Clinical Research & Clinical Trials

Clinical Research aims to advance medical knowledge by studying people, either through direct interaction or through the collection and analysis of blood, tissues, or other samples.
 
A clinical trial involves research participants. It follows a pre-defined plan or protocol to evaluate the effects of a medical or behavioral intervention on health outcomes. By taking part in clinical trials, participants not only play a more active role in their own health care, but they also can access experimental treatments and help others by contributing to medical research.
Clinical trials are part of clinical research and at the heart of all medical advances. Clinical trials look at new ways to prevent, detect, or treat disease.

Treatments might be new drugs or new combinations of drugs, new surgical procedures or devices, or new ways to use existing treatments.

The goal of clinical trials is to determine if a new test or treatment works and is safe. Clinical trials can also look at other aspects of care, such as improving the quality of life for people with chronic illnesses.

People participate in clinical trials for a variety of reasons. Healthy volunteers say they participate to help others and to contribute to moving science forward. Participants with an illness or disease also participate to help others, but also to possibly receive the newest treatment and to have the additional care and attention from the clinical trial staff.

Clinical trials offer hope for many people and an opportunity to help researchers find better treatments for others in the future.

Why Should I Participate in a Clinical Trial?

Definition:
To fully understand this term, we need to break it down into its components:

A clinical trial is one that involves human participants and seeks to answer specific questions about a type of medical intervention. This can be a drug or other type of treatment, such as nutritional changes or massage.

In the context of a clinical trial, double-blind means that neither the patients nor the researchers know who is getting a placebo and who is getting the treatment.

Because patients don't know what they're getting, their belief about what will happen doesn't taint the results. Because the researchers don't know either, they can't hint to patients about what they're getting, and they also won't taint results through their own biased expectations about what the results will be.

If researchers do know who's getting the treatment but the participants do not, it's called a single-blind trial.

A placebo is an inactive substance (often a sugar pill) given to a patient in place of medication.

In drug trials, a control group is given a placebo while another group is given the drug (or other treatment) being studied. That way, researchers can compare the drug's effectiveness against the placebo's effectiveness.

Placebo-controlled refers to a control group receiving a placebo. This sets it apart from studies that simply give participants a treatment and record the results
 

Double-Blind, Placebo-Controlled Clinical Trial

Phase 1 Clinical Trials – Is the Treatment Safe?

Phase 1 clinical trials are the first tests done on humans for an experimental medication or treatment. They usually involve only a small number of people - often 10 to 30 - and are designed to see if a treatment is safe. It's important to note that before a phase I trial is started, a new drug has usually been tested extensively in the lab.

For example, before beginning a phase I clinical trial, a chemotherapy drug may be tested on human cancer cells grown in the lab, and on laboratory animals.

An example of a phase one trial would be testing a drug that has previously only been tested on rats on humans to see if it is safe.
 
Phase 2 Clinical Trials – Does the Treatment Work?  Is it Effective?

After a drug or treatment is found to be relatively safe, it is tested in a phase 2 trial to see if it is effective. Since a larger number of individuals - often 30 to 120 - are enrolled in these studies, further information is obtained about safety as well.

Phases of Clinical Trials

Phase 3 Clinical Trials – Does the Treatment Work Better Than the Standard Treatment or Does it Have Fewer Side Effects Than the Standard Treatment?

Once a medication or treatment is felt to be relatively safe and effective, it is studied in a phase 3 trial to see if it works better than, or has fewer side effects than, the standard treatments available. Phase 3 trials are usually conducted on hundreds to thousands of individuals and are usually “double-blind” studies; meaning that neither the patient nor the investigator knows which treatment is being used. If the experimental treatment is found to be either superior or inferior to the standard treatment, these studies are usually stopped early to allow individuals to receive the best treatment possible.

An example might be to test a less invasive surgery for lung cancer to see if either the survival rate is higher than traditional surgery, or if the new surgery has fewer side effects and complications than the traditional (standard) surgery.
 
Phase 4 Clinical Trials
There are also phase 4 clinical trials. After a drug has been approved for general use, a phase 4 trial may be done to look at the safety of a drug or procedure over time, or to look at other uses for the drug
 

Randomization

A method based on chance alone by which study participants are assigned to a treatment group. Randomization minimizes the differences among groups by equally distributing people with particular characteristics among all the trial arms. The researchers do not know which treatment is better. From what is known at the time, any one of the treatments chosen could be of benefit to the participant.