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Finding new treatments through clinical trials

Children's Mercy has participated in industry-sponsored clinical trials for more than 25 years, working with nearly 60 top pharmaceutical sponsors in a vast range of clinical areas. We participate in robust pediatric clinical research studies, including pediatric pharmacological and drug-therapy trials sponsored by industry, local, and state agencies and the federal government.

What research means to Myah, her family, and her providers

From the outside, Myah looks like an ordinary teen who loves playing soccer. But Myah has Cystic Fibrosis and that makes running around and keeping up with her teammates difficult. Learn why Myah and her family decided to get her involved in research at Children’s Mercy and why Cystic Fibrosis research is so important to her and her providers.

What families need to know about research

Before you decide to have your child participate in a clinical trial, there are several things to consider. It is important to know that the studies are optional, and your child will continue to receive the best possible care whether you choose to participate in a clinical trial opportunity. All research studies are done with the approval and oversight of an ethics committee called the Institutional Review Board.

Learn more from the National Institutes of Health.

Active research projects

Active research projects led by the Children's Mercy Research Institute improve the heath of children everywhere. Learn about projects from our flagship Genomic Answers For Kids work to Talk With Me Baby, their impact and how to support research.

See our active research projects.

Clinical Trials are an important part of research at Children's Mercy. Each year, our researchers participate in more than 100 active clinical trials. Through these endeavors, we have seen a significant increase in grant submissions and research awards received from National Institutes of Health, foundations and the private sector. 

See our active clinical trials.

Pioneers Recruitment Registry

The Pioneers Recruitment Registry is a network of hospitals and doctors located in Kansas and Western Missouri seeking volunteers to participate in clinical trials. 

Learn about the Pioneers Recruitment Registry.

Frequently asked questions about clinical trials

There are benefits and risks associated with clinical trials. By participating in a clinical trial, your child can:

  • Take an active role in improving health care for children

  • Have access to experimental drugs that are not otherwise available

  • Help others by contributing to medical research

Clinical trials have risks:

  • There may be side effects or adverse reactions to drugs that are not known

  • The protocol may require a lot of your time for trips to the study site, study interventions, hospital stays or complex-dosage requirements

The main purpose of a research study is to gain new knowledge that will help doctors and nurses provide better medical care for children. Sometimes being in a study will help your child and sometimes it does not help your child directly. What doctors learn from research may help other children in the future.

If your child takes part in a clinical trial, he or she will receive interventional treatment that otherwise would not have been administered as part of the normal care plan. This may make your child feel better, have no effect or it could possibly cause side effects. Most of the time, these side effects are very mild. In rare cases they could be serious. If your child is asked to take part in a clinical trial involving an experimental drug or device, make sure you are told what phase of trial it is. This will give you a better idea of what is involved and the level of risk your child is taking. The researcher must tell you everything they know about possible side effects. You and your child can then decide whether to participate.

Pharmaceutical trials are divided into four phases.

Phase 1 trials
In Phase 1 clinical trials, researchers test a new drug in a small group of people (20 - 80) for the first time to evaluate its safety, determine a safe dosage range and identify side effects. This research is often conducted initially with healthy adult volunteers.

Phase 2 trials
If a new treatment is determined safe after a Phase 1 trial, it will continue to Phase 2. In Phase 2 clinical trials, the drug is given to a larger group of people (40 - 100) to determine effectiveness and safety. 

Taking part in a Phase 2 study has fewer risks than Phase 1. By this point, researchers will know a lot more about the effects of the drug on humans and will be ready to test it on a larger group of patients or healthy volunteers.

During a Phase 2 study, the researcher will be looking at:

  • Minor side effects of the drug

  • How well the drug works

  • And possibly, alternative ways of giving the drug (i.e. injection versus tablets)

Phase 3 trials
In Phase 3 studies, the drug is given to large groups of people (more than 200) to further determine its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug to be used safely. Much more is now known about the new drug. For this reason, these trials involve a larger number of subjects - often hundreds or even thousands - from many different hospitals.

The main purpose of Phase 3 studies is to compare the new drug with the most commonly used existing treatment.

Researchers may want to find out:

  • Whether the experimental drug is more effective than the existing treatment

  • Whether the experimental drug has more or fewer side effects than the existing treatment

  • The long-term benefit of the experimental drug to a subject

  • Whether a subject's quality of life is changed by the experimental drug

Phase 3 studies are always randomized. This means splitting patients into two or more groups, each of which will receive a different intervention. The purpose is to compare the new drug to existing treatments for the same condition.

Phase 4 trials
Phase 4 studies are conducted after the drug has been approved to be marketed by the Food and Drug Administration (FDA). These studies continue testing the drug to collect information about its effect in various populations and any side effects associated with long-term use.

Once the three phases of a clinical trial are complete, the FDA reviews information from all the studies to see if the drug can be approved.

If the FDA approves it, the new medication will go on sale. At this point the pharmaceutical company might fund a Phase 4 trial. The main purpose of this type of trial is to promote the drug and bring it to the attention of more doctors. There are few risks involved in a phase 4 trial because much is already known about the treatment. This kind of trial is of less scientific importance than earlier phases.

Your child's medical information will be kept private and only be identified by a unique study number of initials so that no one outside of Children's Mercy Kansas City will know who your child is. For some research, personal information will be shared with other trusted people like the Food and Drug Administration or a company which may have asked your child's doctor to do the research. If this is done, your child will be identified only by the study number or initials.

All clinical trials are based on a set of rules called a protocol. A protocol is a scientific paper that describes what types of people may participate in the trial, the schedule of tests, procedures, medications, dosages and the length of the study.

This is the most important part of the study. Informed consent means providing parents and children with the necessary facts about a clinical trial before deciding whether to participate. A form will be given to you explaining things such:

  • Why the research is being conducted

  • What the researchers want to accomplish

  • What will be done during the trial and for how long

  • What risks are involved in the trial

  • What benefits can be expected from the trial

  • What other treatments are available

  • The fact that you have the right to leave the trial at any time

  • What identifiable information will be used and who will have access to it

If you are considering a clinical trial for your child, the research staff will give you and your child - if he or she is old enough - an informed permission/assent document with details about the study.

It is also a good idea to take the permission/assent documents home and discuss them with family members or friends. Talking about the options can help you and your child feel comfortable with your decision. If you decide to have your child join the clinical trial, be sure the staff provides you with a copy of the form you sign as a reference for you and your child during the trial.

Informed parental permission and child assent is more than signing a form. It is a process that continues through the study. You and your child are free to ask the research team questions throughout the study.

A placebo is an inactive substance that has no treatment value. In drug studies they are known as "sugar pills." In clinical trials, experimental drugs are often compared with placebos to assess the effectiveness of the experimental drug. In some studies, the participants in the control group will receive a placebo instead of an active drug.

A control group is the standard by which experimental observations are evaluated. In many clinical trials, one group of subjects will be given an experimental drug and the control group is given either a standard treatment for the illness or a placebo.

A blinded or masked study is one in which participants do not know whether they are in the experimental or control group in a research study. Those in the experimental group get the medications being tested, and those in the control group get a standard treatment or no treatment. The study staff usually knows which subject is in which group.

A double-blind or double-masked study is one in which neither the participants nor the study staff know which participants are receiving the experimental drug and which ones are receiving either a standard treatment or a placebo. These studies are performed so that neither the patients' nor the doctors' expectations about the experimental drug can influence the outcome.

For safety, a member of the scientific team at the company carrying out the protocol monitors what group each participant is in. If there is a question and the study doctor needs to know which group a subject is in, this information can be "un-blinded" under very strict rules by that safety member.

Side effects are any undesired effects of a drug or treatment. Negative or adverse effects may include minor events such as a headache, nausea, hair loss, skin irritation or major events such as liver or kidney damage or other physical problems. Experimental interventions must be evaluated for both immediate and long-term side effects.