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

ConditionGRIN-related Neurodevelopmental Disorder
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age1-18
SponsorGRIN Therapeutics, Inc.

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • Radiprodil

    Drug

    Radiprodil oral suspension

  • Placebo

    Drug

    Placebo-to-match radiprodil oral suspension

Treatment groups

100 Participants
are divided into 2 treatment groups
Group A: RadiprodilExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Part A - Phase 3 Cohort 1 (Qualifying Seizures Cohort): Countable motor seizures (CMS)

Time frame
12 weeks
2

Part A - Phase 3 Cohort 2 (Without Qualifying Seizures Auxiliary Cohort): Adverse events (AEs), serious adverse events (SAEs), and adverse drug reactions (ADRs)

Time frame
24 weeks
3

Part B - Open-Label Extension: Adverse events (AEs), serious adverse events (SAEs), and adverse drug reactions (ADRs)

Time frame
Average of 2 years

Secondary outcomes

1

Part A - Phase 3 Cohort 1 (Qualifying Seizures Cohort): Proportion of participants with ≥50% reduction in CMS

Time frame
12 weeks
2

Part A - Phase 3 Cohort 1 (Qualifying Seizures Cohort): CMS-free days

Time frame
12 weeks
3

Part A - Phase 3 Cohort 1 (Qualifying Seizures Cohort): GRIN-NDD-specific Clinical Global Impression - Change [CGI-C] scale

Time frame
12 weeks
4

Part A - Phase 3 Cohort 1 (Qualifying Seizures Cohort): Aberrant Behavior Checklist-Community (ABC-2C) irritability subscale

Time frame
12 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

GRIN Therapeutics, Inc.

Lead sponsor

Aziende Chimiche Riunite Angelini Francesco S.p.A

Collaborator

Source ClinicalTrials.gov
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