What do you know about participating in a research study?

The most comprehensive source of information about clinical studies is the database of clinical studies in the US found at clinicaltrials.gov. All new studies that are in search of participants are listed there, and the database can be searched by the state of one’s disease. However, the clinical trial listings on this site are complicated, and difficult for the average person to understand. Here’s a quick guide to the terminology you might find in a clinical trial listing.

Phases of a clinical trial

These are the three stages of study for a new medication before it’s submitted to the FDA so that it can be approved for prescription.

Phase I studies are designed to study the safety of the medication.
Phase 2 studies are designed to test the medication’s efficacy.
Phase 3 studies are the final step, and have more participants to make sure the Phase 2 results are similar in a larger group of people with the condition or disease.

Types of study designs

In addition to a study’s phases, the study type can be described as:

Biomarker: Studies that are looking for risk factors, signs of disease, or severity of disease.
Natural history studies: These studies observe and determine how the symptoms of a disease can change or progress over time.
Interventional studies: Studies to assess new medications, devices, or non-medical therapies that can be used to treat a condition or symptoms of a condition.

Inclusion and exclusion criteria

Another important element in a clinical trials listing is the inclusion/exclusion criteria. The inclusion criteria refer to the qualities a person must have to be eligible to participate in a study. By reviewing these qualifications, you can tell if you are likely to be accepted as a participant. Here are some examples of the kinds of criteria a patient might need to meet in order to qualify for a study:

A specific diagnosis and diagnostic age
Age
Symptoms experienced
Current medications and treatment plans
Lab test results (e.g. blood test results; platelet levels (high or low); white blood cell levels; kidney and liver results)

Exclusion criteria are the factors that may prevent a patient from participating, such as:

Certain medications or a recent change in medication
Other conditions (such as heart disease, liver disease, etc.)
If pregnant or nursing

How the efficacy of a new potential medication is determined

Interventional studies, which test a new drug or treatment, must compare the potential new treatment to a pre-existing medication or treatment to determine if the new alternative really did make a difference and is better than what is currently available. In some studies, a comparison is made by giving an equal number of participants either the study drug or a placebo – which is a pill/injection/infusion that looks like the active treatment but does not contain an active drug ingredient. This way, the new potential treatment has to demonstrate a positive effect compared to the placebo. However, in many rare disease studies, placebos are not used alone without any treatment, but may be added to the existing usual treatment to see if the new drug has an increased beneficial effect.

The determination of which patients will receive the active drug or the placebo is randomized, like the toss of a coin. After the treatment period is over, in some studies you will find out whether you had the placebo or the active drug. You may be able to participate in an extension of the study, where every participant will receive the active study drug and be monitored while on it.

More resources are available about participating in a clinical trial:

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