frequently asked questions about clinical research

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Here is information about research studies that may help potential participants make an informed decision.

What is a research study?
Research studies are designed to answer a specific question about health or illness. A plan is developed to learn if a new medicine, device or procedure is better than what is already available, how well it works and if it is safe. Children’s bodies sometimes respond to medicines and treatments differently than adult bodies. Physicians need to learn if there are effects or safety concerns that are unique to infants and children.

Why participate in a research study?
Study participants may potentially benefit from new treatments. Even if the new treatment is not directly helpful, information learned may lead to better understanding of a diagnosis or disease, which may ultimately benefit others.

What protections are there?
All studies done at Cook Children’s are required to be reviewed and approved by the Cook Children’s Institutional Review Board (IRB) before the study begins, and at least annually for the duration of the study. The IRB consists of community members, physicians, scientists, clergy and others. The IRB’s role is to see that each research study has safeguards in place and is well designed so that study participant rights are protected. The IRB evaluates risks to participants to determine if the risks are reasonable when compared to the potential benefits.

Who can participate in a research study?
Since research studies are designed to answer specific questions, there are strict guidelines regarding who may be enrolled. The investigator will determine who meets those guidelines and will decide whom to invite to participate. No one is included in a research study without his/her written permission.

What happens during a research study?
The steps for conducting a research study are contained in a document known as a protocol. The protocol explains the strict guidelines that have been developed to answer the specific study questions while protecting participants. The research staff will discuss protocol details with a participant and will answer participant’s questions. It is important that participants be thoroughly informed before joining a study. There are common words seen in protocols that may be unfamiliar to you. These include:

Participants who will receive the treatment being studied are part of the study group.

Participants who will receive a standard treatment or placebo are known as the control group.

When there is no standard treatment available to compare to the study treatment, participants may be given a placebo. A placebo looks like the experimental drug but contains no medicine. You may have heard this referred to as a “sugar pill”.

A blinded study is one in which participants, and sometimes the investigators, do not know which group has been assigned which treatment. The study treatment for participants may be a new treatment, standard treatment or a placebo. The study doctor can learn, if necessary, which participant is in which group.

Randomized studies will assign patients to different groups (such as new treatment, standard treatment or a placebo). Researchers us a mathematical process to ensure that each study participant has the same chance of being placed in a particular group, and that each group has a similar mix of age, gender and state of health.

Parents may accompany children to most treatments and procedures.

How does someone decide to join a research study?
Participating in a study is an important decision that should be carefully made. No one joins a study without his/her written permission. Potential study participants should:

• discuss the study with research staff and doctors
• ask questions
• understand answers to questions
• take time to think about participation
• discuss the study with family
• discuss the study with their doctor

Potential study participants should ask:

• How long will study visits last?
• How will daily schedules be affected?
• Will participation require any school to be missed?
• Will regular activities be interrupted?
• Will current medications change?
• Who will answer questions during the study?
• If painful procedures are part of the study, what options are available to help decrease the discomfort?
• What will costs be?
• Will confidentiality be maintained?
• What will happen at the end of the study?
• Will results be available to participants?

No one is included in a research study without his/her written permission.
A participant who agrees to join a study will be asked to sign a consent form. This form should be signed if the participant:

• understands what is expected
• is able to meet the study requirements
• is satisfied with answers to questions
• has had time to make an informed decision

Can a participant leave the study?
Participants may leave a study at any time. The participant should inform research staff of a desire to leave a study, then discuss treatment options with the study doctor.

For more information about Cook Children's pediatric care, community services or opportunities for charitable giving, please call 1-800-934-COOK (2665).