About
Clinical Trials
What is a Clinical Trial?
A clinical trial is a research study that helps doctors, scientists and drug development companies, also known as study sponsors, understand whether a new treatment is safe and effective.
Health authorities, such as the United States Food and Drug Administration (U.S. FDA), use the results of clinical trials to decide if a new treatment can be approved for patients to use outside of research studies. They also use the information to determine for which patients the new treatment is most appropriate.
Clinical trials give patients access to investigational treatments prior to marketing approval, while contributing to research that may help others in the future.
Clinical trials follow carefully designed research plans, called protocols, that are developed before the trials begin.
Protocols determine:
- Who is eligible for the trial
- Which investigational treatments are studied
- What questions the researchers will try to answer
- How participants are cared for throughout the trial
It’s important to note that,
if you choose to participate in a clinical study:
- Your participation is always voluntary
- You will work closely with your, or your child’s, doctor and the research team
- The trial follows strict safety rules set by institutional review boards and health authorities
Clinical Trial Phases
When an investigational treatment is studied, it is evaluated in stages, or “phases.” Each phase assesses the potential benefits and risks of the treatment being studied. This approach provides progressively more data on an investigational treatment’s safety and effectiveness over time. At every phase, doctors, scientists, drug development companies, and health authorities use the data, among other factors, to determine whether the drug program should progress to the next phase.
Phase 1
Tests safety in a small group of healthy volunteers initially, followed by patients.
Goal: Is it safe? What is the correct dose?
Phase 2
Tests safety in more patients and looks for signs of potential benefit.
Goal: Is it safe? Does it show signs of potential benefit, also called effectiveness?
Phase 3
Confirms effectiveness in a larger number of patients, as compared to the benefits and risks of the current standard of medical care.
Goal: Is it safe? Does it work better and/or is safer than the standard of care?
Phase 4
Conducted after a drug or treatment has been approved by regulatory agencies, such as the U.S. FDA.
Goal: To monitor long-term safety and effectiveness, or to study effectiveness in other patients not part of the earlier trials or in other diseases.
In some cases, clinical trial phases may be combined, such as Phase 1/2, to allow researchers to evaluate safety and gather early signs of effectiveness in patients more efficiently.
Interested in exploring whether you or a family member may be eligible for the ABS-1230 Phase 1b/2 clinical trial? Explore eligibility.
KCNT1 Epilepsy Foundation
For more information about KCNT1-related epilepsy, new therapies in development, the latest research information and resources for families and caregivers, please visit the KCNT1 Epilepsy Foundation website. The Foundation provides educational materials and a supportive community for families navigating this rare disease.