Your practical guide to clinical trials

by Amanda Hill, FSHD Society

How do I find clinical trials that are recruiting participants?

Our website is one of the best ways to find clinical trials that are recruiting. We will always have current information for you on our Clinical Trials web page. Another great way to find clinical trials is at clinicaltrials.gov. This website is a database for clinical trials and other clinical research studies all over the world. To get started on clinicaltrials.gov, use the “Find a study” search tool on the homepage. Check the “Recruiting and not yet recruiting studies” status box and enter “FSHD” as the condition or disease. If you want, you can also add your country, state, and city to find clinical trials with study sites near you. Once you get the list of results, you can use filters to narrow your search. Find the “Study Type” filter and check the box for the type of studies in which you are interested. Here’s a video showing you how to search for clinical trial information:

 

How do I know if I am eligible for a clinical trial?

You should review the eligibility criteria posted on our website and on clinicaltrials.gov to see if you might qualify for the clinical trial. Some criteria must be assessed by the study team, like your clinical severity score or reachable workspace area. But other criteria you can assess yourself, like your age and health history.

What Can I Do to Be Trial Ready?

These steps will reduce delays and improve your chances of enrolling in a trial. You will also need to meet the eligibility criteria for the specific trial. There’s no guarantee, as many factors must be weighed in selecting people for a trial, but these measures can only help.

• Make sure you have been diagnosed by a doctor
• Get a genetic test confirming FSHD
• Become a patient at an FSHD Clinical Trials Research Network location*
• Enroll in the MOVE or MOVE+ study
• See your neurologist every year
• Follow exercise recommendations & be as healthy as possible
• Sign up to get FSHD Society email alerts.

How do I choose which clinical trial to participate in?

There are many factors to think about when deciding among clinical trials. You should think about the risks and benefits, and about your ability to commit to each study. For example:

• Are you able to attend all the study visits? You will most likely have to attend multiple study visits over weeks, months, or even years. Study visits vary in length but can sometimes last an entire day.
• Will you need to travel? You may need to travel for some or all of the study visits if there isn’t a study site near you. If you need a companion to travel with you, will they be able to take time off from work? Ask if the study can reimburse travel costs for you and/or your travel companion.
• How much risk are you willing to accept? The medicines in Phase 1 and Phase 1/2 studies have usually not been tested in people before. These studies can be riskier, and the study team will want to monitor you very closely. This may also mean more work for you, such as keeping a diary of any side effects or going to more study visits.
• Are you willing to take a placebo? Phase 2 and 3 studies often compare a medicine to a placebo. This means that some participants do not receive the real medicine. However, you won’t know if you are taking the medicine or the placebo until the end of the study.
• Is there an open-label extension? Phase 2 and 3 studies sometimes have an open-label extension, which means you may be able to keep taking the medicine for even longer. If you were receiving a placebo during the study, an open-label extension gives you the chance to take the medicine instead.

How do I sign up to participate in a clinical trial?

1. Use our website or clinicaltrials.gov to find the contact information for the study site you want to go to. This is usually an email address, phone number, or website form.
2. Contact the study site and let them know the name of the clinical trial you are interested in. You might have to wait for the study site to call or email you back. This could take several weeks, but be patient.
3. Usually, the first step in the screening process is a phone call. The study team will tell you more about the study and ask you some basic questions. If they think you will meet the eligibility criteria, they will schedule a time for you to come to the study site for a screening visit.
4. At the screening visit, the study team will talk you through the informed consent. If you agree to participate, the study team will perform all the tests needed to find out if you meet the eligibility criteria. It might take a couple of days to get all the results of your tests back.
5. Finally, the study team will tell you whether you meet all the eligibility criteria. If you meet the criteria, you can choose to continue participating in the study. If you do not meet the criteria, you will not be able to participate.

What types of activities could I be asked to do during a clinical trial?

You will almost always have to attend study visits in person during a clinical trial, though you may be able to do some visits virtually. The study team may also call you on the phone to see how you are doing or ask you to do certain activities at home. Examples of activities you may be asked to do at study visits or at home include:

• Physical exam.
• Blood draw.
• Electrocardiogram or other tests to monitor your heart.
• Answering questions about your health and ways your disease affects your life. Sometimes, these questions can be very personal. For example, you might be asked about your mental or sexual health.
• Tests to measure your strength, mobility, or range of motion.
• MRI scan.
• Muscle biopsy.
• Taking your medicine at specific times.
• Notifying the study team about any illness, injuries, or side effects you experience.

Is the medicine safe? What happens if the medicine makes me sick?

One purpose of clinical trials is to learn about the safety of a new medicine. The study team doesn’t always know what side effects could happen. This is a risk of participating in clinical trials. However, clinical trials must meet strict standards to help ensure the medicine is as safe as possible.

If the medicine makes you sick, your study team will record what happened. The study team will decide whether they think it is safe for you to continue taking the medicine or if you should stop. They will also help make sure you receive any medical care you need. However, your insurance might have to pay for that medical care. The informed consent form will explain this information.

Will I learn about the results of the clinical trial?

Yes! You will get to learn about the overall results of the trial, but not your personal scores. However, it could take a long time. Even after you finish your part in the study, other participants might not finish for several more months. The study team must wait for all participants to finish before they can do a full analysis of the data collected. It could take the study team 6-18 months to perform the analysis and prepare to share the results. Sometimes, a study team will share preliminary results. Often, the FSHD Society will schedule a webinar to explain the results to the community.

Many study teams will notify participants when they have results to share, for example, through an email newsletter. Sometimes, you will have to watch out for news stories, social media posts, or a press release. Most study teams will also publish their results in scientific journals or present them at scientific conferences.

Glossary

Eligibility criteria: Health traits that allow you to be included in a study, or that cause you to be excluded from a study. These are also called “Inclusion and Exclusion Criteria.”

Informed consent: The process of learning everything you need to know about a study and deciding if you want to participate. This includes learning about:

• the purpose of the study
• what you will be asked to do
• what tests and procedures will be performed
• the schedule of study visits
• the risks and benefits
• how your data are used

Intervention: A medicine or physical treatment that interferes with a disease. Clinical trials are studies that test interventions.

Open-label extension (OLE): An optional period after the end of the study when all participants receive the medicine, and the study team continues to collect data.

Placebo: A mock version of a medicine which looks the same but does not contain the active ingredients that interfere with disease.

Screening: A series of questions and tests the study team will perform on you to find out if you meet all the eligibility criteria for a study.

Study: A research process to learn about a disease or test a new idea. Study site: A place where a study is performed, often hospitals, clinics, or research centers.

Study visit: Defined times when the study team needs to perform tests or collect data from you. Some study visits may be virtual, but many will be in-person. You will receive a copy of this information in an “Informed Consent Form.” If you agree to participate in the study, you will give your consent by signing the Informed Consent Form. Importantly, you can withdraw your consent and decide not to participate in a study at any time and for any reason.