What is a Clinical Trial?

Before joining a clinical trial, it is important to learn as much as possible. Discuss your questions and concerns with members of the team conducting the trial. Also, discuss the trial with your health care provider to determine whether or not the trial is a good option based on your current treatment.

Be sure you understand:
• How long is the trial going to last and what will I be asked to do as a participant?
• How will the treatment be given?
• What are the risks and benefits associated with participating in the trial?
• Will I also receive health care during the trial?
• Do I have to pay for any part of the trial? Does insurance cover these costs?
• Will I be reimbursed for travel costs or childcare?
• Who is conducting the clinical trial?

If you decide to participate, here are the initial steps:
• Visit clinicaltrials.gov website to learn more about the trial details and contact information to the most convenient trial site coordinator
• The site coordinator will send you information about the trial and an informed consent document. The coordinator will schedule an interview to review this information
• If the coordinator determines that you are a candidate for the trial, you will be scheduled to be seen at the clinical trial site for further questions and tests. This is known as a “screening” process to see if you meet all of the criteria for being included in the trial
• If you meet the trial criteria, you will be instructed on how to begin your participation in the trial.

Other things you should know:
• Your participation is voluntary. You can leave at any time.
• There may be some risks, and the experimental drug may not help you.
• It’s a commitment. You may need to visit the research clinic multiple times and have several tests or procedures.

There are many types of trials with varying goals, all of which are very important for improving patient care and outcomes.

• Observational trial, also called a natural history study, is one of the most important and fundamental types of trials for developing a treatment.
• Treatment trials test new therapies such as new drug combinations or new surgical approaches.
• Prevention trials research ways to prevent disease, including medicines, vitamins, vaccines, or lifestyle changes.
• Diagnostic trials research tests or procedures for diagnosing specific diseases or conditions.
• Screening trials identify ways to detect certain diseases or health conditions.
• Quality of Life trials, also called Supportive Care trials, look at ways to improve comfort and the quality of life for people with chronic illness.

The duration of a clinical trial protocol is determined by the minimum length of time in which investigators think they will be able to detect the effect of the experimental treatment. For example, in a Phase 2 trial, a drug designed to stop DUX4 gene expression (measured by biochemical analysis of patients’ muscles) might be expected to show this effect in a relatively short time, perhaps a few months. BUT a Phase 3 trial, which has to show that the drug has an impact on patients’ symptoms (not just their muscle biochemistry), might take several years, because patients show an average rate of decline in function of only 3-5 percent. Such a small change can be obscured by “noise” unless there are a large number of patients and/or a longer study.

Other factors contribute to the overall length of time of a trial, as described below. The two ways in which patients make a critical difference are the first one (the speed with which people volunteer for a clinical trial) and the third (the number who volunteer so that enough people make it through the screening process).

• How quickly volunteers sign up. If a one-year trial needs 24 volunteers and 24 people sign up right away, the trial can collect its data in one year. But if only 1 volunteer signs up each month, it will take 24 months just to get all the volunteers needed, and so it will end up taking three times as long (36 months) to finish collecting the data.
• The trial site has limited capacity to screen volunteers. Clinical trials have to screen volunteers to make sure they meet the criteria for the trial. If the screening process is complicated and requires multiple staff to run tests for each volunteer over several days, or requires the use of equipment that is heavily used by others and so has to be scheduled weeks in advance, etc., the trial site may be able to screen only a few volunteers a month.
• The trial criteria screen out many volunteers. Great care is put into reducing any factors that could “muddy the waters” and make clinical trial data unreliable or hard to interpret. This is why clinical trials typically limit the age of participants, turn away people who have certain illnesses, are unable to perform the tests required in the trial, etc. Thus, several volunteers may have to be screened for each one who makes it into the trial.
• Data analysis takes time. The clinical trial data cannot be properly analyzed until the last patient has completed the trial. And even then, it can take many months before the analysis is completed. Enormous effort goes into making sure both the data and analytical methods are “bullet proof.”

A clinical trial is NOT THE SAME as medical treatment. Understand the differences between clinical research and medical treatment and what those differences mean for you. (Source: FDA, Clinical Research Versus Medical Treatment)

Any experiment involving human volunteers must be conducted with the greatest attention paid to safety and ethics. All clinical trials in the United States must be reviewed and approved by an Institutional Review Board (IRB). This independent committee of professionals and members of the community ensures that risks are low and that the trial is worth the potential benefits.

When studying a new drug, it can sometimes take many years to learn whether a drug actually provides a “clinical benefit”—a positive effect on how a patient survives, feels, or functions. Mindful of the fact that it may take an extended period of time to measure a drug’s intended clinical benefit, in 1992 FDA instituted the Accelerated Approval regulations. These regulations allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint.

A surrogate endpoint used for accelerated approval is a marker: a laboratory measurement of certain molecules (“biomarkers”) in blood or other fluids or tissues, an image (MRI, X-ray, ultrasound), physical sign, or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit. 

The FSHD Society's Therapeutic Accelerator initiative includes projects to validate surrogate endpoints including blood biomarkers and imaging biomarkers.

There is good reason to be concerned, as the internet is full of companies and "doctors" touting miracle cures. Here are some basic guidelines to help you determine if a treatment or trial is legitimate.

Legitimate clinical trials are:

• Listed on clinicaltrials.gov
• Provide an information flyer that has been IRB-approved
• Require that you read and sign an informed consent (also IRB-approved)
• Free to volunteers. You should never be asked to pay in order to participate.

Legitimate treatments:

• Have gone through the FDA approval process
• Have had studies of the treatment's efficacy, side-effects, etc. published in legitimate, peer-reviewed scientific and medical journals
• Have been independently validated (i.e. by researchers with no financial ties to the manufacturer)
• Are manufactured under Good Manufacturing Practice (GMP) standards