Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

NCT ID: NCT06392009

Last Updated: 2025-07-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-07-10

Study Completion Date

2026-07-31

Brief Summary

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Study RAD-GRIN-201 is a phase 1B/2A trial to assess safety, tolerability, pharmacokinetics (PK), and potential efficacy of radiprodil in participants with Tuberous Sclerosis Complex (TSC) or Focal Cortical Dysplasia (FCD) type II. 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 and one year in Part B/long-term treatment period. The treatment period in Part B may be extended based on a favorable benefit/risk profile.

Detailed Description

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Approximately 20 participants with TSC and 10 participants with FCD type II will be enrolled.

The effects of radiprodil are assessed in participants with treatment-resistant seizures (with or without behavioral symptoms). The daily doses of radiprodil will be individually titrated for every participant and all the participants will receive study drug.

This study is divided into the following periods:

PART A:

* Screening/Observation Period (up to six(6) weeks): Investigators assess eligibility followed by an Observation Period (at least four(4) weeks) to evaluate seizure frequency.
* Titration Period (approx. four(4) weeks): Radiprodil twice daily will be administered in escalating doses and plasma concentrations, safety, and tolerability assessed. Once a safe and potentially effective dose has been established, the participant will immediately enter the Maintenance Period.
* Maintenance Period (approx. twelve(12) weeks): The participant will continue to take the safe and potentially effective dose identified during the Titration Period. At the end of the Maintenance Period the participant will either be invited to enter Part B or the Tapering and Safety Follow-up Period.
* Tapering (15 days) and Safety Follow-up Period (14 days): a participant who doesn't take part in the long-term treatment period (Part B) will taper (ie gradually decrease) the study medicine for 15 days and enter a safety Follow-up Period (14 days). In this case, the participant will have one (1) last visit at the end of the safety Follow-up Period.

PART B:

* Long-Term Treatment Period (one(1) year): During the Long-Term Treatment Period (Part B), participants will continue taking radiprodil at the usual dose level and making regular visits to the study site.
* Tapering (15 days) and Safety Follow-up Period (14 days): at the end of the long-term treatment period (Part B), the participant will taper (ie gradually decrease) the study medicine for 15 days and enter a safety Follow-up Period (14 days) after his/her last dose of radiprodil. The participant will have one (1) last visit at the end of the safety Follow-up Period.

Conditions

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Tuberous Sclerosis Complex Focal Cortical Dysplasia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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TSC

Liquid suspension of radiprodil, at concentrations 0.25 mg/mL or 2.50 mg/mL for 1% and 10% formulation respectively. It will be administered twice a day (bid) either orally or via gastric or nasogastric tube.

Group Type EXPERIMENTAL

Radiprodil

Intervention Type DRUG

Radiprodil is an orally active, negative allosteric modulator of the NR2B subunit of the NMDA receptor.

FCD Type II

Liquid suspension of radiprodil, at concentrations 0.25 mg/mL or 2.50 mg/mL for 1% and 10% formulation respectively. It will be administered twice a day (bid) either orally or via gastric or nasogastric tube.

Group Type EXPERIMENTAL

Radiprodil

Intervention Type DRUG

Radiprodil is an orally active, negative allosteric modulator of the NR2B subunit of the NMDA receptor.

Interventions

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Radiprodil

Radiprodil is an orally active, negative allosteric modulator of the NR2B subunit of the NMDA receptor.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Failed to respond to at least 2 anti-seizure medications (ASMs) at appropriate dosages and duration.
* Disease specific criteria:

1. diagnosis of FCD Type II based on clinical symptoms and confirmed by a positive magnetic resonance imaging (MRI)
2. diagnosis of TSC by either clinical or genetic diagnostic criteria (Northrup, 2021) as documented in the participant's medical record.
* Participant on average has had at least 8 countable/witnessed primary seizures during a 4-week baseline period with at least 1 seizure occurring in at least 3 of the 4 weeks of baseline
* All medical interventions for epilepsy / behavior (including ketogenic diet and any neurostimulation devices) should be stable for 28 days prior to screening with no more than 6 days per month use of rescue medication. Participants must remain on a stable regimen throughout the treatment period.
* Participant has had an MRI scan within 12 months of the planned date of first dose of study drug.

Exclusion Criteria

* Any other clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder unrelated to TSC or FCD Type II that would preclude or jeopardize participant's safe participation or administration of study drug 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 within 6 months of screening for epilepsy or any other reason.
* Contraindications to radiprodil or with known hypersensitivity to the active substance or the excipients or other chemically closely related substances.
* Receiving treatment with contraindicated concomitant drugs such as agonists or antagonists of the glutamate receptor, including but not limited to felbamate, memantine, and perampanel.
* body weight \<10kg for whom a gastric tube is the only possibility for radiprodil dosing.
Minimum Eligible Age

6 Months

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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GRIN Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Queensland Children Hospital

South Brisbane, , Australia

Site Status RECRUITING

Universitair Ziekenhuis Antwerpen (UZA)

Antwerp, , Belgium

Site Status RECRUITING

University Hospitals Leuven, Pediatric Neurology

Leuven, , Belgium

Site Status RECRUITING

Alberta Children's Hospital

Calgary, , Canada

Site Status RECRUITING

The Hospital for Sick Children (Sick Kids)

Toronto, , Canada

Site Status RECRUITING

BC Children's Hospital

Vancouver, , Canada

Site Status RECRUITING

IRCCS Istituto Giannina Gaslini

Genoa, Liguria, Italy

Site Status RECRUITING

AOU Meyer

Florence, Tuscany, Italy

Site Status RECRUITING

Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS)

Roma, , Italy

Site Status RECRUITING

Universita Cattolica del Sacro Cuore - Policlinico Universitario "Agostino Gemelli"

Roma, , Italy

Site Status RECRUITING

UMC Uthrecht - Wilhelmina Kinderziekenhuis

Utrecht, , Netherlands

Site Status RECRUITING

Uniwersyteckie Centrum Kliniczne

Gdansk, , Poland

Site Status RECRUITING

Centrum Medyczne Plejady

Krakow, , Poland

Site Status RECRUITING

Uniwersytecki Szpital Kliniczny w Poznaniu

Poznan, , Poland

Site Status RECRUITING

Instytut Pomnik - Centrum Zdrowia Dziecka

Warsaw, , Poland

Site Status RECRUITING

Hospital Universitario Vall D´Hebrón

Barcelona, , Spain

Site Status RECRUITING

Hospital Materno Infantil Sant Joan de Deu de Barcelona

Barcelona, , Spain

Site Status RECRUITING

Hospital Universitario Vithas La Milagrosa

Madrid, , Spain

Site Status RECRUITING

Hospital Ruber Internacional

Madrid, , Spain

Site Status RECRUITING

University Hospitals Bristol and Weston NHS Foundation Trust Bristol Royal Hospital for Children

Bristol, , United Kingdom

Site Status RECRUITING

Countries

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Australia Belgium Canada Italy Netherlands Poland Spain United Kingdom

Central Contacts

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Clinical Operations

Role: CONTACT

+1-877-225-0014

Other Identifiers

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2023-506301-20-00

Identifier Type: OTHER

Identifier Source: secondary_id

RAD-GRIN-201

Identifier Type: -

Identifier Source: org_study_id

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