Clinical Trials

Clinical Trials

CNF believes Clinical Trials are essential to finding new treatments and offering hope to children with neurologic conditions. 

Families in our community told us they needed help understanding and finding clinical trials. Clinicians told us they needed resources to help families find and evaluate clinical trials. Our goal is to provide an educational hub with helpful and reliable information that addresses these needs for both communities.

Why should I consider having my child participate in a clinical trial?

Joining a clinical trial can be a rewarding experience. Children and their families who join studies may: 

• Learn about new medicines and treatments 
• See specialized doctors and learn more about the child’s condition 
• Help other children and contribute to the advancement of treatments 

Clinical trials also have risks. So, it is essential to learn everything you can about any clinical trial you consider.

Are clinical trials suitable for my child?

Deciding if a clinical trial is a good option for your family and child is a personal choice. It involves many considerations. There is no right or wrong answer. 

• Part of the clinical trial process is “informed consent.” This makes sure parents are fully informed and can ask questions before starting the trial. See the Courageous Parents Network resources below for more information on informed consent. These resources may also help you evaluate a trial.  
• By enrolling in a clinical trial, you may be able to connect with other families. Finding others who are in a similar situation can be incredibly helpful.  
• Talking to your child’s medical providers can provide more information to help you make a decision. 

What should I ask my doctor about a clinical trial?

Your child’s doctor may recommend a clinical trial for you to explore. Don’t hesitate to ask them if they know of a trial or to share a clinical trial you found. Your doctor may not know about every trial available. See below for ideas on how to find clinical trials. 

Your doctor cannot tell you if you will qualify for a clinical trial. However, they can help you develop a list of questions to ask when you contact the clinical trial coordinators. They can also answer questions you may have after talking to the clinical trial coordinators. 

What questions should I ask researchers to help guide my decision? 

• What is the purpose of the trial? 
• What kinds of tests and treatments are involved? 
• How much time will be required and how long will the trial take? 
• What are the possible side effects or risks? 
• What happens if I want to take my child out of the trial? 
• Will there be any costs we will have to pay? 
• How will the doctor know if the treatment is working? 
• How and when will we be told about the trial’s results? 

How can I learn about clinical trials specific to my child’s condition?  

• Use the CNF Disorder Directory to: 
  • Learn more about your child’s disorder 
  • Get links to available trials 
  • Connect with advocacy organizations
• If your child does not currently have a diagnosis, consider visiting the Undiagnosed Disease Network and applying to be a part of their research study. 
• The Rare Diseases Clinical Research Network (RDCRN) is a national network of top rare disease researchers. It is powered by patient partnerships and funded by the National Institutes of Health. The RDCRN Contact Registry collects and stores the contact information of people who want to participate in RDCRN-sponsored research or learn more about their research. 
• RARE-X is a collaborative platform for global data sharing and analysis to accelerate treatments for rare diseases.   

Where can I find clinical trials?  

• ClinicalTrials.gov is a database of privately and publicly funded clinical studies conducted worldwide. 
• The Center for Information and Study on Clinical Research Participation (CISCRP) has a free service to help people find clinical trials. Visit their Search Clinical Trials page to learn more. 
• Trials Today is a website created by ResearchMatch that asks a few short questions to create a list of studies on ClinicalTrials.gov that may interest you. 

Where can I learn more about how clinical trials work and what participation involves?  

• Courageous Parent Network is a non-profit organization and educational platform that orients, empowers, and accompanies families and providers caring for children with serious illnesses. Helpful resources include: 
  • Evaluating the Clinical Trial Option: Questions to Consider (printable PDF) 
  • Clinical Trial Unit  
  • CPN Evaluating the Clinical Trial Option (video)
• The Center for Information and Study on Clinical Research Participation (CISCRP) has information in its Clinical Trial Education Center.  
• How Do Clinical Trials Work (video)

Thank you for our 2022 Funding Partners

Industry Partners 

• Jazz Pharmaceuticals  
• Ovid Therapeutics  
• Praxis Precision Medicines 
• PTC Therapeutics 
• REGENXBIO 
• UCB 
• VNS Therapy by LivaNova  

Advocate Partners 

• Epilepsy Foundation 
• International Foundation for CDKL5 Research  
• Dravet Syndrome Foundation  
• Phelan-McDermid Syndrome Foundation 
• Epilepsy Alliance of America 
• NeurAbilities Healthcare 
• Pediatric Epilepsy Research Foundation 

The information in the CNF Clinical Trials section is not intended to provide diagnosis, treatment, or medical advice and should not be considered a substitute for advice from a healthcare professional. Content provided is for informational purposes only. CNF is not responsible for actions taken based on the information included on this webpage. Please consult with a physician or other healthcare professional regarding any medical or health related diagnosis or treatment options.