Being Involved

Following the Study Plan

Research that involves people is done following strict scientific and ethical principles. Every trial has a person in charge, usually a doctor, who is called the Principal Investigator, most commonly known as the PI.  The PI prepares a plan for the study, called a protocol. Every clinical trial has a protocol, which acts like a “recipe” for conducting the trial. The same protocol is used by every doctor or research center taking part in the trial. The plan describes what will be done in the study, how it will be conducted, and why each part of the study is necessary. The protocol also describes what types of patients may enter the study, how many people are needed for the study, what information will be gathered about people and how it will be stored, schedules of tests and procedures, drugs, dosages, and length of study, as well as the outcomes (the traits or attributes that will be compared between study groups) that will be measured.  Each person participating in the study must agree to the rules set out by the protocol. Clinical trials follow strict guidelines.

Informed Consent

The informed consent process is meant to help participants understand the study goals, process, risks, and benefits. When a participant visits the study team for the first time, the team discusses the Informed Consent Form. The Informed Consent Form includes details about the clinical trial such as the length of the study, required tests and steps, risks, benefits, side effects, rights as a volunteer, and key contact information. The informed consent process continues throughout the study. If new benefits, risks, or side effects are found during the study, the researchers must tell the participants.

Privacy and Safety

There are many things that the study team does to protect the safety, privacy, and confidentiality of study participants. In the US, clinical trials must be approved and monitored by an Institutional Review Board (IRB), which is an independent committee of medical, research, and community volunteers. An IRB protects the rights of participants by reviewing each protocol to ensure that the trial follows ethical principles, that any risks of participation are minimized, and that the benefits outweigh the risks. If anything serious goes wrong during a study, the researchers have to tell the IRB to help protect participants’ safety.  The IRB can stop a study if the researchers are not following the study protocol, if the trial is causing unexpected harm to participants, or if the new treatment is obviously working (so that the treatment can be made available to everyone).

The Importance of Committing to the Trial 

A successful trial is a shared effort between the research team and the participants. It is extremely important that study participants take their involvement in the trial very seriously.  Participating in a trial is a commitment that researchers and the community count on—remember, the study participants are as important as the researchers and the therapy to the success of a trial. Research studies are often very expensive and researchers may not get a second chance. Studies have failed because they do not have enough participants, and because participant drop out made it impossible to say if a potential new treatment would work.  In those cases, everyone loses.
If you think that you are not going to be able to stay in the study until the end, enrolling in a study is not the best decision for you. Clinical trials have limited space.  Though researchers often enroll slightly more participants than they need, everyone who is selected for the trial is truly important to determining if a potential new treatment has benefit.  If you are unsure about your ability to commit to the trial, do not take the spot of someone who will participate in the trial to the end.
Document adapted from Duchenne Connect written by Holly Peay (Parent Project Muscular Dystrophy)