Norton Children’s Research Institute
Learn more about participating in a clinical trial.
Clinical trials help researchers find new and better ways to diagnose, treat or prevent diseases in children. These studies have improved treatment outcomes, decreased side effects and more.
Pediatric Phase 1 Clinical Trials
Norton Children’s Research Institute, affiliated with the UofL School of Medicine, offers a wide range of clinical trials for children and young adults in the Kentucky and Southern Indiana region. The clinical trial process is made up of four phases. The first is known as Phase 1 or the “early phase.”
What does Phase 1 mean?
A Phase 1 clinical trial is the first step in evaluating a new treatment in humans. It examines the safety, side effects, best dose and timing of a new treatment. It may also test the best way to give a new treatment (for example, by a pill you swallow, infusion into a vein or injection) and how the treatment affects the body. Phase 1 trials can only occur when there has been demonstrated preclinical safety and efficacy data available from animal studies, modeling or other predictive studies.
Why are Phase 1 clinical trials important?
Phase 1 clinical trials play a critical role in the development of new treatments by evaluating their safety and dosage levels in human participants. For children, phase 1 trials are more commonly tied to severe or life-threatening conditions for which there is no proven treatment or standard therapies have failed. These trials are pivotal, as they provide the first opportunity to assess how a potential treatment interacts with the human body and identify any adverse effects or risks it may pose. Researchers carefully monitor participants to determine the optimal dosage and assess the treatment’s safety profile.
The data obtained from Phase 1 trials inform subsequent phases of clinical testing and guide decisions regarding further development. Additionally, regulatory agencies rely on the findings of Phase 1 trials to assess the treatment’s potential benefits and risks, ultimately influencing its approval process. In summary, Phase 1 clinical trials are essential for ensuring the safety and efficacy of new treatments and advancing them through the clinical development pipeline.
Why should I consider a Phase 1 trial for my child?
Participation in Phase 1 clinical trials has led to significant advances in child health outcomes and treatments. Because children’s bodies are very different from adults’, research tied to development of treatments specific to the needs of children, from infants to teens, is more important than ever.
By participating in a Phase 1 clinical trial, you and your child are at the forefront of groundbreaking research aimed at developing new and more effective treatments for children and teens all over the world.
Additionally, participation in a Phase 1 clinical trial can provide your child with an opportunity to have access to new therapies that may not be available otherwise. Many families also find they have closer collaboration with their health care team and have access to additional education about the child’s condition.
As a participant in a Phase 1 trial, your child will receive careful monitoring, education and support from our team of dedicated medical professionals who prioritize each child’s safety and well-being. For answers to more questions, read our FAQ page.
Current Phase 1 Clinical Trials
Pediatric research is conducted in a multitude of areas. If you are interested in available research trials, talk to your child’s physician about options.
Inpatient clinical trial studies conducted at Norton Children’s Hospital include:
Outpatient clinical trial studies conducted at the Novak Center for Children’s Health include the following conditions:
See all active clinical trials at Norton Children’s Research Institute.