About Clinical Trials

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About Clinical Trials

Choosing to volunteer or to let your child participate in a clinical trial is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.

Before you decide to participate, or to let your child participate, it is important for you to understand why the research is being done and what it will involve.

A clinical trial, also called a study, is a scientific way to test medicines to help determine if they are safe and if they work to treat people with diseases.

The drug that is the subject of a clinical trial is called an investigational medicine because its effects on study participants are still being investigated, and because it has not yet been approved for sale by regulatory authorities, like the United States Food and Drug Administration (FDA), Health Canada, European Medicines Agency (EMA) and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA).

In a clinical trial the effects of the investigational medicine on all study participants are measured, and those measurements are collected and analyzed to help determine how the medication may affect other people who may take it in the future.

Before a drug can be approved for sale, it must be investigated in clinical trials to show that it is safe and that it works.

Clinical trial research is done according to a plan called a protocol. The protocol is a guide carefully designed to answer specific research questions and to protect the participating patients’ health. The protocol describes who can participate in the trial, the tests, medications and procedures that are part of the trial, the length of the study and what information will be collected.

There are specific guidelines in the clinical trial protocol for who can and cannot participate called Inclusion & Exclusion Criteria. Inclusion criteria allow someone to participate while Exclusion Criteria exclude or do not allow participation. Participants are selected based on whether their age, gender, type and stage of disease, previous treatment history and other medical conditions fit with the guideline requirements for the study.

Clinical trials are part of clinical research and are at the heart of medical advances. Clinical trials look at new ways to prevent, detect or treat diseases. Through clinical trials, we can get a better understanding about the safety and efficacy of new drugs and therapies.

Anyone interested in participating in a clinical trial should know as much about the study as possible, and feel comfortable asking the doctor and research team questions about the study.

Participation in a trial is always voluntary. Whether you or your child takes part in a clinical trial is up to you. No one has to be part of a clinical trial, and patients can elect to stop participating at any time without compromising standard medical care.

Clinical trials involve risks, just as routine medical care and the activities of daily living.

Most clinical trials pose the risk of minor discomfort that lasts only a short time. However, some study participants experience complications that require medical attention. The specific risks of a clinical trial are described in the informed consent document.

Potential risks to participating in clinical trials include the following:
There may be unpleasant, serious or even life-threatening side effects to experimental treatment. The study may require more time and attention than standard treatment would, including visits to the study site, more blood tests, more treatments, hospital stays or complex dosage requirements.

While clinical trials have risks, there is a lot that goes on “behind the scenes” to ensure safety before participants are invited to be in a study and while the study is being conducted. Many people review a study protocol in detail before a single person is enrolled, including doctors, nurses, pharmacists, scientists, statisticians and other medical investigators. Each of these people is focused on ensuring that the study is done the right way and with the right participants. Most trials are reviewed by Institutional Review Boards (IRBs), independent committees that review and monitor the study before it starts and while it is being conducted. In addition, each study has a research team responsible for running the study and making sure that participants are safe and informed.

Participation in a trial is always voluntary. Whether you or your child takes part in a clinical trial is up to you. No one has to be part of a clinical trial, and no one should be made to feel that way.

Informed Consent is a process in which doctors and nurses communicate with potential and enrolled participants about the key facts of a clinical trial. A member of the research team will review the details of the study and provide an Informed Consent document for you to sign. The Informed Consent describes the potential benefits and risks to the patient, and also informs the participant that they can discontinue participation in the trial at any time.

Participants in a clinical trial must freely give their consent after both verbal and written information about their trial has been given to them by their investigator. In almost all cases, parents have to give legal consent for their child to join a research study. Even though a child may not be legally able to provide consent, they are still informed about the clinical research at a level that they can understand. They may also have the opportunity to provide their agreement to participate in the study through a process called assent.

To learn more about clinical trials, please visit:

NIH Clinical Trials Website: https://clinicaltrials.gov/ct2/about-studies/learn
NIH Clinical Research Trials and You: http://www.nih.gov/health/clinicaltrials/index.htm
NIH Children & Clinical Studies: http://www.nhlbi.nih.gov/childrenandclinicalstudies/index.php
The Center on Information & Study on Clinical Research Participation: https://www.ciscrp.org