Learning about clinical research & clinical trials
Clinical research is research conducted in human volunteers. Clinical research can have many purposes, such as understanding the cause of disease, how a disease progresses, and how to diagnose or treat a disease.
A clinical trial is a type of clinical research study involving human volunteers. Usually, the term refers to a research study that tests a medicine, medical device, or other type of therapy to see if it is a safe and effective way to treat a disease. It may also be called an “interventional clinical trial” or simply a “clinical study.”
Taking part in clinical research is not the same as getting standard clinical care. You should see your doctor for clinical care at least once per year. Your doctor will monitor your FSHD and your overall health and can provide any medical treatments or referrals you may need. During a clinical care visit, your doctor may talk to you about taking part in a clinical trial if there aren’t good treatment options available.
However, when you take part in clinical research or a clinical trial, there is no guarantee that the research will directly benefit you. Any treatment you may receive as part of a clinical trial is experimental. This means that the researchers don’t know if it will work. But by taking part, you are supporting research that will help patients in the future.
Sometimes, the doctors or clinicians you see for clinical care are the same people that perform clinical research. However, they are usually supported by different teams of professionals and often use different facilities. If you are interested in taking part in clinical research or clinical trials, it’s usually a good idea to establish care in a clinic that also does research.
Here’s a helpful breakdown of the differences between clinical trials and medical treatments provided as part of research and clinical care:
- Designed to answer specific research questions by involving numerous volunteers
- Generally intended to benefit patients in the future
- Clinical trials test unproven medicines to see if they are safe and effective
- In a clinical trial, you may not know whether you are receiving medicine or placebo
- A research study usually lasts for a defined period of time (for example, 2 years)
- Paid for by drug developers, government funding, and other research grants
- NOT a replacement for clinical care. You may receive some care, but only for problems triggered by the research study
- Designed to address the medical needs of an individual patient
- Intended to benefit the individual patient immediately
- Uses medicines already accepted by the medical community as safe and effective
- Always receive the medicine you expect, never a placebo
- Clinical care is an ongoing process and relationship with doctors and care providers
- Paid for by patients and their health plans
- Refers you to additional providers that can help you manage disease, for example, a physical therapist or dietician
You can read more about the difference between clinical research and clinical care in Amanda Hill's article in Issue 3 of FSHD Advocate (2023).
Clinical trials are typically conducted in 4 phases. All phases are used to assess how safe a medicine is, what the side effects might be, and how well it works. However, each phase has a slightly different focus. What researchers learn in one phase helps them design the next phase.
- Usually the first time the medicine is put in a human
- Understand how the medicine acts in the human body
- Find the best dose(s) of the medicine
- Begin to define how well the medicine works
- Usually compares the medicine to a placebo or existing treatments
- Better define how well the medicine works
- Always compares the medicine to a placebo or existing treatments
- The final data-gathering step before applying for approval from regulatory bodies
- Continued study of the medicine after approval
A placebo is a mock version of a medicine. It looks like the real medicine, but it does not contain the active ingredients that interfere with disease. Researchers can compare results from the placebo to those from the real medicine. This helps them to determine if the medicine works.
Getting started with clinical trials
Yes, you will most likely need a clinical genetic test. The test must be from your doctor or a clinical laboratory service, such as TestFSHD. Genetic tests from researchers, such as Dr. Peter Jones, do not currently meet the criteria. If you do not have a clinical genetic test, the clinical trial may be able to provide one for you.
Our website is one the best ways to find clinical trials that are recruiting. We will always have current information for you at https://www.fshdsociety.org/clinical-trials/. Another great way to find clinical trials is on 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, try following these steps:
- Use the “Find a study” search tool on the homepage
- Check the “Recruiting and not yet recruiting studies” status box
- 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.
- Hit “Search”
- Once you get the list of results, you can use filters to narrow your search
- Find the “Study Type” filter and check the box to see only “Interventional (Clinical Trial)” studies.
Use our website or clinicaltrials.gov to find the contact information for the clinical trial study site you want to go to. This is usually an email address, phone number, or website form. 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 or even months, but be patient.
- If you live near a clinical trial site, make an appointment as a patient and establish clinical care at the site. Often, sites draw from their patient databases first for clinical trials.
- Check clinicaltrials.gov often for updates on the clinical trial’s locations and contact information.
- If you are able and willing to travel to multiple different sites, contact each site individually and let them know you are interested in the clinical trial.
- Provide your email and phone number to make it easier for the coordinators to contact you.
- Keep checking in on a monthly basis. This helps the coordinators know that you are committed to completing the clinical trial.
Screening and eligibility
Inclusion criteria are health traits that allow you to be included in a study. Exclusion criteria are health trains that cause you to be excluded from a study. Together, inclusion and exclusion criteria define who is eligible for a clinical trial.
Screening is the process to find out if you are eligible for a study. This usually involves answering questions and performing tests with the study team. For example, you might need do bloodwork, an MRI, and perform tests to measure your strength. Screening almost always happens at an in-person visit. A screening visit can take several hours and be exhausting.
Pre-screening is the process to narrow down patients before scheduling an in-person screening visit. This might involve learning more about the study and answering questions about your health. Pre-screening typically happens via phone, email, or video call.
Informed consent is the process of learning everything you need to know about a study and deciding if you want to take part. Informed consent is often part of the screening visit, though sometimes it is done over the phone or a video call. Informed consent 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 is used
You will receive a copy of this information in an “Informed Consent Form.” If you agree to take part in the study, you will give your consent by signing the Informed Consent Form. Importantly, you can withdraw your consent and decide not to take part in a study at any time and for any reason.
Not getting into a clinical trial can be very disappointing, especially if you spent a lot of time and effort to do pre-screening and screening. Sometimes, studies have more volunteers than they need, so you may not have even been contacted for pre-screening or screening.
In any case, you may be able to get into a different clinical trial! You can learn about other clinical trials on our website at https://www.fshdsociety.org/for-patients-families/clinical-trials/. You should check back every couple of months to see if there are any new clinical trials you might be eligible for.
If you do not meet the eligibility criteria for any clinical trials, you can still help advance research in many ways:
- Take part in the MOVE natural history study
- Check our website for other clinical research opportunities
- Help your family members or loved ones with FSHD get involved in clinical trials and research
- Help us raise awareness of FSHD
- Donate to the FSHD Society to support our work advancing research and clinical trials
You may also be interested in “How can I increase my chances of getting into a clinical trial?”
What to expect during a clinical trial
Yes, most clinical trials will reimburse you for reasonable travel expenses. This may include economy flights, gas, hotel, public transit, and food costs. You should talk to your study team about your travel plans and ask if they can reimburse you.
No. In general, it should not directly cost you anything to be in a clinical trial. However, there may be indirect costs you may need to consider. For example, you may need to take time off work or find additional childcare to attend study visits. Or, you may need to postpone or reschedule a vacation or other social commitments to attend study visits.
Some clinical trials may pay a stipend for each study visit or at the end when you complete the study. You should talk to the study team and ask if they provide compensation.
An adverse event is any troublesome medical event that happens to you during a clinical trial. This could include many different signs or symptoms, for example:
- Catching a common cold
- Developing a bruise where the study team performed a blood draw
- Getting a headache sometime after taking the medicine
- Having a blood test come back with an abnormal result
- Tripping and falling while walking around your house
- Feeling very tired
An adverse event may or may not be related to the clinical trial. Even so, all adverse events must be reported. You should tell your study team about any adverse event you experience, even if you don’t think it is related.
Your health and well-being are critically important during a clinical trial. The study team carefully watches patients to ensure they are safe and reports every adverse event. Outside experts, such as clinical monitors and data and safety monitoring boards, monitor compliance and review safety data. Finally, regulatory bodies like the FDA and EMA guide the study teams and must be notified when there are safety concerns.
Yes, you should continue to see your normal doctor(s) while in a clinical trial. You should also let your doctor(s) know that you are taking part in the clinical trial. Sometimes, the study team will want to talk to your doctor or coordinate care with them.
Most clinical trials will provide medical care for urgent issues related to the clinical trial. However, they do not typically provide primary care, comprehensive care, or longer-term care. You should continue to see your normal doctor(s) while taking part in a clinical trial.
Yes, you can decide to stop taking part in a clinical trial at any time and for any reason. This is your right, there is no penalty, and you don’t need to give a reason for your decision.
However, you should carefully consider your ability to commit to the whole clinical trial before agreeing to take part. Clinical trials are expensive and take a lot of time and resources to complete. If too many people quit part-way through, the whole study could be at risk. If you are thinking about quitting, you should talk to your study team and let them know your thoughts or concerns.
What happens after a clinical trial
Maybe. Some clinical trials allow all patients to receive the medicine after they complete the study, even if they were in the placebo group. This is called an open-label extension. Open-label extensions are optional. If you take part, the study team will continue to collect data on you.
Yes, you will get to learn about the results of the clinical trial. However, it could take a long time. The study team must wait for all patients to finish the study before they can analyze the data. Then, it could take 6-18 months to perform the analysis and share the results.
Many study teams will notify patients directly 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 publish their results in scientific journals or present them at conferences.