Beeline Phase 3: radiprodil for GRIN-related neurodevelopmental disorder

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This randomized, double-blind, placebo-controlled Phase 3 trial enrolls participants with GRIN-related neurodevelopmental disorder caused by gain-of-function variants into two cohorts (with qualifying motor seizures or without qualifying seizures). Participants are randomized 1:1 to receive radiprodil or matching placebo, undergo a 4-week fixed titration, then enter a maintenance period of 12 weeks (cohort 1) or 24 weeks (cohort 2) followed by taper and follow-up. After the blinded Part A, eligible participants may enter an open-label extension to receive radiprodil long-term while safety and outcomes are monitored. Key eligibility includes confirmed GoF variants in GRIN1, GRIN2A, GRIN2B, or GRIN2D, stable background antiseizure/nonpharmacologic therapies, and cohort-specific seizure or symptom thresholds.

Why it matters

Who can join: the full rules

Inclusion Criteria:

Part A, Participant:

* Diagnosed with GRIN-NDD with GRIN1, GRIN2A, GRIN2B, or GRIN2D gene variants known to result in GoF of the NMDA receptor
* Phase 3 Cohort 1 (Qualifying Seizures Cohort) ONLY: Experiencing at least 1 CMS per week and ≥4 CMS (generalized or focal) during screening
* With history of inadequate response to at least 2 standard antiseizure medications (ASMs)
* Phase 3 Cohort 2 (Without Qualifying Seizures Auxiliary Cohort) ONLY: With significant neurodevelopmental symptoms and a GRIN-CGI-S score ≥4
* On a stable dose of standard ASMs for at least 4 weeks prior to screening and should remain on stable doses throughout study participation
* On stable nonpharmacological treatments such as ketogenic diet and should remain stable throughout study participation

Part B:

\- Participant has completed Part A and is eligible to continue study participation according to the judgement of the investigator and sponsor.

Exclusion Criteria:

PART A, Participant:

* Has clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder (including those related to GRIN-NDD) that would preclude or jeopardize participant's safe participation or study drug administration or the conduct of the study according to the judgement of the investigator or sponsor.
* Is receiving \>4 standard ASMs at screening
* Has a body weight of less than 5 kg at screening

Part B:

\- Participant has clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder (including those related to GRIN-NDD) that would preclude or jeopardize participant's safe participation of study drug administration or the conduct of the study according to the judgement of the investigator or sponsor.

Where it's running

• UCLA Health - Ronald Reagan UCLA Medical Center
  Los Angeles, California, United States Recruiting
• Lucile Packard Children's Hospital
  Palo Alto, California, United States Recruiting
• Children's Hospital Colorado - Anschutz Medical Campus
  Aurora, Colorado, United States Recruiting
• Children's National Hospital
  Washington D.C., District of Columbia, United States Recruiting
• Nicklaus Children's Hospital
  Miami, Florida, United States Recruiting
• Pediatric Neurology and Epilepsy
  Winter Park, Florida, United States Recruiting

Study contacts

• Study coordinator: Clinical Operations
• Email: ClinicalTrials@GrinTherapeutics.com
• Phone: +1-877-225-0014

Similar studies

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.