Clinical Trials in Spain / NCT05818943
Active, not recruiting
Phase 1
Honeycomb: Evaluation of Radiprodil in Children with GRIN-related Disorder
NCT05818943 · tracked via the Priya Life Science Spain tracker
Condition(s) studied
GRIN-related Disorders
Investigational drug(s) / intervention(s)
Radiprodil: Radiprodil is an orally active, negative allosteric modulator of the NR2B subunit of the NMDA receptor.
Study summary
Study RAD-GRIN-101 is a phase 1B trial to assess safety, tolerability, PK, and potential efficacy of radiprodil for the treatment of GRIN-related disorder in children with a Gain-of-Function (GoF) genetic variant. The study is open-label, so all participants will be treated with radiprodil.
Subjects' participation in the study is expected to last up to six months in Part A.
After the end of part A, all participants who are still eligible can choose to continue to receive radiprodil as part of an open-label long-term treatment period (Part B).
Eligibility
Inclusion Criteria:
* Age: ≥6 months to ≤12 years, with GRIN gene variants known to result in GoF of the NMDA receptor.
* Cohort 1 must have at least 1 observable motor seizure per week and ≥4 observable motor seizures (generalized or focal) during the prospective 4-week Observation Period and must have failed to obtain adequate seizure control with at least 2 antiseizure medications (ASMs) used at appropriate dose and duration.
* Cohort 2 must have significant behavioral and/or motor symptoms based on caregiver report with a CGI-S score ≥4.
* Stable antiseizure therapies and nonpharmacological treatments such as ketogenic diet throughout screening and study participation.
Exclusion Criteria:
* Any other clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder unrelated to GRIN-related disorder that would preclude or jeopardize participant's safe participation or the conduct of the study according to the judgement of the investigator.
* Clinically significant laboratory or ECG abnormalities.
* Severe hepatic dysfunction (Child-Pugh grade C).
* History of brain surgery for epilepsy or any other reason.
* Receiving treatment with contraindicated concomitant drugs such as agonists or antagonists of the glutamate receptor, including but not limited to felbamate, memantine, and perampanel.
* Receiving treatment with hormonal therapy such as adrenocorticotrophic hormone or prednisolone.
Primary outcome measure(s)
- Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), TEAEs Leading to Discontinuation and Severity of TEAEs — through study completion (average of 2 years).
Frequency, type, severity and duration of adverse events, serious adverse events and adverse drug reactions. - Maximum Plasma concentration of radiprodil (Cmax) — Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
- Pharmacokinetic plasma concentration of radiprodil: half-life (T1/2) — Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
- Plasma concentration of radiprodil versus time, area under the curve (AUCt) — Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
- Pharmacokinetic plasma concentration of radiprodil, clearance (Cl) — Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
- Pharmacokinetic plasma concentration of radiprodil, Time corresponding to occurrence of Cmax (Tmax) — Titration Visit 1 (week 5): 0,1,2,4,6,8,10,12 hours post-dose. Titration Visits 2,3,4 (week 6 to 11) and Maintenance Visit 4 (week 20): 0,1,2,5 hours post-dose.
Trial sites (15)
| Facility | City | Region | Status |
|---|---|---|---|
| Mid-Atlantic Epilepsy and Sleep Center | Bethesda | Maryland | |
| Columbia University Irving Medical Center, Dept of Neurology | New York | New York | |
| Queensland Children's Hospital | South Brisbane | Queensland | |
| The Hospital for Sick Children (Sick Kids) | Toronto | Ontario | |
| BC Children's Hospital | Vancouver | Canada | |
| Abteilung für Neuropädiatrie, Klinik und Poliklinik für Kinder - und Jugendmedizin, Universitätsklinikum Leipzig | Leipzig | Germany | |
| KBO-Kinderzentrum München gemeinnützige GmbH | München | Germany | |
| Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale Pediatrico Bambino Gesu | Rome | Lazio | |
| Azienda Ospedaliero Universitaria Careggi (AOUC) Firenze - Azienda Ospedaliera Universitaria Meyer | Florence | Tuscany | |
| ERASMUS Medisch Centrum, Developmental & Genetic pediatrics | Rotterdam | Netherlands | |
| UMC Utrecht - Wilhelmina Kinderziekenhuis, Polikliniek Kinderneurologie | Utrecht | Netherlands | |
| Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB) | Barcelona | Spain | |
| Hospital Ruber Internacional | Madrid | Spain | |
| Royal Hospital for Children Glasgow | Glasgow | United Kingdom | |
| Great Ormond Street Hospital For Children NHS Foundation Trust | London | United Kingdom |
More GRIN Therapeutics, Inc. trials in Spain
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05818943 on ClinicalTrials.gov ↗ ← All trials in Spain