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Beeline: A Phase 3 Study in GRIN-related Neurodevelopmental Disorder
Beeline: A Phase 3 Study in GRIN-related Neurodevelopmental Disorder

NCT07224581

RecruitingPhase 3

Sponsor: GRIN Therapeutics, Inc.

Conditions: GRIN-related Neurodevelopmental Disorder

Interventions: Radiprodil, Placebo

Countries: United States, Belgium, France, Germany, Italy, Netherlands, Poland, Slovenia

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 (radiprodil) or matching placebo (Part A). Following completion of Part A, all eligible participants (including those previously on placebo) may continue into the open-label extension period (Part B) to receive radiprodil. The placebo-controlled portion is expected to be approximately 16 weeks for participants in Phase 3 Cohort 1 and 28 weeks for participants in Phase 3 Cohort 2. The study will evaluate the effect of radiprodil on seizures and non-seizure symptoms and assess safety.

Eligibility overview

Sex: ALL

Age: 1 Month to 18 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
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.
Locations (34)
  • 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
  • Iowa City, Iowa, United States
  • Boston, Massachusetts, United States
  • Hackensack, New Jersey, United States
  • New York, New York, United States
  • Durham, North Carolina, United States
  • Cincinnati, Ohio, United States
  • Philadelphia, Pennsylvania, United States
  • Austin, Texas, United States
  • Dallas, Texas, United States
  • Houston, Texas, United States
  • Seattle, Washington, United States
  • Brussels, Belgium
  • Angers, France
  • Bron, France
  • Paris, France
  • Toulouse, France
  • Greifswald, Germany
  • Leipzig, Germany
  • Florence, Viale Pieraccini, Italy
  • Pavia, Italy
  • Roma, Italy
  • Rotterdam, South Holland, Netherlands
  • Lodz, Poland
  • Ljubljana, Slovenia
  • + 4 more on CT.gov