Clinical Trials Directory
Clinical Trials Directory

For patients and their families, research means there is hope!

Where there is research for the advancement of treatments and cures, there are accompanying clinical trials. All clinical trials listed on this page are registered with

If you have a pediatric neurologic study that you would like added to this page, click here and send us your trial title and link. CNF is happy to help with promotion and dissemination of your clinical trial opportunity.

Stand Together Against pRolonged Seizures (STARS) Study

UCB is searching for people who experience prolonged epileptic seizures (i.e. lasting more than 3 minutes) to join this clinical research study. The STARS Study is testing an inhaler containing an investigational drug that has been designed to potentially stop a prolonged seizure once it has begun.

Who can join the STARS Study?
Up to 250 people in study sites across the United States, Europe, Asia and Australia are expected to take part in the STARS Study. You and your study partner may be able to take part if you:

  • Are 12 years of age or older
  • Have focal or generalized epilepsy, or a combination of the two
  • Have experienced 2 prolonged seizures within the past 3 months
  • Prolonged seizures last more than 3 minutes
  • Have an adult who can act as a study partner to assist you throughout the study

Visit the website for more information or download the brochure.

CNF is pleased to share this information behalf of UCB BioSciences.

EXPAND clinical research study

Researchers are looking for children and young adults who may be interested in taking part in the EXPAND clinical research study. This study is evaluating how well the body tolerates an investigational medication and whether it works for childhood absence epilepsy (CAE) and juvenile absence epilepsy (JAE).

You or your child may be eligible to participate in the EXPAND Study if you/they:
• are 2–25 years of age
• have been diagnosed with CAE or JAE
• have not had tonic-clonic seizures in the past.

To see if you (or your child) may be able to take part in this absence epilepsy study, learn more here.

Visit the EXPAND webpage for this trial here.

CNF is pleased to share this information behalf of UCB BioSciences.

Gene Therapy Study For Tay-Sachs Disease And Sandhoff Disease (GM2 Gangliosidosis)

Researchers are conducting a clinical trial to learn if an investigational gene therapy called TSHA-101 can help children with Tay-Sachs disease or Sandhoff disease. This will be the first time that TSHA-101 is studied in humans.

The clinical trial will investigate:

  • Safety of TSHA-101 in humans
  • Effectiveness of TSHA-101 in improving survival and symptoms

Every qualified child enrolled in the clinical trial will receive TSHA-101, the one-time investigational gene therapy. For more information contact the research team at [email protected] or visit and search for NCT04798235.

CNF is pleased to share this information behalf Taysha Gene Therapies. 

New Clinical Study Offers Chance to Further Research in Dravet Syndrome

Momentum 1 is a new research study sponsored by Eisai Inc. that is evaluating the safety and efficacy of an investigational drug called lorcaserin. (“Investigational” means that the drug is not approved by the FDA for use in Dravet syndrome or other epilepsies.)  The study is investigating whether adding lorcaserin to a person’s current epilepsy medication will help reduce the number of seizures in people with Dravet syndrome when compared to a sugar pill (placebo). The Momentum 1 study will take place at approximately 25 sites across the US and Canada.

In order to be eligible for participation, your child or your loved one need to meet the following criteria: Age two years and older, and confirmed diagnosis of epilepsy with Dravet syndrome. More information on the study and participating study locations can be found at

CNF is pleased to share this information behalf of Eisai Inc.

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER & LILAC)

Update March 31, 2021 – Four new clinical trial sites for the Phase 3 LAVENDER study are open in: Seattle, WA (Seattle Children’s Hospital), Chapel Hill, NC (University of North Carolina), Baltimore, MD (Kennedy Krieger Institute/ Johns Hopkins School of Medicine) and Bronx, NYC (Montefiore Medical Center). 

LAVENDER is a 12-week study that will evaluate the efficacy and safety of trofinetide and placebo in approximately 180 girls and young women aged 5 to 20 years with Rett syndrome. All girls and young women completing the LAVENDER trial are eligible to enroll in the LILAC study, a 40-week extension study in which all participants receive trofinetide and are followed to evaluate long-term tolerability, safety, and effectiveness of the drug.

Find more information about trofinetide and Rett syndrome here, and more information about the study at the website.

CNF is pleased to share this information on behalf of Acadia, a member of our Corporate Advisory Board

RESPOND: Global Phase 4 study Evaluating Efficacy and safety of SPINRAZA® in patients with a suboptimal clinical response to Zolgensma®

The Phase 4 study will examine the clinical benefit and assess the safety of SPINRAZA® (nusinersen) in infants and children with spinal muscular atrophy (SMA) who still have unmet clinical needs following treatment with gene therapy Zolgensma® (onasemnogene abeparvovec). RESPOND will be conducted at approximately 20 sites worldwide and aims to enroll up to 60 children, up to 3 years old, with SMA.

More information on the study is available at

CNF is pleased to share this information on behalf of Biogen, a member of our Corporate Advisory Board.

Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A

Origin Biosciences is seeking participants for a study to evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A. Children up to the ages of 5 are eligible to participate.

More information and the clinical trial listing is available here.

CNF is pleased to share this information on behalf of Origin Biosciences, a member of our Corporate Advisory Board.

A Combination Retrospective Medical History and Prospective Observational Study of Patients With Canavan Disease for Assessment of Natural History of Canavan Disease

Aspa Therapeutics, a subsidiary of BridgeBio, is currently conducting a Natural History Study for Canavan Disease patients and are seeking participants for several different cohorts, including children under the age of five.

CANInform, the Canavan disease natural history study, will be the first multinational effort to rigorously gather both retrospective and prospective data from this patient population. Data collection will include extraction of retrospective data from medical records of living patients and deceased patients, and collection of prospective, longitudinal data from living patients and their parent(s)/caregiver(s). Motor function assessments will be performed in the home by qualified study team members. In addition, families will be invited to attend clinic visits or will be followed by the clinical site remotely for up to 3 years.

For a description of this study and sites enrolling patients, visit

CNF is pleased to share this information on behalf of BridgeBio, parent company of Origin Biosciences who is a member of our Corporate Advisory Board. 

Creatine Transport Deficiency (CTD)

With only limited prospective longitudinal data available on Creatine Transporter Deficiency (CTD), its natural history is incompletely understood. This international study is designed to provide additional insights into disease progression, to characterize how patients perform on clinical neurodevelopmental assessments, and to evaluate magnetic resonance spectroscopy (MRS) and event-related potentials (ERPs) in patients with CTD.

More information about enrollment. Learn more about CTD from the Association for Creatine Deficiencies.

CNF is pleased to share this information on behalf of Lumos, a member of our Corporate Advisory Board.


ELARA is an open-label study to evaluate the long-term safety, tolerability, and efficacy of ov101 in individuals with Angelman Syndrome. The study is seeking to enroll roughly 82 participants between the ages of 13 Years to 49 years and will evaluate the long-term (52 weeks) safety of OV101 in subjects with AS and provide additional OV101 treatment to those subjects who completed Study OV101-15-001.

More information on inclusion criteria and enrollment can be found here.

Visit the page for this study here.

CNF is pleased to share this information on behalf of OVID Therapeutics, a member of our Corporate Advisory Board.


The ENDYMION study will assess the long-term safety and tolerability of TAK-935 (OV935) as adjunctive therapy in patients with rare epilepsy. Participants must be between the ages of 2 to 65 years of age, and must have participated in a previous TAK-935.

More details can be found here.

CNF is pleased to share this information on behalf of OVID Therapeutics, a member of our Corporate Advisory Board.

Lennox-Gastaut Syndrome (LGS)

LGS Foundation, in partnership with the Epilepsy Genetics Initiative, is piloting a project called LEEP: the LGS/EGI Exome Project, to fund the exomes of a limited number of patients in EGI. The goal is to test the feasibility of funding exomes for patients that cannot get the test covered by their insurance company and to advance gene discovery in LGS. If successful, more families may be able to get exome testing through EGI and more LGS genes may be found. EGI is an initiative created by CURE and NINDS to advance precision medicine in epilepsy. EGI has created a centralized database to hold the exome and genome sequence data of people with epilepsy. The data is analyzed every six months and if a cause is found, it is reported back to the treating physician. Participants may also choose to share their EGI data to advance cutting-edge epilepsy research.

If you are interested in participating, go to and fill out the interest form to receive additional information. Please note: LGS Foundation does not decide who is eligible to enroll. Enrollment is decided by your physician and an EGI genetic counselor. Enrollment is limited and the opportunity is available on a first come, first serve basis.

Tuberous Sclerosis Complex (TSC)

List of current clinical trials/studies related to TSC:

CNF thanks our 2022 partner Ovid Therapeutics for their support.

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