Clinical Trials

Frequently Asked Questions

• Why participate in a clinical trial?

  Participating in a trial may help accelerate the development of new treatments, increase knowledge about a disease or even lead to a cure. In addition, trial participants often feel empowered as trials offer another way to fight back against disease. It is important to remember that some trials may provide participants with direct medical benefits, while others may not or may even result in harm to participants.

• Who can participate in a clinical trial?

  People of all ages, races, ethnicities and genders can participate. Each study has its own inclusion and exclusion criteria to determine who is an appropriate candidate.

• Who is on a clinical trial team?

  Clinical trials are run at institutions and hospitals by a clinical trial team. Each trial is led by a principal investigator, often a medical doctor, and research teams often include doctors, nurses, study coordinators, social workers and other health care professionals.

• What are my rights as a clinical trial participant?

  As a potential participant, you have a right to receive a written description of the trial specifics and what will be expected of you. You will also have the right to ask questions of the study team until you are comfortable with your understanding of the study, and the right to take time to consider whether participation makes sense for you. You will have every opportunity to ask questions, even after you give consent to participate. Once the study begins, you have a right to withdraw from the study at any time, and the right to learn about new risks or findings as they emerge.

• What are my responsibilities as a participant?

  You are required to comply with the study protocol (visits, treatment, keeping notes, etc.). Tell study investigators about any possible side effects, as well as any hospital, ER and outpatient clinic visits. Do not conduct your own experiments, as you may invalidate study results. It’s also important to maintain confidentiality. Sharing impressions of study experiences can bias study investigators and participants and compromise the integrity of the study. 

• What can I talk about?

  You may discuss any questions or concerns with the study coordinators or principal investigator. The study coordinators and investigators are obligated to maintain your confidentiality and the confidentiality of other participants.

• What can’t I talk about?

  You should not talk about whether you think you are receiving a placebo or drug. However, if you have a concern about the treatment you are receiving, you should always contact the study coordinator or principal investigator.

• What is a placebo and why is it used in clinical trials?

  A placebo, or dummy pill, is an inactive version of the treatment being tested in a clinical trial. Placebos are designed to look exactly like the test treatment, but they lack the active chemical ingredient. Placebos are used to ensure that any measured benefit of the test treatment is due to the active chemical ingredient and not simply to the act of participating in the study.
  
  Not every clinical trial uses a placebo group, but in some studies, a subset of participants will receive a placebo for all or for part of the study. Participants are assigned to either a treatment or placebo group, often by random chance, and are not able to influence their group assignment.

• What if I enrolled but now don’t want to or can’t continue?

  You can withdraw or decide not to participate at any time. If you withdraw from the study, it is very important to contact your study coordinator and inform them of your decision. If you were taking a study medication, the study team likely will request a final visit with you to ensure you are OK. Your health and well-being come first in research.

• Will insurance pay for treatment associated with a clinical trial?

  Some trials do bill insurance. In many cases, treatment received in connection with a clinical trial will be covered. This should be disclosed in the study consent form.
  
  Some studies provide financial support for costs associated with traveling to the clinic or hospital during the trial. Ask the clinical trial team if these resources are available.

• Why is genetic testing important with regard to clinical trials?

  Many studies require genetic confirmation of a disease before a person can participate in a clinical trial. Genetic testing can pinpoint an individual’s disease-causing mutation, helping investigators ensure that all people enrolled in the study are similar. 
  
  Use this free tool from the National Society of Genetic Counselors to find a genetic counselor for you.

• How does the MDA Neuromuscular Disease Registry support clinical research?

  The MDA U.S. Neuromuscular Disease Registry, launched in 2013, is the first comprehensive registry for ALS, BMD, DMD and SMA that collects information from health professionals through MDA’s network of more than 150 Care Centers. This registry serves as a powerful tool to help improve quality of life and survival, facilitate research, and expedite clinical trials.

* Clinical trial information originally written for “Types of Clinical Trials” and “Trial Run: Is Participating in a Clinical Trial Right for Me?” in MDA’s Quest Magazine.