About 95 percent of rare diseases have no approved treatments, according to the National Organization for Rare Disorders (NORD). Clinical trials are important research studies that can help researchers find new or better treatments for rare diseases such as Duchenne muscular dystrophy (DMD).

If you or a loved one has been diagnosed with DMD, enrolling in a clinical trial can help researchers learn more about DMD. Deciding whether to participate in a clinical trial is a personal choice. Below, we explain what clinical trials are and what you should consider before joining.

What Is a Clinical Trial?

Clinical trials are research studies that test new ways to prevent, diagnose, or treat medical conditions. Examples of what researchers might study in a clinical trial include:

• Newly discovered drugs
• New combinations of existing drugs
• Older drugs used in new ways
• Medical procedures
• Medical devices

Some clinical studies don’t test treatments but, instead, aim to learn more about a disease. These are called observational studies. In these studies, researchers observe a group of people with a disease over time to learn more about it. Participants don’t receive any treatment meant to change their health outcomes.

A natural history study is a type of observational study. These studies help researchers learn more about how a disease develops and progresses. In this type of study, researchers will collect information from people who have or are at risk for a specific disease. Natural history studies are especially important for rare diseases like DMD because they help scientists develop new treatments.

Types of Clinical Trials

New therapies go through three phases (steps) of testing before they’re approved by agencies, like the U.S. Food and Drug Administration (FDA). Sometimes, a fourth phase happens after a drug is FDA-approved. The type of clinical trial depends on what researchers want to learn.

Phase 1

In a phase 1 clinical trial, researchers test a new treatment to see if it’s safe and identify possible side effects. They also try to find the best dose — one that works well and causes the fewest side effects. Phase 1 clinical trials usually involve 20 to 80 people.

Most phase 1 trials use healthy volunteers, but sometimes they include people who are very sick, such as those with cancer.

Phase 2

In a phase 2 clinical trial, researchers test what they learned about the new therapy in Phase 1 on people who have the disease being studied. Phase 2 clinical trials typically include 100 to 300 people. The goal is to see if the treatment works and is safe. Phase 2 clinical trials can last from several months to two years.

After phase 1 and phase 2 clinical trials are completed, the FDA meets with the company that made the treatment to decide whether to have a Phase 3 trial.

Phase 3

Phase 3 clinical trials involve even more people — often thousands of participants. In this phase, researchers compare the new treatment to a placebo (no treatment) or the current standard treatment to prove it’s safe and effective.

If the results are positive, the company making the new treatment can apply for FDA approval to make the treatment available to the public.

Phase 4

Phase 4 trials happen after a drug is FDA-approved. At this phase, researchers continue to monitor the treatment’s safety and effectiveness in the general public. Many phase 4 trials are observational studies, meaning researchers observe participants without giving them specific treatments.

Who Can Join a Clinical Trial?

Before a clinical trial starts, researchers create eligibility criteria. These are guidelines that decide who can and can’t join the study. Following these eligibility criteria helps researchers keep participants safe and make sure the results apply to the right group of people.

Some common factors in eligibility criteria include:

• Age
• Sex
• Type of disease
• Severity of disease
• Past treatments (medical history)
• Overall health and other medical conditions
• Other factors, like weight or genetic factors

If you have DMD, you may be eligible to participate in a phase 2, 3, or 4 clinical trial testing new DMD treatments. If you don’t have DMD, you may still be able to participate in DMD clinical research as a healthy volunteer in a phase 1 clinical trial.

What Are the Benefits of Joining a DMD Clinical Trial?

Clinical research is vital for the Duchenne community. By participating in clinical research, you help advance knowledge of DMD, which could eventually lead to a cure. Many people with DMD feel empowered by contributing to the clinical research process. In addition to benefiting the DMD community as a whole, clinical trials can also benefit individual participants.

When you join a clinical trial, you’ll receive regular medical care from healthcare providers who specialize in your condition. You may also get access to new treatments before they’re FDA-approved. In fact, some treatments are only available by enrolling in a clinical trial. For example, some types of gene therapy are only available by enrolling in a clinical trial. Additionally, financial assistance may be available to help with costs.

What Are the Risks of Joining a DMD Clinical Trial?

While there are benefits to joining a clinical trial, it’s important to remember that new treatments in clinical trials may have risks. These can include physical harm, as well as emotional and psychological stress. Before joining a clinical trial, it’s important to fully understand these risks.

The process of learning about the risks and benefits of a clinical trial is called informed consent. During this process, a member of the clinical trial team will explain key details about the study to help you decide if you want to participate. In rare cases, life-threatening complications can occur from a new treatment.

Another risk of participating in a clinical trial is that you might not receive the new treatment. In some clinical trials, a new treatment is compared to the current standard treatment. In other clinical trials, a new treatment is compared to a placebo. Be sure to ask the clinical trial team if placebos or other treatments are used in the study.

Another factor to think about before joining a clinical trial is the time commitment needed to participate. Participating in a clinical trial may require frequent visits to the study site or additional medical tests. Make sure the clinical trial’s schedule works with your life before joining.

It’s also important to know that you can leave a clinical trial at any time, for any reason.

Finding a Clinical Trial

If you’re interested in joining a clinical trial for DMD, start by talking to your healthcare provider. Your healthcare provider may know about studies you may qualify for. You can also search for DMD clinical trials by using a clinical trial search tool. Some advocacy groups, such as the Muscular Dystrophy Association and the Parent Project Muscular Dystrophy, have their own clinical trial search tools.

If you’re not ready to join a clinical trial for a new treatment, you can still help DMD research in other ways. For example, you could join an observational study, where researchers track changes in your health over time. One example is the Duchenne Registry, which is open to people with DMD, Becker muscular dystrophy, and female carriers.

Find Your Team

On myMDteam, the site for people with muscular dystrophy and their loved ones, members come together to ask questions, give advice, and share their stories with others who understand life with muscular dystrophy.

Have you participated in a clinical trial? What was your experience as a clinical trial participant? Share your experiences in the comments below or by posting on your Activities page.