What’s a Research Study?

Without research studies, we wouldn’t have scientifically tested treatments for thyroid cancer and other conditions. Research studies provide an organized blueprint for testing the effectiveness and safety of an investigational medication or treatment.

Research studies can also be called clinical trials, clinical studies, clinical research, or other things. They are designed to answer specific questions about an investigational medication or treatment.

Some questions that research studies can help us answer include:

Is the investigational medication more effective than a placebo?

Is the investigational medication or treatment safe?

What are the side effects of the investigational medication being studied?

Thyroid Cancer Research Study Phases

This is a phase 3 research study. Phase 3 research studies are the third step in a four-step drug approval process. These studies take place after the investigational medication’s safety has been tested in a small group of people. Phase 3 studies are usually the first study to look at an investigational medication’s effectiveness and safety compared to the standard treatment for a disease or condition.

Is It Safe?

Scientists test the safety and dosing range of an investigational medication or treatment in a small group of healthy people.

Timeline:

Months

Participants:

20-100 healthy volunteers

Goal:

The goal of phase 1 research studies is to see if the investigational medication is appropriately safe to keep testing in more people.

Success Rate:

70%
If the clinical trial is successful, the investigational medication moves to phase 2 clinical trials.

Does It Work, and What are the Side Effects?

Scientists test whether an investigational medication works in a larger group of people with a specific disease or condition. Side effects are noted.

Timeline:

Months to Years

Participants:

100-300 people with the specific disease or condition

Goal:

The goal of phase 2 research studies is to see if the investigational medication works and to note side effects.

Success Rate:

33%
If the clinical trial is successful, the investigational medication moves to phase 3 clinical trials.

How Well Does It Work?

Scientists test whether an investigational medication is more effective than the standard treatment in people with a specific disease or condition.

Timeline:

Months to Decades

Participants:

300-3000 people with the specific disease or condition being studied

Goal:

The goal of phase 3 research studies is to compare the investigational medication to the standard treatment.

Success Rate:

25%-30%
If the clinical trial is successful, the investigational medication becomes eligible for approval to be prescribed and sold, and moves to phase 4 clinical trials.

How Else Could It Work?

Scientists collect data about the real-world experiences of people taking the new drug, over a long time.

Timeline:

Months to Decades

Participants:

Several thousand people who have the disease or condition

Goal:

The goal of phase 4 research studies is for the potential medication to become the new standard treatment.

Success Rate:

Not Applicable
If the clinical trial is successful, the potential medication can continue to be prescribed and sold, possibly for new uses.

5 Things To Know About Participation

Informed Consent
Before you begin participating in a research study, you will become involved in the informed consent process. As part of the process, you will receive an informed consent document that outlines the risks and benefits of participation. You can take all the time you need with this document and ask any questions you think of. You can also take the document home with you and share it with your loved ones. You will need to sign the informed consent document if you decide to participate in the study.

Informed consent is an ongoing process that continues throughout the study. Risks and benefits are provided to you throughout the study, and you can ask questions at any time. Informed consent is designed to provide you with information so that you can make an informed decision about your participation.

Ethical Rules & IRB Review
In addition to informed consent, researchers are bound by ethical guidelines which are designed to protect participants and uphold the integrity of science. Also, research studies involving humans are approved and monitored by an Institutional Review Board (IRB). The IRB makes sure that the study’s risks are reduced and that any potential benefits outweigh the risks.

According to the Center for Information & Study of Clinical Research Participation’s (CISCRP) 2019 Participation Experiences survey, 96% of research study participants would participate in another study.

New MTC treatments cannot be developed without volunteers like you. Research studies in people are an important part of testing any potential new drug. The safety and effectiveness of a drug must be tested in larger and larger groups of people, before it can be approved to be sold and prescribed the people like you.

By taking part in a MTC research study, you may be able to make important contributions to medullary thyroid cancer research and help other people with the disease.

Not all clinical trials have a placebo group. In fact, many clinical trials involve testing a known, effective treatment, also known as the standard of care, against a new, investigational medication.

Our study is not using a placebo group.

In our trial, all study volunteers will receive an active medication. One group of participants will receive the investigational medication and the other group will receive the standard of care, which is either cabozantinib or vandetanib. If you are in the group receiving the standard of care, you may be able to receive the investigational medication later in the trial, depending on how you are doing.

Investigational medicines have been tested for years before they make it to clinical trials. On average, a new cancer drug has been studied for at least 6 years before it even makes it to clinical trials.

About Our Thyroid Cancer Research Study

If you’re someone with medullary thyroid cancer that is spreading or getting worse, and are not a candidate for surgery, our study might be for you. Click the I’m Interested button if you are interested in participating.

About Lilly Thyroid Cancer Research

Are you interested in learning more about research? We have an entire website devoted to research, including our own research. If you’re interested in learning about other Lilly thyroid cancer research studies, you can search for those too.