose range by seeing how the body metabolizes the drug while evaluating side effects.
Evaluate drug safety and gathers preliminary data about effectiveness.
This phase is conducted after the drug has been shown to be effective in a small number of patients with the condition/disease being studied.
This phase of the trial usually involves a large number of patients.
It tests treatment effectiveness, monitors side effects and compares the product being trialed to any existing treatments to see which is better.
If the data is then sufficient the researchers may use it to apply to the licensing authorities for marketing approval.
Phase 4 trials are usually done after the drug has been approved by the therapeutic Goods Administration in Australia.
Long term risks, benefits and optimal use of the drug are studied.
The protocol includes:
- Specifications for the type of people who may participate.
e.g. Age, sex, disease type and stage, previous treatment etc.
- The medications and dosages to be used and the tests to be used to monitor the participants.
- The length of the trial.
Every patient entering a trail will be asked to give informed consent.
This will involve the doctor/researcher explaining all the important information about the trial to:
- Ensure the patient understands the risks and benefits of the trial.
- Ensure the patient understands that enrolment is voluntary.
- Ensures they understand that they can with draw at any time.
The patient then signs the informed consent form before they can participate.
Why participate in a clinical trial?
- Clinical trials are one of the extremely important steps in discovering new treatments for amyloidosis and other diseases.
- Clinical trials are also used to find new ways to detect, diagnose and reduce the risk of disease.
- Clinical trials show researchers what does and does not work in people.
- Clinical trials help researchers and doctors decide if the side effects of a new treatment are acceptable when weighed
against the benefits offered by the new treatment.
- Clinical trials are independently monitored and thus ensure high levels of quality control with in clinical trial units.
- Patients participating are closely monitored and may have to visit hospitals more often for tests than those undergoing standard treatment.
- Participants can choose to leave a clinical trial at any time for any reason.
Clinical trials do not always result in improved outcomes. They can result in unexpected outcomes which may be less favourable.
Types of clinical trials
In this type of clinical trial, a number of similar people are randomly assigned to two or more groups.
One group (experimental group) will receive the drug being tested. The second group (control group) will receive dummy or sham treatment known as a placebo or no treatment.
Researchers compare the outcomes of both groups.
In this trial neither the patients or the researcher/doctor know who is receiving the experimental drug or the placebo.
The placebo and the experimental drug are coded by a third independent party unknown to the researcher. This secret code will remain in place until the trial is completed or stopped because of severe adverse outcomes.
Trials are conducted using patients and researchers in a number of different treatment sites, often around the world.
Other types of clinical studies
In these studies, a number of people are recruited and followed up over time to gather data to answer the researchers particular question.
In these studies, data that has been collected from clinical records and previous studies is analyzed to answer new questions that the original study was not designed to address.
Finding a clinical trial or study for all types of amyloidosis in Australia
Click here for current or pending Clinical Trials in Australia.
Conducting a clinical trial is complicated. It requires cooperation between many people and organizations. These may include research sponsors, industry, pharmaceutical companies, academia, government, nonprofit organizations, clinical investigators, patients, physicians, and regulators.
Time, money, personnel, materials such as medical supplies, support systems and manpower are also essential plus a clear plan for completing the necessary steps to conduct the trial. At any point along the way to opening a trial there may be delays i.e. when an Ethics and Governance Committee want to check safety issues.
Often trials take much longer to the recruitment phase than was originally envisaged.
Even when a trial is open no one patient can be guaranteed that they will be recruited to take part. Each trial has strict inclusion and exclusion criteria as well as caps on numbers allowed to enroll.
A trial may be stopped if there are unexpected side effects or if the investigational product is not found to be of use in an “interim analysis”.
In the end it is important to know that most trials are gathering information before the true effectiveness of a drug is known and a patient may well not be “missing out” if they cannot be registered.
The AAN web site will endeavor to post on this website page when a trial for any type of amyloidosis in Australia is open for recruitment.
Most clinical trials are listed on the Australian New Zealand Clinical Trials Registry (ANZCTR).This registry has been established at the National Health and Medical Research Council (NHMRC) Clinical Trials Centre, University of Sydney, with funding from the NHMRC and New Zealand Health Research Council.The registry lists trials that have been approved and are recruiting in the full spectrum of therapeutic areas of pharmaceuticals, surgical procedures, preventive measures, lifestyle, devices, treatment and rehabilitation strategies, and complementary therapies. Patients can also ask their treatment team or GP if they are aware of any trials that could be available and appropriate to them.
Around the world
The web site Centrewatch site gives an overview of some of the trials and studies being conducted for most types of amyloidosis around the world.
Although most of these trials are not available in Australia, Australian patients may in the long run benefit from the results of many of them as they add to the knowledge about diagnosing and treating all types of amyloidosis.