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

Conditions

• GRIN-related Neurodevelopmental Disorder

Eligibility Criteria

Sex

ALL

Age

1 Month - 18 Years

Healthy Volunteers

Not accepted

Interventions

• Radiprodil — DRUG
  Radiprodil oral suspension
• Placebo — DRUG
  Placebo-to-match radiprodil oral suspension

Study Details

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.

Key Dates

Start date

Jan 5, 2026

Status verified

Feb 2026

Primary completion

May 31, 2027

Completion

Jul 31, 2028

Study Design

Enrollment

100 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Radiprodil
  Liquid suspension of radiprodil, at varying concentrations depending on participant's weight. The following dose-escalation regimen (twice daily \[BID\]) will be used: Titration Period Visit 1 (Visit T1): Dose 1, Visit T2: Dose 2, Visit T3: Dose 3, Visit T4: Maintenance Dose.
• Placebo Comparator: Placebo
  Liquid suspension of placebo matching radiprodil oral suspension and dose-escalation regimen.

Primary Outcome Measure

Part A - Phase 3 Cohort 1 (Qualifying Seizures Cohort): Countable motor seizures (CMS) [ Time Frame: 12 weeks ]

Central Contacts

• Clinical Operations
  +1-877-225-0014
  [email protected]

Locations (17)

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