Cure in Progress – December 2025

Lauren and I just got back from the American Epilepsy Society meeting where we met with four biopharma companies running clinical trials open to people with GRI Disorders. We’re thrilled that approximately 70% of our patient population is currently eligible for one or more trials, specifically those who fall into one of the following two categories:

• People with GRIN, GRIA, GRIK, and GRID Disorders who have at least four seizures per month and / or occasional seizures of three minutes or more. 
• People with Gain of Function GRIN Disorders up to age 18, regardless of whether or not they have epilepsy.

So which trial is right for you? CureGRIN has built a simple screener to help you determine which of the four clinical trials might be open to you. Responses entered into the chatbot are only used to determine potential eligibility and are not stored.

Screener: https://landbot.online/v3/H-3269524-YX332RPUAK4VB09O/index.html

Understanding Placebo-Controlled Clinical Trials

A Phase 3 clinical trial is the final and largest testing stage before a medicine can be approved by health authorities like the US Food and Drug Administration or European Medicines Agency. This stage confirms the medicine works and monitors for side effects in a large group of people.

To make sure the results are scientifically sound, these trials are designed to be placebo-controlled. This means participants are randomly assigned to receive either the investigational medicine or a placebo (a dummy pill with no active drug). By comparing these two groups, researchers can figure out the medicine’s true effect.

But even if your child starts on a placebo, they will gain access to the active drug in a subsequent open-label extension phase in any of the four trials listed here.

How do we know if we are Gain or Loss of Function?

Determining whether a GRI variant is Gain-of-Function (GoF—too active) or Loss-of-Function (LoF—not active enough) is often the first step in seeing if a child qualifies for a gene-specific trial like Radiprodil.

• For US Patients: GRIN Therapeutics partners with InformedDNA to offer free genetic counseling to help determine GoF / LoF status and trial eligibility.
  • Call: 888-503-7727 to schedule an appointment.
  • Website: https://informeddna.com/grin/
• For International Patients: Other families can contact us at info@curegrin.org for help in determining if functional analysis has been performed on their gene.

What if we don’t qualify for Clinical Trials?

If your specific diagnosis does not meet the criteria for the trials listed below, please know that work is underway to include you in future opportunities:

• GRIN Loss of Function/Undetermined: GRIN Therapeutics is actively working on a future trial that will apply to this patient population. Note: This study is not yet recruiting and details are currently available but we will share more information when we have it.
• GRIA, GRIK, and GRID Disorders: CureGRIN is in active discussions with multiple drug-makers about additional trials for all GRI disorders (GRIN, GRIA, GRIK, and GRID), encompassing both genetic and pharmaceutical approaches.

What are the trials? 

Here are the high level details on the four trials currently recruiting GRI patients. Note that all of these trials have additional exclusion criteria that will prevent some patients from participating.

The Radiprodil Study (NCT07224581)

Radiprodil targets the NMDA receptor, which is created from DNA instructions contained in the GRIN genes. As a result, this drug is hoped to be more effective than general epilepsy drugs like Keptra and Valproic Acid. A smaller, earlier trial showed promise in treating GRIN-related epilepsy and other symptoms.

The Relutrigine Study (NCT07010471)

This trial focuses on people with Developmental and Epileptic Encephalopathies (DEEs) which include GRIN, GRIA, GRIK and GRID Disorders.  

• Drug Candidate: Relutrigine
• Clinical Sponsor: Praxis
• Trial Name: EMERALD
• Gene Eligibility: All GRI Disorders (GRIN, GRIA, GRIK, and GRID)
• Age Eligibility: 2 years to 65 years
• Epilepsy Requirement: Yes. Minimum of 4 countable seizures per month.
• Will patients on placebo get access to the drug candidate? Yes.If a child receives the placebo, they will still gain access to the active Relutrigine drug once the first 16 weeks of the study are finished.
• Sites Currently Open (Recruiting): US (Maryland). Study is decentralized so patients anywhere in US can participate.
• Sites Coming: Australia, Brazil, Germany, Italy
• To Learn More: To learn more about the DEEp Ocean study, you can visit the study-specific website: https://www.resiliencestudies.com/emerald

The LP352 Study (NCT06719141)

This trial focuses on people with Developmental and Epileptic Encephalopathies (DEEs) which include GRIN, GRIA, GRIK and GRID Disorders.  

• Drug Candidate: Bexicaserin
• Clinical Sponsor: Lundbeck (previously Longboard)
• Trial Name: DEEp Ocean
• Gene Eligibility: All GRI Disorders (GRIN, GRIA, GRIK, and GRID)
• Age Eligibility: 2 to 65 years
• Epilepsy Requirement: Yes. Minimum of 4 countable seizures per month.
• Will patients on placebo get access to the drug candidate? Yes. Patients who receive the placebo will transition to receiving active Bexicaserin once the first 17 weeks of the study are finished.
• Sites Currently Open (Recruiting): US: (Arkansas, California, Colorado, Florida, Georgia, Idaho, Iowa, Maryland, Massachusetts, Minnesota, New Jersey, Oregon, Tennessee, Texas, Washington. Europe (sites in France, Germany, Italy, Latvia, Netherlands, Portugal, Serbia, Spain, UK), Asia-Pacific (sites in Australia, China).
• To Learn More: To learn more about the DEEp Ocean study, you can visit the study-specific websitehttps://deepdeestudy.com/ 

The Staccato Alprazolam Study (NCT05077904)

This study is testing a special, quick-to-breathe-in medicine to see if it can quickly stop long-lasting seizures.

• Drug Candidate: Staccato Alprazolam
• Clinical Sponsor: UCB
• Trial Name: STARS
• Gene Eligibility: All patients with the required seizure type (not gene-specific)
• Age Eligibility: 12 years of age and older 
• Seizure Requirement: Yes. Patients must have occasional prolonged seizures such as a focal seizure of at least 3 minutes, or a cluster of myoclonic/absence seizures for at least 5 minutes.
• Will patients on placebo get access to the drug candidate? All participants in the follow-up STARS Extension trial (NCT05076617) receive the active drug for home use.
• Sites Currently Open (Recruiting): USA (Alabama, California, Colorado, Connecticut, Florida, Georgia, Illinois, Indiana, Kentucky, Louisiana, Massachusetts, Maryland, Michigan, Minnesota, Missouri, North Carolina, Nebraska, New Jersey, New York, Ohio, Oklahoma, Pennsylvania, South Carolina, Tennessee, Texas, Utah, Virginia, Washington, Wisconsin), Canada, Australia, Europe (Austria, Czechia, Finland, France, Germany, Israel, Netherlands, Poland, Spain, Switzerland, UK).
• To Learn More: Contact Information: To learn more about the STARS study, you can visit the study-specific website www.starsepilepsystudy.com.

CureGRIN’s goal is to help GRIN Therapeutics, Praxis, Lundbeck and UCB to enroll 100-125 GRI patients across these four trials. We encourage each family to review trial details and speak to their physicians to whether participating in one of these trials is right for them.

We’re planning more webinars in the new year to help you learn more about each of these opportunities. Meantime, watch your inbox for a special letter from me on December 15th that shares my personal reflections and vision for 2026!

Keith McArthur
Executive Director
CureGRIN Foundation