4-FLUORO-N-[2-[4-(2-METHOXYPHENYL)PIPERAZIN-1-YL]ETHYL]-N-PYRIDIN-2-YLBENZAMIDE for Dravet syndrome | Drug-resistant focal epilepsy

Inclusion criteria

• {"criterion_text":"- Patients with Dravet Syndrome: Adult patients (≥ 18 but < 60 years)\n- Patients with drug-resistant focal epilepsy: Patient who gave her/his written informed consent to participate to the study\n- Patients with drug-resistant focal epilepsy: For women of childbearing potential, use highly effective contraception during study participation\n- Healthy controls: Adult (≥ 18 years < 50 years)\n- Healthy controls: Without history of neurological disorders, and/or general medical disorders\n- Healthy controls: Subject who gave her/his written informed consent to participate to the study\n- Healthy controls: For women of childbearing potential, use highly effective contraception during study participation\n- Patients with Dravet Syndrome: Diagnosis of Dravet syndrome will be confirmed based on medical history, type of seizures, EEG data and results of genetic testing 3.\tNo restriction related to the seizure frequency\n- Patients with Dravet Syndrome: No restriction related to the seizure frequency\n- Patients with Dravet Syndrome: Patient assent and patient (or patient’s legal representative guardianship) who gave its written informed consent to participate to the study\n- Patients with Dravet Syndrome: For women of childbearing potential, use highly effective contraception during study participation.\n- Patients with drug-resistant focal epilepsy: Adult patient (≥ 18 years)\n- Patients with drug-resistant focal epilepsy: Patient suffering from drug-resistant focal epilepsy according to ILAE classification\n- Patients with drug-resistant focal epilepsy: Patient in whom presurgical evaluation is considered\n- Patients with drug-resistant focal epilepsy: No restriction related to the seizure frequency"}

Exclusion criteria

• {"criterion_text":"- Patients with Dravet Syndrome: Subject in exclusion period of another study\n- Patients with Dravet Syndrome: Persons not affiliated to a social security scheme or beneficiaries of a similar scheme\n- Patients with drug-resistant focal epilepsy: Subject in exclusion period of another study\n- Patients with drug-resistant focal epilepsy: MRI contra-indication (presence of metallic elements, claustrophobia)\n- Patients with drug-resistant focal epilepsy: Presence of Vagal Nerve Stimulation\n- Patients with drug-resistant focal epilepsy: Ongoing serotoninergic treatment, including selective serotonin reuptake inhibitor\n- Patients with drug-resistant focal epilepsy: Pregnant women, women in labor or breastfeeding women.\n- Patients with drug-resistant focal epilepsy: Severe renal failure (Glomerular filtration rate < 30 ml/min)\n- Patients with drug-resistant focal epilepsy: Hypersensitivity to [18F] MPPF\n- Patients with drug-resistant focal epilepsy: Persons deprived of their liberty by a judicial or administrative decision\n- Patients with drug-resistant focal epilepsy: Persons under psychiatric care\n- Patients with Dravet Syndrome: MRI contra-indication (presence of metallic elements, claustrophobia, Patients unable to maintain a minimul level of immobility during the imaging acquisition)\n- Patients with drug-resistant focal epilepsy: Persons admitted to a health or social institution for purposes other than research\n- Patients with drug-resistant focal epilepsy: Adults subject to a legal protection measure (guardianship, curatorship)\n- Patients with drug-resistant focal epilepsy: Persons not affiliated to a social security scheme or beneficiaries of a similar scheme\n- Healthy controls: Presence of the symptoms of anxiety and/or depression as defined by a score ≥ 11 at the French version of the Hospital Anxiety and Depression Scale (HADS)\n- Healthy controls: Ongoing treatment with selective serotonin reuptake inhibitor\n- Healthy controls: MRI contra-indication (presence of metallic elements, claustrophobia)\n- Healthy controls: Pregnant women, women in labor or breastfeeding women.\n- Healthy controls: Severe renal failure (Glomerular filtration rate < 30 ml/min)Hypersensitivity to [18F] MPPF\n- Healthy controls: Hypersensitivity to [18F] MPPF\n- Healthy controls: Persons deprived of their liberty by a judicial or administrative decision\n- Patients with Dravet Syndrome: Presence of Vagal Nerve Stimulation\n- Healthy controls: Persons under psychiatric care\n- Healthy controls: Persons admitted to a health or social institution for purposes other than research\n- Healthy controls: Adults subject to a legal protection measure (guardianship, curatorship)\n- Healthy controls: Persons not affiliated to a social security scheme or beneficiaries of a similar scheme\n- Patients with Dravet Syndrome: Patients unable to maintain a minimul level of immobility during the imaging acquisition\n- Patients with Dravet Syndrome: Pregnant women, women in labor or breastfeeding women.\n- Patients with Dravet Syndrome: Severe renal failure (Glomerular filtration rate < 30 ml/min)\n- Patients with Dravet Syndrome: Hypersensitivity to [18F] MPPF\n- Patients with Dravet Syndrome: Persons deprived of their liberty by a judicial or administrative decision\n- Patients with Dravet Syndrome: Persons under psychiatric care"}

Primary endpoints

• {"endpoint_text":"- Comparison of the brainstem BPND of [18F]-MPPF across patients groups (DS and focal epilepsy)","definition_or_measurement_approach":"Brainstem non-displaceable binding potential (BPND) of [18F]-MPPF measured by PET using the [18F]-MPPF radioligand (PET-MRI acquisition with injection of the radioligand)."}

Secondary endpoints

• {"endpoint_text":"- Comparison of the brainstem BPND of [18F]-MPPF of each patients group (DS and focal epilepsy) with the one of healthy subjects","definition_or_measurement_approach":"Brainstem BPND of [18F]-MPPF measured by PET comparing each patient group with healthy subjects."}
• {"endpoint_text":"- Comparison of the cortical BPND of [18F]-MPPF across patients groups (DS and focal epilepsy) and with the one of healthy subjects","definition_or_measurement_approach":"Cortical BPND of [18F]-MPPF measured by PET across patient groups and compared to healthy subjects."}
• {"endpoint_text":"- aluating the relation between [18F]-MPPF PET brainstem regional distribution of 5HT1A receptors and inter-ictal respiratory function in patients with DS","definition_or_measurement_approach":"Assessment of association between regional PET distribution of 5HT1A receptors (using [18F]-MPPF PET) and measures of inter-ictal respiratory function in DS patients."}
• {"endpoint_text":"- Comparing brainstem regional volumes on MRI of patients with DS with the one of patients with drug-resistant focal epilepsy","definition_or_measurement_approach":"Comparative measurement of brainstem regional volumes using MRI between DS patients and patients with drug-resistant focal epilepsy."}
• {"endpoint_text":"- Comparing brainstem regional volumes on MRI of each patients group (DS and focal epilepsy) with the one of healthy subjects","definition_or_measurement_approach":"MRI-derived brainstem regional volumes compared between each patient group and healthy subjects."}
• {"endpoint_text":"- Evaluating the relation between the brainstem regional volumes on MRI and inter-ictal respiratory function in patients with DS","definition_or_measurement_approach":"Assessment of correlation between MRI-measured brainstem regional volumes and inter-ictal respiratory function in DS patients."}

Methods

• Site-based recruitment at Hospices Civils De Lyon (Department of Functional Neurology and Epileptology) using site contact (email/telephone) in France
• Recruitment email targeted to healthy control participants (document: L2_Other subject information material recruitment email control)
• Recruitment flyer targeted to healthy control participants (document: L2_Other subject information material recruitment flyer control)
• Recruitment of patient groups (Dravet syndrome and drug-resistant focal epilepsy) via clinic lists/presurgical evaluation pathways at the investigational site

France

Earliest CTIS Part Ii Submission Date

15-01-2026

Latest Decision Or Authorization Date

06-02-2026

Processing Time Days

22

Number Of Sites

1

Number Of Participants

30

Primary sponsor

Full Name

Hospices Civils De Lyon

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France