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Tuberous Sclerosis ComplexPhase 1/2NCT06392009

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

A Phase 1/2 study testing Radiprodil in people with Tuberous Sclerosis Complex and Focal Cortical Dysplasia, run by GRIN Therapeutics, Inc. at 18 sites in 8 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 6 months to 18 years
What is being tested?
Radiprodil
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
18 sitesin 8 countries
Study dates
10 Jul 2024to Jun 2028
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 months to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Failed to respond to at least 2 anti-seizure medications (ASMs) at appropriate dosages and duration
  • Disease specific criteria:
  • diagnosis of FCD Type II based on clinical symptoms and confirmed by a positive magnetic resonance imaging (MRI)
  • 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

Who cannot join

  • 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.

Trial locations and contacts

18 sites in 8 countries.

18 sites in 8 countries

Italy4 sites

  • AOU Meyer

    Florence, Tuscany, 50139

  • IRCCS Istituto Giannina Gaslini

    Genoa, Liguria, 16146

  • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS)

    Roma, 00165

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

    Roma, 00168

Other trials in Italy

Poland4 sites

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80952

  • Centrum Medyczne Plejady

    Krakow, 30363

  • Uniwersytecki Szpital Kliniczny w Poznaniu

    Poznan, 60356

  • Instytut Pomnik - Centrum Zdrowia Dziecka

    Warsaw, 04730

Other trials in Poland

Spain4 sites

  • Hospital Universitario Vall D´Hebrón

    Barcelona, 08035

  • Hospital Materno Infantil Sant Joan de Deu de Barcelona

    Barcelona, 08950

  • Hospital Universitario Vithas La Milagrosa

    Madrid, 28010

  • Hospital Ruber Internacional

    Madrid, 28034

Other trials in Spain

Canada2 sites

  • Alberta Children's Hospital

    Calgary, T3A 2X6

  • The Hospital for Sick Children (Sick Kids)

    Toronto, M5G 1X8

Other trials in Canada

Australia1 site

  • Queensland Children Hospital

    South Brisbane, 4101

Other trials in Australia

Belgium1 site

  • University Hospitals Leuven, Pediatric Neurology

    Leuven, 3000

Other trials in Belgium

Netherlands1 site

  • UMC Uthrecht - Wilhelmina Kinderziekenhuis

    Utrecht, 3508

Other trials in Netherlands

United Kingdom1 site

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

    Bristol, BS2 8BJ

Other trials in United Kingdom

About this study

Official title: A Multicenter, Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms of Radiprodil in Patients With Tuberous Sclerosis Complex (TSC) or Focal Cortical Dysplasia (FCD) Type II

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.

Read the detailed description

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 (not specified): 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.
Being tested
Radiprodil
Keywords
TSC, FCD, Tuberous Sclerosis Complex, Focal Cortical Dysplasia, Astroscape
Medical subject headings
Tuberous Sclerosis, Focal Cortical Dysplasia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RadiprodilDrug

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

Study groups in NCT06392009
Study groupRoleWhat they receive
TSCExperimentalLiquid suspension of radiprodil, at concentrations 0.25 mg/mL, 2.50 mg/mL or 7.5 mg/mL for 1%, 10% and 30% formulation respectively. It will be administered twice a day (bid) either orally or via gastric or nasogastric tube.
Receives: Drug: Radiprodil
FCD Type IIExperimentalLiquid suspension of radiprodil, at concentrations 0.25 mg/mL, 2.50 mg/mL or 7.5 mg/mL for 1%, 10% and 30% formulation respectively. It will be administered twice a day (bid) either orally or via gastric or nasogastric tube.
Receives: Drug: Radiprodil

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), Adverse Drug Reactions (ADRs), TEAEs Leading to Discontinuation and Severity of TEAEs

    Frequency, type, severity and duration of adverse events, serious adverse events and adverse drug reactions.

    Measured: from Baseline to End-of-study: 1 year 6 months

  2. Plasma concentration of radiprodil and maximum plasma concentration (Cmax)

    Measured: Titration Visit 1 (week 7): Pre-dose to 12 hours post-dose. Titration Visits 2,3,4 (week 8 to 13) and Maintenance Visit 7 (week 25): pre-dose to 5 hours post-dose

  3. Plasma concentration of radiprodil versus time, area under the curve (AUCt)

    Measured: Titration Visit 1 (week 7): Pre-dose to 12 hours post-dose. Titration Visits 2,3,4 (week 8 to 13) and Maintenance Visit 7 (week 25): pre-dose to 5 hours post-dose

  4. Pharmacokinetic plasma concentration of radiprodil: half-life (T1/2)

    Measured: Titration Visit 1 (week 7): Pre-dose to 12 hours post-dose. Titration Visits 2,3,4 (week 8 to 13) and Maintenance Visit 7 (week 25): pre-dose to 5 hours post-dose

  5. Pharmacokinetic plasma concentration of radiprodil: time to Cmax (Tmax)

    Measured: Titration Visit 1 (week 7): Pre-dose to 12 hours post-dose. Titration Visits 2,3,4 (week 8 to 13) and Maintenance Visit 7 (week 25): pre-dose to 5 hours post-dose

  6. Pharmacokinetic plasma concentration of radiprodil, clearance (Cl)

    Measured: Titration Visit 1 (week 7): Pre-dose to 12 hours post-dose. Titration Visits 2,3,4 (week 8 to 13) and Maintenance Visit 7 (week 25): pre-dose to 5 hours post-dose

  7. Number of participants with abnormal laboratory tests results

    The clinical laboratory tests include Hematology, Serum Chemistry and Coagulation

    Measured: from Baseline to End-of-study: 1 year 6 months

  8. Number of participants with abnormal physical and neurological examination findings

    A complete physical and neurological examination according to standard of care excluding the genitourinary examination will be performed

    Measured: Baseline, MV7, and in Part B: Month 3, 6, 9, 12: week 6, week 28, week 40, week 52, week 64, week 76

  9. Clinically relevant changes in safety parameters: systolic blood pressure

    changes from Baseline to End of study for systolic blood pressure

    Measured: from Baseline to End-of-study: 1 year 6 months

  10. Clinically relevant changes in safety parameters: diastolic blood pressure

    changes from Baseline to End of study for diastolic blood pressure

    Measured: from Baseline to End-of-study: 1 year 6 months

  11. Clinically relevant changes in safety parameters: pulse rate

    changes from Baseline to End of Treatment for pulse rate

    Measured: from Baseline to End-of-study: 1 year 6 months

  12. 12-Lead ECG: Mean change from Baseline to End-of-Treatment in RR interval

    Measured: from Baseline to End-of-study: 1 year 6 months

  13. 12-Lead ECG: Mean change from Baseline to End-of-Treatment in PR interval

    Measured: from Baseline to End-of-study: 1 year 6 months

  14. 12-Lead ECG: Mean change from Baseline to End-of-Treatment in QRS interval

    Measured: from Baseline to End-of-study: 1 year 6 months

  15. 12-Lead ECG: Mean change from Baseline to End-of-Treatment in QT interval

    Measured: from Baseline to End-of-study: 1 year 6 months

  16. 12-Lead ECG: Mean change from Baseline to End-of-Treatment in QTcF interval

    Measured: from Baseline to End-of-study: 1 year 6 months

Secondary outcomes (9)

  1. Percent change from baseline in Video-EEG seizure burden

    Assessed by 8- to 24- hour video electroencephalogram

    Measured: Baseline to end-of-treatment: week 6 to week 76

  2. Change from baseline in seizure frequency

    assessed by seizure diaries

    Measured: Baseline to Maintenance Visit 7: week 6 to week 25 and Baseline to end-of-treatment: week 6 to week 76

  3. Change from baseline in number of seizure-free days and longest period with no seizures

    assessed by seizure diaries

    Measured: Baseline to end-of-treatment: week 6 to week 76

  4. Aberrant Behavior Checklist-Community (ABC-2C)

    The ABC-2C is a standardized 58-item caregiver-reported problem-behavior rating scale, originally designed to assess treatment effects in people with intellectual disabilities. Each item is scored from 0 (never a problem) to 3 (severe problem). Items load onto one of five empirically derived subscales: Irritability, Agitation, \& Crying (15 items); Lethargy/Social Withdrawal (16 items); Stereotypic Behavior (7 items); Hyperactivity/Noncompliance (16 items); and Inappropriate Speech (4 items). A total score would range from 0 to 174.

    Measured: Baseline to end-of-treatment: week 6 to week 76

  5. Caregiver Global Impression of Change (CaGI-C)

    The CaGI-C is a 7-point caregiver-rated scale ranging from 1 (very much improved) to 7 (very much worse).

    Measured: Baseline to end-of-treatment: week 6 to week 76

  6. Clinical Global Impression of Change [CGI-C]

    The CGI scale is a clinician-rated measures of change of a symptom or condition, using a single item, 6- or 7-point scale. The CGI-C scale ranges from 1 ("Very much worse") to 7 ("Very much improved").

    Measured: Baseline to end-of-treatment: week 6 to week 76

  7. Pediatric Quality of Life Inventory [PedsQL]

    The PedsQL is a 23-item generic health status instrument assessing 5 domains of health in children. It's a 0-100 scale, and higher scores are indicative of better health-related quality of life.

    Measured: Baseline to end-of-treatment: week 6 to week 76

  8. Caregiver Burden Inventory (CBI)

    The CBI is a validated scale providing information regarding the impact of caregiving on the lives of caregivers. It comprises 24 closed questions divided into 5 dimensions. Each dimension includes 4 or 5 items. Each item is given a score between 0 and 4, where higher scores indicate greater caregiver burden.

    Measured: Baseline to end-of-treatment: week 6 to week 76

  9. Columbia-Suicide Severity Rating Scale (C-SSRS)

    The C-SSRS is a validated tool designed to systematically evaluate the severity and intensity of suicidal ideation and behavior. The scoring system ranges from 0 to 5 for suicidal ideation and from 0 to 25 for suicidal behavior, with higher scores indicating greater severity or greater frequency of suicidal thoughts or actions.

    Measured: Baseline to end-of-treatment: week 6 to week 76

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Apr 2024
  2. Study start10 Jul 2024
  3. Primary completionJun 2026 (estimated)
  4. Study completionJun 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06392009
Sponsor's protocol ID
RAD-GRIN-201
Other IDs
2023-506301-20-00, 2023
Status last verified
Mar 2026
First submitted
23 Apr 2024
Last updated
1 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Radiprodil in people with Tuberous Sclerosis Complex and Focal Cortical Dysplasia, run by GRIN Therapeutics, Inc. at 18 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 1 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites in Italy, Poland, Spain, Canada, Australia and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), Adverse Drug Reactions (ADRs), TEAEs Leading to Discontinuation and Severity of TEAEs (time frame: from Baseline to End-of-study: 1 year 6 months). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06392009.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Apr 2026.

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