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Clinical research FAQs

Cook Children's Clinical Research FAQsWhether this is the first time participating in a clinical trial or your child is an old pro, we understand that you might have questions. Please read the questions and answers below. If you still have questions, feel free to contact us.

What happens during a clinical trial?

At the beginning of a trial, the research team-including doctors, nurses and other health care professionals-checks your child's health and tells you everything you need to know about taking part in the clinical trial. This health check may involve a physical exam and screening tests.

Children who are eligible for the trial and agree to participate are asked to sign an informed consent form (link to glossary) to become officially enrolled. In the meantime, the research team continues to carefully check the participant's health during the trial and for a period of time after the trial is finished.

Some clinical trials require more tests and doctor visits than usual. For the trial to be successful, it is important for participants do everything asked of them and to stay in touch with the research team.

Who can participate in a clinical trial?

All clinical trials have guidelines about who can join. Some enroll healthy people. Others enroll only people with certain health conditions. These guidelines help make sure the research results will be accurate and useful. If a trial's guidelines match your condition, you may be eligible to join.

To find the right people to enroll in research and to keep them safe, researchers must look at reasons for including and excluding people from trials. Inclusion criteria are traits everyone must have to be in a certain trial. For example, a trial for a new cystic fibrosis (CF) medication would require that all participants have been diagnosed with CF. Exclusion criteria are traits people cannot have in order to be in a certain trial. For instance, a trial for medication to clear mucus from the airways of young children would exclude adults.

Will my child's health care team stay involved in my care?

Yes, we encourage you to discuss trial participation with your health care team.

What do words like protocol, placebo, randomized and control group mean?

A protocol is a detailed plan for a clinical trial. To help answer certain research questions, this plan is carefully put together to protect the health of participants.

A protocol outlines:

A placebo is an inactive medicine that will not affect a person in any way. It is a sugar pill. In clinical trials, people taking experimental drugs are often compared with those taking placebo drugs to learn how well a drug really works.

When a patient is randomized, it means that he/she is assigned by chance to one of the treatment groups. This helps make sure that each study participant has an equal chance of receiving the study drug or the placebo.

In many clinical trials, one group of patients is given the experimental drug or treatment, while the control group is given standard treatment or a placebo.

These and many other commonly used research terms are defined in the Glossary section.

What are the different phases of clinical trials?

The process of drug development begins in a laboratory. New ways to treat diseases are tested for several years-first in the test tube, then in living systems or models. These early tests are called preclinical research. If this research proves promising, researchers will ask for approval from the Food and Drug Administration (FDA) to begin clinical trials with patients.

Clinical trials with patients are done in phases. Trials at each phase are done for different reasons and help scientists answer different questions.

  1. In Phase 1 trials, researchers test an experimental drug or treatment in a small group of people to 1) learn if it is safe, 2) find a safe and tolerable dosage range, 3) decide how to administer the drug (i.e., orally, intravenously, inhaled, etc.) and 4) learn the side effects.
  2. In Phase 2 trials, the experimental drug or treatment is given to a larger group of people to see how well it works (i.e., efficacy) and to keep testing its safety. At this stage, the trials do not provide enough information to know whether or not a drug works to treat an illness, but people may report some benefits.
  3. In Phase 3 trials, the experimental drug or treatment is given to large groups of people to 1) continue testing how well it works, 2) determine dosage amounts, 3) watch for side effects, 4) compare it with commonly used treatments and 5) collect information regarding its safety. The drug is usually put on the market after Phase 3 trials are completed and the FDA approves the drug.
  4. Phase 4 trials provide more details about a drug, including how well it works over a long period and how it affects people's quality of life. They also look at how much a drug costs compared with how well it works and may compare that with other new and standard treatments.

What are some of the benefits and risks of participating in clinical trials?

There are both possible benefits and risks for your child participating in clinical trials.

Possible benefits include:

Possible risks include:

Can my child leave a clinical trial after joining?

Yes, your child can leave a clinical trial at any time. If you choose to leave or withdraw from a trial, you should let the research team know. They may ask you to come for a final visit.

How safe are clinical trials?

Nothing is more important than safety in developing new disease treatments. There are four layers of protection in every clinical trial.

Each trial must be determined as safe and appropriate for patients by the:

How is my child's safety protected?

The United States government has strict guidelines and safeguards to help protect people who choose to participate in clinical research. Every clinical trial in the United States must be approved and monitored by an IRB. This is to keep risks as low as possible and ensure that the risks are worth any potential benefits.

The IRB is usually made up of doctors and the general public. They look at each trial's protocol (a clear and detailed plan of the experiment) to make sure that participants' rights are protected and the trial does not cause them unnecessary risk.

In the United States, anyone participating in a clinical trial must sign an informed consent form. This form explains the trial in full, including the risks, and a research team member will explain the trial and the consent form to individuals before they sign it.

How long does each phase of a clinical trial last and what are the risks?

It usually takes an average of five to 10 years to take a new drug from the start of a clinical trial to the day it's approved.

Generally speaking:

Each phase carries a risk that the drug might not work or may have unwanted side effects. Only one in five drugs tested on humans ever becomes available to the public.

How do you choose which drugs will be tested in clinical trials?

Because only one in five drugs tested ever becomes available to the public, it's important to have many different trials at the same time. And, with both money and participants for clinical trials in short supply, it is important to choose those drugs carefully.

At what age can someone join a clinical trial?

The age that someone can participate varies for each trial. The age range depends on a number of factors, with safety always first in mind.

In clinical trials where participants take medications, drugs must be shown to be safe and work in older patients before they can be tested in young children. Also, young children metabolize and break down drugs differently than adults, so it is important to test drugs in children as well as adults.

Who at my health care center should I ask about clinical trials?

You can ask your child's doctor or any other member of the health care team about clinical trials. Also, you can ask to talk with the research coordinator at your care center who can answer questions about studies and trials.

What questions should I ask about my child participating in a clinical trial?

When you speak with your child's doctor or the research coordinator at your care center, consider asking the following questions:

Will my child get early access to drugs if participating in clinical trials?

Trial participants-even those who received a placebo at first-sometimes get to take the test drug for a period of time. Ask the trial's contact person to see if this is the case in the trial you're considering.

My child has participated in trials and we have never heard the results. Why?

It's very important to communicate results with people who participate in trials. However, there are several reasons why trial results may be released either very slowly or not at all.

Who decides how much money clinical trial participants are paid?

How much money is paid to participants, if anything, is up to the trial sponsor. The amount of payment often depends on the kind of trial and what participants will be asked to do. Some researchers want to pay more so that it does not take as long to recruit participants. Others feel that money should not be offered.

Please note, because accepting payments for participation may affect your continued eligibility for Supplemental Security Income (SSI), Medicaid or Medicare low-income subsidies, you should talk to your research coordinator before participating in a clinical trial that offers payment for participation.

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