[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100618678":3},{"organization":4,"armGroups":7,"interventions":10,"overallOfficials":10,"centralContacts":10,"locations":13,"responsibleParty":33,"collaborators":10,"id":35,"slug":36,"hasResults":37,"nctId":38,"briefTitle":39,"officialTitle":39,"acronym":40,"eligibilityCriteria":41,"healthyVolunteers":42,"sex":43,"minAge":44,"maxAge":10,"enrollmentInfo":45,"targetDuration":10,"studyType":48,"phases":10,"briefSummary":49,"conditions":50,"keywords":10,"overallStatus":16,"whyStopped":10,"lastUpdateSubmitDate":53,"lastUpdatePostDateStruct":54,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":61,"locationsCount":62},{"fullName":5,"class":6},"Fondation Ophtalmologique Adolphe de Rothschild","NETWORK",[8,11],{"label":9,"type":10,"description":10,"interventionNames":10},"patients with ALS",null,{"label":12,"type":10,"description":10,"interventionNames":10},"controls without ALS",[14],{"facility":15,"status":16,"city":17,"state":10,"zip":18,"country":19,"countryCode":20,"cosmosGeoPoint":21,"geoPoint":26,"contacts":27},"Hopital Fondation Adolphe de Rothschild","RECRUITING","Paris","75019","France","FR",{"type":22,"coordinates":23},"Point",[24,25],2.3488,48.85341,{"lat":25,"lon":24},[28],{"name":29,"role":30,"phone":31,"phoneExt":10,"email":32},"Antoine Gueguen, MD","CONTACT","01 48 03 65 65","neurologie@for.paris",{"type":34,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR","100618678","identifying-biomarkers-in-als-patients-using-neuronal-derived-extracellular-vesicles-100618678",false,"NCT07334743","Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles","BALNEV","Inclusion Criteria:\n\nFor patients :\n\nAge ≥ 18 years. Diagnostic suspicion of amyotrophic lateral sclerosis (ALS). Planned inpatient admission in the HFAR neurology department for the standard diagnostic work-up as part of routine care: clinical evaluation, neuropsychological assessment, nerve conduction studies\u002FEMG, motor evoked potentials (TMS\u002FMEP), brain and spinal MRI, pulmonary function testing, lumbar puncture, and standard blood tests.\n\nExplicit written informed consent to participate in the study. Affiliation with or beneficiary of a social security system.\n\nNon-inclusion criteria:\n\nPatient under legal protection\u002Fguardianship. Pregnant or breastfeeding woman. Active infection within the 4 weeks preceding biological sampling. Ongoing diagnosis and management of cancer.\n\nSecondary exclusion criterion:\n\nALS diagnosis not confirmed.\n\nFor Healthy Controls :\n\nAge ≥ 18 years. Explicit written informed consent to participate in the study. Affiliation with or beneficiary of a social security system.\n\nExclusion Criteria:\n\n* Patient under legal protection\u002Fguardianship.\n* Pregnant or breastfeeding woman.\n* Diagnosed neurological disease.\n* Self-reported cognitive impairment.\n* Active infection within the 4 weeks prior to biological sampling.\n* Current diagnosis and management of cancer.",true,"ALL","18 Years",{"count":46,"type":47},60,"ESTIMATED","OBSERVATIONAL","Rationale. ENGRAILED1 (EN1) is under consideration as a therapeutic approach for amyotrophic lateral sclerosis (ALS). To assess EN1 target engagement in patients, we aim to identify EN1-responsive biomarkers suitable as Prentice-style surrogate endpoints. We will discover candidates by RNA-seq of neuron-derived extracellular vesicles (NVEC) immuno-isolated from blood. Establishing such biomarkers would enable and de-risk early-phase (I\u002FII) EN1 trials.\n\nPrimary endpoint. Discovery: RNA-seq identification of circulating NVEC-borne biomarkers that differ between sporadic ALS patients and healthy controls.\n\nEN1 modulation: Demonstration that these biomarkers are modulated by EN1 in En1+\u002F- mouse models and in ALS patient iPSC-derived motor neurons.\n\nDesign. Prospective cohort, N=60 (30 sporadic ALS; 30 healthy controls matched on age\u002Fsex).\n\nPopulation. Adults undergoing diagnostic work-up for suspected sporadic ALS; healthy volunteers without neurological disease.\n\nKey procedures and timeline. Baseline (M0, inpatient): ALSFRS-R, MRC, hand dynamometry, eye-movement recording (MOC); NCS\u002FEMG (NUMIX), TMS\u002FMEP with cortical excitability; neuropsychology; brain \\& spinal MRI; pulmonary function testing; CSF (10 mL) and blood (15 mL) for clinical labs and research (NVEC immunocapture → RNA-seq; proteomics).\n\nFollow-up: M6 clinic visit (repeat clinical\u002Felectrophysiology\u002Fneuropsychology\u002FPFTs as per care) with blood (15 mL); additional routine follow-ups at M12, M18, M24 (clinical; MOC at M12 and M24).\n\nControls: single visit with blood (3×5 mL EDTA) and cortical excitability; brain MRI for targeting.\n\nSample size. 60 participants total (30 ALS, 30 controls).",[51,52],"Amyotrophic Lateral Sclerosis","ENGRAILED1","2025-12-31",{"date":55,"type":56},"2026-01-12","ACTUAL",{"date":58,"type":56},"2025-09-17",{"date":60,"type":47},"2029-09",{"name":5,"class":6},1]