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Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or 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.
Study details:
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.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 6 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2024-07-10
Primary completion: 2025-07-01
Study completion finish: 2026-07-01
Study type
TREATMENT
Phase
PHASE1
PHASE2
Trial ID
NCT06392009
Intervention or treatment
DRUG: Radiprodil
Conditions
- • Tuberous Sclerosis Complex
- • Focal Cortical Dysplasia
Find a site
Closest Location:
Queensland Children Hospital
Research sites nearby
Select from list below to view details:
Queensland Children Hospital
South Brisbane, Not Specified, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
| Participant Group/Arm | Intervention/Treatment |
|---|---|
EXPERIMENTAL: TSC
| DRUG: Radiprodil
|
EXPERIMENTAL: FCD Type II
| DRUG: Radiprodil
|
What is the study measuring?
Primary outcome
| Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
|---|---|---|
| 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. | from Baseline to End-of-study: 1 year 6 months |
| Plasma concentration of radiprodil and maximum plasma concentration (Cmax) | Not Specified | 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 |
| Plasma concentration of radiprodil versus time, area under the curve (AUCt) | Not Specified | 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 |
| Pharmacokinetic plasma concentration of radiprodil: half-life (T1/2) | Not Specified | 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 |
| Pharmacokinetic plasma concentration of radiprodil: time to Cmax (Tmax) | Not Specified | 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 |
| Pharmacokinetic plasma concentration of radiprodil, clearance (Cl) | Not Specified | 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 |
| Number of participants with abnormal laboratory tests results | The clinical laboratory tests include Hematology, Serum Chemistry and Coagulation | from Baseline to End-of-study: 1 year 6 months |
| 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 | Baseline, MV7, and in Part B: Month 3, 6, 9, 12: week 6, week 28, week 40, week 52, week 64, week 76 |
| Clinically relevant changes in safety parameters: systolic blood pressure | changes from Baseline to End of study for systolic blood pressure | from Baseline to End-of-study: 1 year 6 months |
| Clinically relevant changes in safety parameters: diastolic blood pressure | changes from Baseline to End of study for diastolic blood pressure | from Baseline to End-of-study: 1 year 6 months |
| Clinically relevant changes in safety parameters: pulse rate | changes from Baseline to End of Treatment for pulse rate | from Baseline to End-of-study: 1 year 6 months |
| 12-Lead ECG: Mean change from Baseline to End-of-Treatment in RR interval | Not Specified | from Baseline to End-of-study: 1 year 6 months |
| 12-Lead ECG: Mean change from Baseline to End-of-Treatment in PR interval | Not Specified | from Baseline to End-of-study: 1 year 6 months |
| 12-Lead ECG: Mean change from Baseline to End-of-Treatment in QRS interval | Not Specified | from Baseline to End-of-study: 1 year 6 months |
| 12-Lead ECG: Mean change from Baseline to End-of-Treatment in QT interval | Not Specified | from Baseline to End-of-study: 1 year 6 months |
| 12-Lead ECG: Mean change from Baseline to End-of-Treatment in QTcF interval | Not Specified | from Baseline to End-of-study: 1 year 6 months |
Secondary outcome
| Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
|---|---|---|
| Percent change from baseline in Video-EEG seizure burden | Assessed by 8- to 24- hour video electroencephalogram | Baseline to end-of-treatment: week 6 to week 76 |
| Change from baseline in seizure frequency | assessed by seizure diaries | Baseline to Maintenance Visit 7: week 6 to week 25 and Baseline to end-of-treatment: week 6 to week 76 |
| Change from baseline in number of seizure-free days and longest period with no seizures | assessed by seizure diaries | Baseline to end-of-treatment: week 6 to week 76 |
| 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. | Baseline to end-of-treatment: week 6 to week 76 |
| 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). | Baseline to end-of-treatment: week 6 to week 76 |
| 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"). | Baseline to end-of-treatment: week 6 to week 76 |
| 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. | Baseline to end-of-treatment: week 6 to week 76 |
| 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. | Baseline to end-of-treatment: week 6 to week 76 |
| 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. | Baseline to end-of-treatment: week 6 to week 76 |
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