Clinical Trials Information

Clinical Trials

What are clinical trials and who can take part?

Have you considered taking part in a clinical trial?
Before you take part in a clinical trial, you need to understand what clinical trials are, why we need them, how they work, and who can take part.

You need to think carefully about taking part in a clinical trial and discuss your decision with your doctor.

What are clinical trials?
Clinical trials are research studies that involve people. They test whether new treatments can improve the health of people and how safe these treatments are. New treatments can include vaccines, medicines, medical devices such as pacemakers, and surgical treatments. Most treatments that we have today, including those for heart disease, diabetes and cancer, have been tested in clinical trials.

Why do we need clinical trials?
All new treatments need to be tested in clinical trials. This helps researchers to learn about how new treatments work in people, how safe these treatments are and what dosage should be used.

Clinical trials can also help researchers to compare new treatments with existing treatments in terms of how they work and how safe they are.

Why do people want to take part in clinical trials?
There are many reasons why people want to take part in clinical trials; below are some examples.

  • They may want the newest treatment because they hope that it will work better or have fewer side effects than their current treatment.
  • They may want the additional medical care they can get during a clinical trial.
  • There are people who have a disease for which there is currently no available treatment. Taking part in a clinical trial may be the only way for these people to get access to treatment.

How do clinical trials work?

Clinical trial phases
Before researchers study how a new treatment works in people during clinical trials, they need to
study how it works in a laboratory. Only the treatments that work well in a laboratory can then be
studied in people.

There are 4 main types of clinical trials, also called phases. Each phase is a different step in the
development of a drug and tests the new treatment in a different way.

The image below shows the main differences between the 4 clinical trial phases.

In rare diseases, such as SCN2A, the clinical trial phases can be combined or may overlap.

If you are thinking about taking part in a clinical trial, it is important to consider what phase the trial is. This is because each clinical trial phase has different benefits and risks. For example, in a phase 1 trial, researchers do not yet know what side effects a treatment may have and what dosage works best. You can find more information about the potential benefits and risks of taking part in a clinical trial later in this brochure.

How do clinical trials work?

Do all trial participants get the new treatment?
To find out if a new treatment improves participants’ health, researchers often give some participants a new treatment and others a placebo. A placebo looks like the new treatment but has no medicine in it. Researchers use a placebo to make sure that any of the effects they see in participants taking a new treatment are actually caused by the new treatment. 

In some clinical trials, instead of a placebo, researchers give some participants a treatment that is already available for their disease. This treatment is the one usually given to people to treat their disease and is called a standard treatment. By giving some participants a new treatment and other participants a standard treatment, researchers can study how well the new treatment works compared with the standard treatment.

In many clinical trials, a computer can be used to randomly decide which participants will receive the new treatment and which participants will receive a placebo or a standard treatment. This is done so that the treatments are chosen fairly and so that comparing the results of each treatment is as accurate as possible.

In some clinical trials, neither the participants nor the researchers know which participants got the new treatment and which participants got a placebo or a standard treatment until the end of the study. These types of trials are called double-blind trials. Researchers use double-blind trials because knowing what treatment the participants are receiving can affect the results of the trial.

Taking part in clinical trials

Potential benefits and risks
Before you can join a clinical trial, you need to understand all the possible benefits and risks
of and alternatives to the trial. To make sure that you understand this, the trial researchers will
explain the trial, and give you information to read and possibly questionnaires to complete.
If you have any questions about the clinical trial, ask the trial staff.
The table below shows some of the potential benefits and risks of taking part in a clinical trial.

Clinical trials are run by researchers and a medical team that includes doctors, nurses, pharmacists and other healthcare professionals. The clinical trial medical team is responsible for checking the health of trial participants during and for a period after the trial. If participants have serious or unexpected side effects from the trial treatment, the trial doctors will decide if the treatment dosage needs to be changed or if the participants need to stop taking their treatment.

Eligibility
The aim of each clinical trial is to answer a research question. Researchers choose clinical trial participants carefully to be able to best answer the research question.

For each clinical trial, researchers decide what traits the participants need to have or cannot have to take part in a clinical trial. The traits the participants need to have are called inclusion criteria.

The traits the participants cannot have are called exclusion criteria. These traits can include being a certain age or having a particular type or stage of disease.

If a person meets all inclusion criteria and none of the exclusion criteria for a clinical trial, they are considered eligible to take part in that trial.

Informed consent
Before you take part in a clinical trial, you will be asked to sign an informed consent form.

An informed consent form has information about the trial, such as its purpose, duration, and potential benefits and risks of taking part in the trial.

It is important that you read it carefully and make sure that you understand how the trial works and what is expected of you. You can take this form home and discuss it with your doctors, family and friends. By signing this form, you confirm that you have understood all information in the form. If you have any questions, ask the trial researchers.

Even if you have signed the informed consent form and joined a clinical trial, you can leave the trial at any time and for any reason! The trial researchers may remove you from the trial if they think that your health is getting worse.

Clinical Trials Information Sheet

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