Clinical trials are very important for finding safe and effective treatments for vitiligo.¹ While there are some treatment options for vitiligo, it can still be hard to treat.² People with vitiligo may be eligible to participate in a clinical trial, helping scientists study new investigational treatments for vitiligo and learn more about the condition.¹

If you’re thinking about joining a clinical trial for vitiligo, here are seven things you should know.

1. A Clinical Research Program Typically Has Four Phases

The steps of a research programs are called phases.¹

Phase 1

A phase 1 clinical trial focuses on safety and side effects on a few people. It may include people with or without the condition being studied.¹

Phase 2

A phase 2 clinical trial tests the treatment on more people with the condition to check its safety and effectiveness. Sometimes, several hundred people with a condition will participate in this phase.¹

Phase 3

A phase 3 clinical trial aims at confirming the treatment’s safety and effectiveness in patients with the condition. This is the last phase before potential U.S. Food and Drug Administration (FDA) approval. These are very large studies with hundreds — sometimes thousands — of participants.³

FDA Approval

After phase 3, the results of the clinical trial are reviewed by the FDA. If the FDA finds that the treatment is safe and effective, it’s approved for use by health care providers for the condition it was tested for in the trials.³

Phase 4

Once approved, the treatment is available to people with the condition. During a phase 4 clinical trial, researchers continue studying long-term effects in real-world situations. Usually, these are very large studies with thousands of participants.^1,3

2. Treatments Are Tested Before Clinical Trials

Before a clinical trial, an investigational drug or device is studied in the laboratory to check its safety and effectiveness.⁴ If it passes these tests, the FDA approves it for trials with humans.³

3. Safety Is a Priority

Each country has rules for approving clinical trial designs. In the U.S., a clinical trial protocol outlines how study participants will be protected from risks.¹ The FDA requires most studies to be reviewed by an Institutional Review Board (or IRB), which protects the well-being and rights of volunteers.⁵ Clinical trials may also have other committees that monitor the safety of volunteers during a study.⁵

4. You Will Know the Details Before Joining

Before you join a clinical trial, you’ll get detailed information about it, including possible risks and what will happen during the trial. This process, called “informed consent,” gives you the opportunity to discuss the trial with your health care provider and family and ask questions.⁵

5. Placebo May Be Used in a Clinical Trial

In some trials, you’ll receive a placebo (a substance that looks like the investigational drug but has no medicine in it).⁵ This helps researchers compare it to the investigational treatment they’re studying.⁵

Clinical trials often have a group of people who get the placebo and a group of people who get the real investigational treatment. This group is chosen randomly, and neither the participant nor the researcher knows who is getting the placebo. Even if you get the placebo, you might still get the real investigational treatment for your condition along with the placebo.⁵

Sometimes, after the phase 3 trial ends, you might get asked to join another study called an extension study. An extension study gathers more information about the treatment’s safety and effectiveness.⁶

6. Investigational Drugs Have Risks

Every drug, including those in clinical trials, can have side effects. Before joining a clinical trial, you’ll be told about possible risks and side effects.⁵ It’s also possible that the investigational treatment may not work.⁵

7. Joining a Clinical Trial Is Voluntary

Joining a clinical trial is 100 percent voluntary, and you can leave at any time.⁵

Talk With Others Who Understand

MyVitiligoTeam is the social network for people with vitiligo and their loved ones. Here, over 13,000 members come together to ask questions, offer support and advice, and meet others who understand life with vitiligo.

Do you have questions about clinical trials for vitiligo and how they work? Share your thoughts in the comments below or by posting on your Activities page.