Beeline: A Phase 3 Study in GRIN-related Neurodevelopmental Disorder
The Phase 3 portion of Study RAD-GRIN-101 is a multinational, multicenter, randomized, double-blind, placebo-controlled trial followed by an open-label extension to evaluate the efficacy and safety of radiprodil in participants with GRIN-related neurodevelopmental disorder (GRIN-NDD) with a gain-of-function (GoF) genet
| Condition(s) | GRIN-related Neurodevelopmental Disorder |
|---|---|
| Status | Recruiting |
| Phase | Phase 3 |
| Study type | Interventional |
| Summary | The Phase 3 portion of Study RAD-GRIN-101 is a multinational, multicenter, randomized, double-blind, placebo-controlled trial followed by an open-label extension to evaluate the efficacy and safety of radiprodil in participants with GRIN-related neurodevelopmental disorder (GRIN-NDD) with a gain-of-function (GoF) genetic variant. This study will enroll two cohorts: one cohort of participants with a minimal number of countable motor seizures (with or without behavioral symptoms) (Phase 3 Cohort 1: Qualifying Seizures Cohort); and a second cohort with disease symptoms but no seizures or fewer seizures than required for the Qualifying Seizures Cohort (Phase 3 Cohort 2: Without Qualifying Seizures Auxiliary Cohort). Participants in each cohort will be randomized 1:1 to receive active drug (rad |
| Who can participate | 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 participatio |
| Ages | 1 Month to 18 Years |
| Sex | All |
| Lead sponsor | GRIN Therapeutics, Inc. |
| Locations | Los Angeles, California, United States; Palo Alto, California, United States; Aurora, Colorado, United States; Washington D.C., District of Columbia, United States; Miami, Florida, United States; Winter Park, Florida, United States (+22 more sites) |
| Start date | 2026-01-05 |
| NCT ID | NCT07224581 |
| Official listing | https://clinicaltrials.gov/study/NCT07224581 |