Clinical Trials Directory

For young patients and their families, research means There is help! There is hope!

Where there is research for the advancement of treatments and cures, there are accompanying clinical trials. Educational information on clinical trials can be found on our Patient Participation in Clinical Trials landing page.

This page includes clinical trials sponsored or submitted by our partners. All clinical trials listed on this page are registered with Trials are also listed in our Disorder Directory under the “resources” tabs of specific disorders.

If you have information about a current pediatric neurologic study that you would like added to our Clinical Trials page, please click here to send us your trial title and link. Child Neurology Foundation can help with promotion and dissemination of your study information.


CMS-001 Clinical Study: A clinical study for patients with Congenital Myasthenic Syndromes (CMS)

Catalyst Pharmaceuticals and your doctor are trying to learn if the oral investigational medicine – amifampridine phosphate – can be safe and effective for the treatment of people with CMS.

WHAT do You Need to Know?

Learn more at

The sole purpose of the HERO website is to make it easier for people to find and enroll in clinical studies and advance epilepsy research.

Insys Developmental Company is conducting a trial with a synthetic CBD product in children with Infantile Spasms aged 3 to 36 months refractory to steroids and vigabatrin. Learn more at

Current study or fellowship opportunity relating to pediatric stroke:

TSA logo

List of current clinical trials/studies related to TSC:






In The NPC Study, researchers are investigating a new drug candidate for Niemann-Pick Disease Type C. This drug, VTS-270, is a unique form of cyclodextrin. It has shown promise in preclinical and Phase 1 clinical studies. To learn more please visit