Patients, Families and Researchers Team Up for New Treatments

By Rachel Katz and Vera Shively, Lurie Children's Division of Genetics, Birth Defects and Metabolism

What does it take to go from cutting-edge science in the laboratory to new treatments for patients? Clinical trials are the way this is done. There are many clinical trials ongoing at Lurie Children's, each one tailored to a specific healthcare need and providing hope for a better future.

Our Kids Make a Big Difference

A young boy with a big smile and lively personality travels with his parents from a nearby state to Lurie Children's every month to participate in a clinical trial. He has a rare genetic condition that affects the bones and spine, as well as other tissues and organs in his body. The condition makes it hard for him to keep up with other children his age. The courage and dedication of children and families makes it possible to have improved treatments. 

What Is a Clinical Trial?

A clinical trial is a research study performed to determine if a treatment will be effective and safe in people. It is important to know that many years of research are done in the laboratory before studies are ever done in people. In addition, people in a clinical trial must agree, or consent, to be a participant. Participation in a clinical trial is voluntary. For a child, a parent or guardian may consent on their behalf.

The goal of a clinical trial is to find out if a new treatment (intervention) is safe and effective. Interventions may be new drugs, medical devices, surgical procedures or behavioral therapies. Some clinical trials compare a new way to treat a health problem with the current method that is already available. Other clinical trials compare a new intervention, like a new medicine, to a placebo. A placebo is a substance made to resemble a drug but contains no active drug ingredients. They are an important way to see if changes in health outcomes are due to a new medicine or not. In all cases, clinical trials are done according to a specific research plan that is designed to carefully collect the data needed to determine whether a new intervention is safe and leads to improved health outcomes.

A Clinical Trial Has Four Phases

The four phases, or parts, to a clinical trial are:

• Phase 1 is sometimes referred to as a safety study. Researchers want to find the dose range of an intervention, like a drug, that can be given safely. They collect safety data, which means they record any adverse effects of the intervention.
• Phase 2 is where the intervention is tested in people who are affected by a condition or illness. Here researchers are looking at efficacy, or the effectiveness of an intervention. Are participants seeing improvements in their health? Which dose works best and has the fewest side effects? Safety data is also collected in this phase.
• Phase 3 consists of the long-term collection of safety and efficacy data in an expanded group of participants. Once phase 3 is completed, the data collected in the trial can be submitted to the Food and Drug Administration (FDA) to be approved for others with the same condition or illness.
• Phase 4 occurs only after the FDA has approved the intervention. It consists of ongoing, long-term collection of safety and efficacy data.

A Long Process

New drugs take on average 12 years to go from pre-clinical (laboratory) studies to FDA approval. This includes the clinical trials in humans. In addition, only about one in 1,000 drugs progress from pre-clinical studies to clinical trials (Van Norman 2016).

Clinical trials take teams of patients, families and researchers working together to develop new and improved treatments for all. We appreciate their spirit and dedication!

Learn More About Research at Lurie Children's

Additional Information

• Clinical trials at Lurie Children's
• FDA Approval Process
• Van Norman, G. A. (2016). Drugs, devices, and the FDA: Part 1: An overview of approval processes for drugs. J Am Coll Cardiol Basic Trans Science, 1, 170-179.