RADIPRODIL for GRIN-related neurodevelopmental disorder

Inclusion criteria

• {"criterion_text":"- 1. PART A AND PART B - PHASE 1b : 1. For Part A, pediatric participants aged ≥6 months to ≤12 years with GRIN 1, 2A, 2B, or 2D gene variants known to result in GoF of the NMDA receptor. For Part B, pediatric participants aged ≥6 months with GRIN 1, 2A, 2B, or 2D gene variants known to result in GoF of the NMDA receptor.\n- 10. PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: 2. Participant experiences the following: a. At least 1 CMS (defined here) per week and ≥4 CMS (generalized or focal) during the prospective 4-week Observation Period immediately preceding randomization. b.\tHas not obtained adequate response to at least 2 standard ASMs used at appropriate dose and duration with assured medication adherence (if applicable). Participant will continue to receive SOC ASMs while receiving study drug.\n- 11. PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: 3. Participants must be on a stable dose of standard ASMs regardless of indication for at least 4 weeks prior to and during the Screening Period and should remain on stable doses throughout the study. Nonpharmacological treatments such as ketogenic diet should also be kept stable during screening and participation in the study.\n- 12. PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: 4. Participant has signed informed consent or participant’s caregivers have signed informed consent and participant has signed assent (if applicable).\n- 13. PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: 5. Participant’s caregivers are willing and able to complete entries in the eDiary on a daily basis and have demonstrated compliance (based on investigator and sponsor review) with eDiary entries during the Screening Period.\n- 14. PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: 6. Participant is one of the following: a. Not of childbearing potential (premenarchal or male/not in possession of a uterus). b. If of childbearing potential, is nonpregnant (negative serum pregnancy test results at Screening), nonlactating, and practicing one of the following medically acceptable methods of birth control from Screening through 90 days after the last dose of study drug: • Abstinence from heterosexual intercourse as a lifestyle choice. • Hormonal methods such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the participant’s usual menstrual cycle period) before study drug administration. • Intrauterine device. c. If male, is willing to use a condom from Screening through 90 days after the last dose of study drug.\n- 15. PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: 7. Participant is willing to abstain from sperm or egg donation from Screening through 90 days after the last dose of study drug.\n- 16. PART A – PHASE 3 RANDOMIZED WITHOUT QUALIFYING SEIZURES AUXILIARY COHORT: For all other inclusion criteria, see Inclusion Criteria for Part A – Phase 3 Randomized Qualifying Seizures Cohort with the exception of inclusion criterion 2 (eg, participants with seizures that are not CMS or those with <1 CMS per week would be eligible for the Randomized Without Qualifying Seizures Auxiliary Cohort).\n- 17. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 1. Participant’s caregivers are willing and able to complete entries in the eDiary on a daily basis and have demonstrated compliance (based on investigator and sponsor review) with eDiary entries during Part A.\n- 18. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 2. Participant is one of the following: a. Not of childbearing potential (premenarchal or male/not in possession of a uterus). b. If of childbearing potential, is nonpregnant (negative pregnancy test results at the TT1 Visit), nonlactating, and practicing one of the following medically acceptable methods of birth control throughout Part B through 90 days after the last dose of study drug: • Abstinence from heterosexual intercourse as a lifestyle choice. • Hormonal methods such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the participant’s usual menstrual cycle period) before study drug administration. • Intrauterine device. c. If male, is willing to use a condom throughout Part B and for 90 days after the last dose of study drug.\n- 19. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 3. Participant is willing to abstain from sperm or egg donation throughout Part B and for 90 days after the last dose of study drug.\n- 2. PART A AND PART B - PHASE 1b: 2. Participant to be enrolled in the first cohort experiences the following (Part A only): a) At least 1 observable motor seizure per week and ≥4 observable motor seizures (generalized or focal) during the prospective 4 week Observation Period. b) Has failed to obtain adequate seizure control with at least 2 ASMs used at appropriate dose and duration with assured medication adherence (if applicable).\n- 20. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 4. Participant has completed Part A (remained on study and were compliant with dosing and study procedures [based on investigator and sponsor assessment] through the last visit of Part A) of the Phase 3 portion of the study. The participant is eligible to continue study participation according to the judgment of the investigator.\n- 3. PART A AND PART B - PHASE 1b: 3. Participant to be enrolled in the second cohort experiences the following (Part A only): a) Significant behavioral and/or motor symptoms based on caregiver report with a CGI-S score ≥4 at the Screening Visit and Day -1 of Visit T1.\n- 4. PART A AND PART B - PHASE 1b: 4. For Part A, current therapies need to be on a stable dose for at least 4 weeks prior to Screening and should be maintained stable throughout the whole study duration. Nonpharmacological treatments such as ketogenic diet should be kept as stable as possible during screening and participation in the study. Changes in antiseizure medication should be discussed with the sponsor in consultation with the investigator.\n- 5. PART A AND PART B - PHASE 1b: 5. Participant’s caregivers have signed informed consent and participant has signed assent (if applicable).\n- 6. PART A AND PART B - PHASE 1b: 6. Participant’s caregivers are willing and able to complete entries in the eDiary on a daily basis.\n- 7. PART A AND PART B - PHASE 1b: 7. Participant is 1 of the following: a. Not of childbearing potential (premenarchal or male/not in possession of a uterus). b. If of childbearing potential, is nonpregnant (negative serum pregnancy test results at Screening), nonlactating, and practicing 1 of the following medically acceptable methods of birth control: • Abstinence from heterosexual intercourse as a lifestyle choice. • Hormonal methods such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the participant’s usual menstrual cycle period) before IP administration. • Intrauterine device. c. If male, is willing to use a highly effective method of contraception (ie, condom) throughout the study period.\n- 8. PART A AND PART B - PHASE 1b: Rescreening criteria for Part B only: 1. Participant has received at least 8 weeks of treatment (combined Titration and Maintenance Period) with radiprodil during Part A. 2. The benefit-risk of continuing radiprodil treatment remains favorable as determined by the investigator’s clinical assessment and is eligible to continue treatment according to the judgement of the investigator.\n- 9. PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: 1. Participants aged ≥1 month to ≤18 years with GRIN-NDD with GRIN1, GRIN2A, GRIN2B, or GRIN2D gene variants known to result in GoF of the NMDA receptor as determined using a functional characterization method consistent with Myers et al., 2023."}

Exclusion criteria

• {"criterion_text":"- 1. PART A AND B – PHASE 1b: 1.\tParticipant with any other clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder unrelated to GRIN-related disorders 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.\n- 10. PART A – PHASE 3 RANDOMIZED COHORTS: 1. Participant with any other clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder (including those related to GRIN NDD) that would preclude or jeopardize the participant’s safe participation or administration of study drug or the conduct of the study according to the judgment of the investigator or the sponsor.\n- 11. PART A – PHASE 3 RANDOMIZED COHORTS: 2. Participant or caregiver is unwilling or unable to comply with all procedures for the duration of study.\n- 12. PART A – PHASE 3 RANDOMIZED COHORTS: 3. Participant receiving >4 standard ASMs at the time of Screening.\n- 13. PART A – PHASE 3 RANDOMIZED COHORTS: 4. Participant with a body weight <5 kg at Screening.\n- 14. PART A – PHASE 3 RANDOMIZED COHORTS: 5. Participant with any clinically significant laboratory or ECG abnormalities according to the judgment of the investigator or the sponsor.\n- 15. PART A – PHASE 3 RANDOMIZED COHORTS: 6. Participant has severe hepatic dysfunction (Child-Pugh grade C).\n- 16. PART A – PHASE 3 RANDOMIZED COHORTS: 7. Participant has a history of brain surgery within 6 months of randomization for epilepsy or any other reason.\n- 17. PART A – PHASE 3 RANDOMIZED COHORTS: 8. Participant with any known hypersensitivity to radiprodil drug product (DP) active substance or the excipients or other chemically closely related substances.\n- 18. PART A – PHASE 3 RANDOMIZED COHORTS: 9. Participant receiving treatment with prohibited concomitant drugs such as agonists or antagonists of the glutamate receptor, including but not limited to felbamate, memantine, and perampanel. If the participant has been on any of these listed drugs, they must have discontinued the drug at least 5 half-lives or 28 days prior to the planned first dose of study drug, whichever is longer.\n- 19. PART A – PHASE 3 RANDOMIZED COHORTS: 11. Participant has received any prior gene therapy.\n- 2. PART A AND B – PHASE 1b: 2.\tParticipant with a body weight <10 kg on Day -1 of Visit T1 for whom a gastric tube is the only possibility for radiprodil dosing (during treatment with the first dose in Part A only).\n- 20. PART A – PHASE 3 RANDOMIZED COHORTS: 12. Participant is on treatment with hormonal therapy such as adrenocorticotrophic hormone or prednisolone.\n- 21. PART A – PHASE 3 RANDOMIZED COHORTS: 13. Participant has participated in any other investigational clinical study using an IP or device within 3 months or 5 half-lives of the IP, whichever is longer, of Screening.\n- 22. PART A – PHASE 3 RANDOMIZED COHORTS: 14. Participant has previously received radiprodil.\n- 23. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 1.\tParticipant with any other clinically relevant medical, neurologic, or psychiatric condition and/or behavioral disorder (including those related to GRIN-NDD) that would preclude or jeopardize the participant’s safe participation or administration of study drug or the conduct of the study according to the judgment of the investigator or the sponsor.\n- 24. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 2. Participant or caregiver is unwilling or unable to comply with all study procedures for the duration of the study.\n- 25. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 3. Participant receiving >4 standard ASMs.\n- 26. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 4. Participant with a body weight <5 kg.\n- 27. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 5. Participant with any clinically significant laboratory or ECG abnormalities according to the judgment of the investigator or the sponsor.\n- 28. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 6. Participant has severe hepatic dysfunction (Child-Pugh grade C).\n- 29. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 7. Participant with any known hypersensitivity to radiprodil DP active substance or the excipients or other chemically closely related substances.\n- 30. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 8. Participant receiving treatment with prohibited concomitant drugs such as agonists or antagonists of the glutamate receptor, including but not limited to felbamate, memantine, and perampanel.\n- 31. PART B OLE – PHASE 3 RANDOMIZED COHORTS: 9. Participant is on treatment with hormonal therapy such as adrenocorticotrophic hormone or prednisolone.\n- 3. PART A AND B – PHASE 1b: 3. Participant with any clinically significant laboratory or ECG abnormalities.\n- 4. PART A AND B – PHASE 1b: 4. Participant has severe hepatic dysfunction (Child-Pugh grade C).\n- 5. PART A AND B – PHASE 1b: 5.\tParticipant has a history of brain surgery for epilepsy or any other reason.\n- 6. PART A AND B – PHASE 1b: 6.\tParticipant with any contraindications to radiprodil or with known hypersensitivity to the active substance or the excipients or other chemically closely related substances..\n- 7. PART A AND B – PHASE 1b: 7. Participant receiving treatment with contraindicated concomitant drugs such as agonists or antagonists of the glutamate receptor, including but not limited to lamotrigine, felbamate, memantine, and perampanel.\n- 8. PART A AND B – PHASE 1b: 8.\tParticipant is on treatment with hormonal therapy such as adrenocorticotrophic hormone or prednisolone (Part A only).\n- 9. PART A AND B – PHASE 1b: 9.\tParticipant has participated in any other investigational clinical study within 3 months of Screening (Part A only)."}

Primary endpoints

• {"endpoint_text":"- PART A – PHASE 1b: • Adverse events (AEs), serious adverse events (SAEs), and adverse drug reactions (ADRs) (frequency, type, severity, and duration); • Changes in vital signs; • Physical examination findings; • 12-lead electrocardiogram (ECG) findings: •\tClinically significant changes in laboratory parameters; • Emergence of new seizure types; • Occurrence of suicidal ideation or behavior","definition_or_measurement_approach":"Safety outcomes assessed by standard AE/SAE/ADR reporting (frequency, type, severity, duration); vital signs, physical exam, 12-lead ECG, laboratory parameters, new seizure types, and suicidal ideation/behavior assessed during study visits and reported per protocol."}
• {"endpoint_text":"- PART A – PHASE 1b: Plasma concentrations of radiprodil at predefined timepoints","definition_or_measurement_approach":"Pharmacokinetic sampling: plasma concentrations measured at predefined timepoints (PK assessments) per protocol."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: Countable motor seizure frequency (per 28 days) during the Maintenance Period (Weeks 1 through 12), as captured in the daily seizure eDiary","definition_or_measurement_approach":"Seizure frequency measured as countable motor seizure (CMS) frequency per 28 days, recorded by caregivers in a daily electronic seizure eDiary during Maintenance Period (Weeks 1–12)."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED WITHOUT QUALIFYING SEIZURES AUXILIARY COHORT: Change from baseline to end of Maintenance Period (Week 24) in the ABC-2C irritability subscale score","definition_or_measurement_approach":"Behavioral endpoint measured by change from baseline to Week 24 in the Aberrant Behavior Checklist-Community (ABC-2C) irritability subscale score."}
• {"endpoint_text":"- PART B – OLE STUDY: AEs, SAEs, and ADRs (frequency, type, severity, and duration)","definition_or_measurement_approach":"Safety monitoring during the open-label extension using standard AE/SAE/ADR reporting (frequency, type, severity, duration) collected throughout OLE visits."}

Secondary endpoints

• {"endpoint_text":"- PART A – PHASE 1b: • Change from baseline to End of Treatment (EOT) in seizure frequency from daily seizure electronic diary (eDiary); • Percent change from baseline to EOT in video electroencephalogram (V EEG) seizure burden (eg, seizure type, severity, and frequency recorded during V EEGs); • Seizure-free days and longest period with no seizures","definition_or_measurement_approach":"Seizure frequency and burden measured via daily eDiary and video EEG (V-EEG); seizure-free days and longest seizure-free interval assessed from diary/V-EEG records."}
• {"endpoint_text":"- PART A – PHASE 1b: Change from baseline to EOT in behavioral features as measured by the aberrant behavior checklist-community (ABC 2C), as well as other disorder features as measured by gross motor function measure (GMFM), sleep disturbance scale for children (SDSC), quality of life (Pediatric Quality of Life Inventory [PedsQL]), Caregiver Burden Inventory (CBI), and global impression (Caregiver Global Impression of Change [CaGI C]), and Clinical Global Impression of Change [CGI-C] scales)","definition_or_measurement_approach":"Behavioral and other disorder features assessed using ABC-2C, GMFM, SDSC, PedsQL, CBI, CaGI-C and CGI-C instruments at protocol-specified visits; change from baseline to EOT reported."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: Proportion of participants with ≥50% reduction in CMS frequency (per 28 days) from baseline (the last 4 weeks prior to randomization) to the entire Maintenance Period (Weeks 1 through 12), as captured in the daily seizure eDiary","definition_or_measurement_approach":"Responder analysis (≥50% reduction in CMS frequency) comparing baseline (4 weeks pre-randomization) to Maintenance Period (Weeks 1–12) using daily seizure eDiary entries."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: Change in the number of CMS-free days (per 28 days) from baseline (the last 4 weeks prior to randomization) to the entire Maintenance Period (Weeks 1 through 12), as captured in the daily seizure eDiary","definition_or_measurement_approach":"Change in CMS-free days per 28 days computed from caregiver daily eDiary comparing baseline period to Maintenance Period (Weeks 1–12)."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: Cumulative distribution of percent reduction in seizure frequency from Baseline to the entire Maintenance Period (Weeks 1 through 12)","definition_or_measurement_approach":"Cumulative distribution function of percent reduction in seizure frequency from baseline to Maintenance Period using eDiary-recorded seizures."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: GRIN-NDD-specific CGI-C (GRIN-CGI-C) evaluated at the end of the Maintenance Period (Week 12) relative to baseline for relevant domains, including expressive communication, receptive communication, fine motor, gross motor, and dysregulated behavior","definition_or_measurement_approach":"GRIN-specific Clinical Global Impression of Change (GRIN-CGI-C) assessed at Week 12 vs baseline across specified functional domains; clinician/caregiver-rated instrument per protocol."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: Change from baseline to the end of the Maintenance Period (Week 12) in the ABC 2C irritability subscale score","definition_or_measurement_approach":"ABC-2C irritability subscale change from baseline to Week 12."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: Change from baseline to the end of the Maintenance Period (Week 12) in the Vineland Adaptive Behavior Scale, Third Edition (VABS-3) Daily Living personal subdomain score","definition_or_measurement_approach":"VABS-3 Daily Living personal subdomain change from baseline to Week 12 assessed per instrument guidance."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED QUALIFYING SEIZURES COHORT: Change from baseline to the end of the Maintenance Period (Week 12) in quality of life as measured by the PedsQL","definition_or_measurement_approach":"PedsQL change from baseline to Week 12 measured as per questionnaire scoring."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED WITHOUT QUALIFYING SEIZURES AUXILIARY COHORT: GRIN-CGI-C evaluated at the end of the Maintenance Period (Week 24) relative to baseline for relevant domains, including expressive communication, receptive communication, fine motor, gross motor, and dysregulated behavior","definition_or_measurement_approach":"GRIN-CGI-C change from baseline to Week 24 in specified domains for auxiliary cohort; clinician/caregiver-rated per protocol."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED WITHOUT QUALIFYING SEIZURES AUXILIARY COHORT: Change from baseline to the end of the Maintenance Period (Week 24) in the VABS-3 Daily Living personal subdomain score","definition_or_measurement_approach":"VABS-3 Daily Living personal subdomain change from baseline to Week 24."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED WITHOUT QUALIFYING SEIZURES AUXILIARY COHORT: Change from baseline to the end of the Maintenance Period (Week 24) in quality of life as measured by the PedsQL","definition_or_measurement_approach":"PedsQL change from baseline to Week 24 per questionnaire scoring."}
• {"endpoint_text":"- PART A – PHASE 3 RANDOMIZED WITHOUT QUALIFYING SEIZURES AUXILIARY COHORT: AEs, SAEs, and ADRs (frequency, type, severity, and duration)","definition_or_measurement_approach":"Safety monitoring using AE/SAE/ADR reporting throughout the Maintenance Period (Week 24) per protocol."}
• {"endpoint_text":"- PART B – OLE STUDY: Percent change in the CMS frequency per 28 days from baseline to each 12-week interval in the open-label extension (OLE) through the end of the OLE, as captured in the daily seizure eDiary","definition_or_measurement_approach":"Percent change in CMS frequency per 28 days computed at each 12-week interval during OLE using caregiver daily eDiary entries."}
• {"endpoint_text":"- PART B – OLE STUDY: Change in the number of CMS-free days per 28 days from baseline to each 12-week interval in the OLE through the end of the OLE, as captured in the daily seizure eDiary","definition_or_measurement_approach":"Change in CMS-free days per 28 days at each 12-week interval in OLE measured from eDiary."}
• {"endpoint_text":"- PART B – OLE STUDY: Change from baseline to each open-label treatment visit through the end of open-label treatment in the ABC-2C irritability subscale score, the VABS-3 Daily Living personal subdomain score, the PedsQL, and the GRIN-CGI-C scale for relevant domains (including expressive communication, receptive communication, fine motor, gross motor, and dysregulated behavior) Note: VABS-3 and GRIN-CGI-C scales to be assessed for Phase 3 participants only","definition_or_measurement_approach":"Repeated assessments during OLE of ABC-2C, VABS-3, PedsQL and GRIN-CGI-C where applicable; changes from baseline reported at open-label visits."}

Methods

• Patient recruitment, material development and supply, website design and management provided by Innovative Trials Limited (duties listed: Patient recruitment, material development and supply, website design and management).
• Patient reimbursement, home nurse and travel support provided by MD Group (duties: Patient Reimbursement, home nurse and travel support).
• Seizure classification training and review and engagement with epilepsy clinical network provided by Epilepsy Study Consortium Inc. (duties include seizure classification training and review).
• IMP shipment logistics (to/from clinical sites and patient homes) provided by Marken LLP (duties: IMP shipment to/from clinical site and patient home address).
• eCOA/ePRO services and backup device supply provided by Umotif Limited (duties: eCOA/ePRO services and back-up device supply).
• At-home EEG data collection and advanced analysis in rare pediatric epilepsy provided by Clouds of Care (duties: (At-Home) EEG Data Collection and Advanced Analysis in Rare Pediatric Epilepsy).
• Central services (ECG provider and analysis, sample storage/shipment coordination, long-term sample storage, lab analysis) provided by various vendors (e.g., Eresearchtechnology Inc./Clario for ECG provider and analysis; Acm Medical Laboratory Inc. for sample storage and coordination).

Netherlands

Earliest CTIS Part Ii Submission Date

22-12-2023

Latest Decision Or Authorization Date

20-04-2026

Processing Time Days

850

Number Of Sites

2

Number Of Participants

9

France

Earliest CTIS Part Ii Submission Date

26-11-2024

Latest Decision Or Authorization Date

15-04-2026

Processing Time Days

505

Number Of Sites

6

Number Of Participants

8

Belgium

Earliest CTIS Part Ii Submission Date

25-09-2024

Latest Decision Or Authorization Date

17-04-2026

Processing Time Days

569

Number Of Sites

2

Number Of Participants

4

Italy

Earliest CTIS Part Ii Submission Date

22-12-2023

Latest Decision Or Authorization Date

16-04-2026

Processing Time Days

846

Number Of Sites

5

Number Of Participants

14

Spain

Earliest CTIS Part Ii Submission Date

22-12-2023

Latest Decision Or Authorization Date

15-04-2026

Processing Time Days

845

Number Of Sites

1

Number Of Participants

2

Germany

Earliest CTIS Part Ii Submission Date

22-12-2023

Latest Decision Or Authorization Date

15-04-2026

Processing Time Days

845

Number Of Sites

2

Number Of Participants

10

Poland

Earliest CTIS Part Ii Submission Date

05-12-2024

Latest Decision Or Authorization Date

20-04-2026

Processing Time Days

501

Number Of Sites

2

Number Of Participants

7

Slovenia

Earliest CTIS Part Ii Submission Date

15-10-2024

Latest Decision Or Authorization Date

28-04-2026

Processing Time Days

560

Number Of Sites

1

Number Of Participants

2

Primary sponsor

Full Name

Grin Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Innovative Trials Limited

Responsibilities

Patient recruitment, material development and supply, website design and management

Name

Marken LLP

Responsibilities

IMP shipment logistics to/from clinical sites and patient homes

Name

Pharmaceutical Research Associates Group B.V.

Responsibilities

Local site services / IBL site services (sponsor duties code present)

Name

Epilepsy Study Consortium Inc.

Responsibilities

Seizure classification training and review and network support

Third parties

• {"country":"Australia","full_name":"Cogstate Limited","duties_or_roles":"Rater Training, Scale licensing, central monitoring of raters","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Study data platform/services (sponsor duties code present)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG provider and analysis","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Etymax Limited","duties_or_roles":"Study documents translations","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Acm Medical Laboratory Inc.","duties_or_roles":"Sample storage until the end of the study; shipment coordination; laboratory testing duties","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Welocalize Inc.","duties_or_roles":"Translations","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Pharmaceutical Research Associates Group B.V.","duties_or_roles":"Local site services (sponsor duties code present)","organisation_type":"Pharmaceutical company"}
• {"country":"Italy","full_name":"Aptuit (Verona) S.r.l.","duties_or_roles":"Preclinical/ADME services (sponsor duties code present)","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"Clouds of Care","duties_or_roles":"(At-Home) EEG Data Collection and Advanced Analysis in Rare Pediatric Epilepsy","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Certara INC","duties_or_roles":"Modeling/simulation/PKPD support (sponsor duties code present)","organisation_type":"Health care"}
• {"country":"United States","full_name":"Epilepsy Study Consortium Inc.","duties_or_roles":"Seizure classification training and review; network support","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Marken LLP","duties_or_roles":"IMP shipment to/from clinical site and patient home address","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"MD Group","duties_or_roles":"Patient Reimbursement, home nurse and travel support","organisation_type":"Health care"}
• {"country":"France","full_name":"Ennov","duties_or_roles":"Clinical trial system/support (sponsor duties code present)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Lumanity Patient Centered Outcomes LLC","duties_or_roles":"Scheduling and conducting entry and end of maintenance/end of study interviews in English; analyzing interview data and delivering results","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Pacific BioPharma Logistics, Inc.","duties_or_roles":"Long-term storage of de-identified samples","organisation_type":"Industry"}
• {"country":"United Kingdom","full_name":"Umotif Limited","duties_or_roles":"eCOA/ePRO services and back-up device supply","organisation_type":"Non-Pharmaceutical company"}
• {"country":"France","full_name":"Qualworld","duties_or_roles":"Scheduling and conducting entry and end of maintenance/end of study interviews in non-English languages; transcribing and translating non-English interviews","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Innovative Trials Limited","duties_or_roles":"Patient recruitment, material development and supply, website design and management","organisation_type":"Pharmaceutical company"}