Clinical Research

What is clinical research? What are clinical trials?

Clinical research is the process of studying diseases, and developing medications to possibly prevent, treat, or help relieve symptoms caused by different medical conditions. Before a medicine can become widely available, it must be tested in a series of clinical trials, or clinical research studies. The medicine being studied or tested in a clinical trial is called an investigational medication.

Most clinical trials recruit volunteers to participate in the study. Those volunteers are closely monitored to follow the effects of an investigational medication on their health, and that information is used by health authorities to determine if the investigational medication is safe and effective, and if it should be approved for general medical use.

How do clinical trials work?

Clinical trials usually go through 3 phases before an investigational medication can be approved for general use:

Phase1

Healthy volunteers receive the investigational medication to help researchers study its initial safety, and to understand how the drug is processed by the body. After the initial results from healthy volunteers are evaluated, some phase 1 trials will then include people with the disease.

Phase2

The investigational medication is given to a small number of people with the disease to learn more about how safe it is and how it is processed by the body. These trials may also evaluate different doses to determine which should be studied further in future trials.

Phase3

These trials include a larger number of people and are intended to generate enough information to establish whether the investigational medication can safely be administered, and whether it provides sufficient benefit to be made available to the public.

VALIANT is a Phase 3 study

Why do people participate in clinical research studies?

Clinical trial participants can have an impact on the lives of others by helping researchers advance medical science, develop treatment options, and better understand the disease.

It’s important to remember that clinical trials involve some risks:

There may be limited information about the safety or effectiveness of an investigational medication, including whether it works at all, or what side effects it could cause.
Participants may not see improvement, or improvement may take several months or more to appear.
There is potential for a participant’s condition to worsen.

For these reasons trial participants are screened carefully and monitored throughout the trial.

Some trial participants also feel the additional time and support they receive from study doctors and investigators during the study helps them learn more about their condition. Whatever the motivation for joining a study, participation is entirely voluntary, and participants can choose to leave the study at any time, for any reason, if they change their mind.

Understanding clinical research terms

Clinical research terminology can sometimes be confusing. We have created this glossary of clinical trial language to help you understand some of the key terms:

Term	Definition
Clinical trial participants	People who voluntarily consent to take part in a clinical trial and who meet all of the eligibility criteria.
Eligibility criteria	The requirements one must meet to participate in a clinical trial. These may include age, diagnosis, current and previous treatments, allergies, medical history, and other conditions.
Independent ethics committee (IEC) or institutional review board (IRB)	Independent committees made up of medical experts and the general public, who review, approve, and monitor all aspects of clinical research studies involving people. These groups ensure study protocols adhere to all relevant regulations, and that the rights and welfare of all participants are protected.
Informed Consent Form (ICF)	An important written document that is part of a larger consent discussion with the study team. The ICF describes the study, including study-related procedures, timelines, and potential risks and benefits. By signing the ICF, patients agree to participate. Parents or guardians must sign for minors to participate in a clinical trial, however the minor may be asked to sign a simplified “Assent Form” to show they understand what is being asked of them and that they are participating voluntarily.
Investigational medication or drug	The drug or medicine being studied in a clinical trial. Typically, this medication is not yet approved for general use for the condition being studied.
Study medication or drug	This term may be used to describe the investigational medication or other study treatment (such as placebo) that a participant may receive while in the study.
Open-label, open-label extension	In an open-label study, or open-label extension (continuation of the study) both the study team and the participant know which treatment group they have been assigned to, or which study treatment is being administered.
Single-blind	In a single-blind study, the study participant does not know the study treatment group they have been assigned to, but the study team does.
Double-blind	In a double-blind study, neither the participant nor the study team knows which study treatment group the participant has been assigned to, but the study team can find out in case of an emergency.
Placebo	A substance that looks like the investigational medication but has no active medicinal ingredients. Placebos are used to compare the effectiveness of the investigational medication against no treatment. This is important to correct for the “placebo effect” when people feel better because they think they’re taking a medicine, even if the medicine does not do anything.
Protocol	A detailed plan describing the purpose of a study, how the study will be conducted, who can participate, the investigational medication that will be administered, and how the effects will be monitored.
Randomization	Randomized clinical trials assign participants at random to groups or cohorts within the trial, with each group receiving a different study treatment regimen. The different regimens may include the investigational medication, a placebo, an alternative therapy, or some combination of them. Randomization is important in gathering clear, unbiased results. Not all studies involve placebos, alternative therapies, or randomization. If a particular study is randomized, patients will be made aware before agreeing to participate.
Study team, or study site team	The doctors, nurses, and other professionals who are conducting a study at a particular clinic or hospital. The doctors may also be referred to as investigators for the study.

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