Allogenic bone marrow-derived mesenchymal stem cells transduced with ICOVIR-5, ex vivo expanded for Autoimmune refractory epilepsy|Rasmussen encephalitis

Inclusion criteria

• {"criterion_text":"- Acceptance of informed consent.\n- Paediatric population under 16 years of age.\n- Epilepsy of immune etiology refractory to first or second line treatment (antiepileptic drugs and conventional immunomodulatory therapy) in which motor and/or cognitive impairment is observed. A special group will be those undergoing epilepsy surgery in the context of Rasmussen's Encephalitis.\n- Possibility of performing CMR, electrophysiological tests, PET and neuropsychological evaluations."}

Exclusion criteria

• {"criterion_text":"- Concomitant illnesses that may affect patient safety or outcome such as: - Active CNS or systemic active infections - Active tumour - Active chemotherapy - Cardiac disease - Dementia or other degenerative CNS disease\n- Any condition in which catheterisation of the arterial system is contraindicated or not feasible (major behavioural disorders preventing adequate collaboration, difficult arterial access or clinically relevant coagulation parameters).\n- Any condition where therapeutic products cannot be administered (history of frequent adverse reactions or thromboembolic complications associated with blood components).\n- Refusal of the patient or family members to adequate follow-up."}

Primary endpoints

• {"endpoint_text":"- To measure dose-limiting toxicities (DLTs, defined in the protocol as MSC infusion-related grade 3 toxicities occurring in the first 28 days of treatment).","definition_or_measurement_approach":"DLTs defined in the protocol as MSC infusion-related grade 3 toxicities occurring in the first 28 days of treatment."}

Secondary endpoints

• {"endpoint_text":"- To asses clinical response","definition_or_measurement_approach":"Clinical response assessed by detecting changes in neurological assessments, neuropsychological evaluations, structural and functional neuroimaging tests, and neurophysiological tests (as described in trial secondary objectives)."}

Other endpoints

• {"endpoint_text":"- Determine the effect of therapy on the patient's immune system and mRNA profile, both peripherally (peripheral blood) and locally in the primary sample obtained during surgery.","definition_or_measurement_approach":"Immune system and mRNA profile measured peripherally (peripheral blood) and locally in primary surgical samples."}
• {"endpoint_text":"- To study the molecular profile of the mesenchymal stem cells used in the trial and correlate them with the clinical outcome.","definition_or_measurement_approach":"Molecular profiling of the mesenchymal stem cells used in the trial and correlation of those profiles with clinical outcomes."}

Spain

Earliest CTIS Part Ii Submission Date

27-12-2024

Latest Decision Or Authorization Date

24-01-2025

Processing Time Days

28

Number Of Sites

1

Number Of Participants

10

Primary sponsor

Full Name

Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Princesa

Organisation Type

Laboratory/Research/Testing facility

Country Of Registered Address

Spain