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โ† Trials
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Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles

Recruitingn/aAges 18 Years - anyAllFondation Ophtalmologique Adolphe de Rothschild
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In plain words

What the trial is, and whether it could suit you

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/II) EN1 trials. Primary endpoint. Discovery: RNA-seq identification of circulating NVEC-borne biomarkers that differ between sporadic ALS patients and healthy controls. EN1 modulation: Demonstration that these biomarkers are modulated by EN1 in En1+/- mouse models and in ALS patient iPSC-derived motor neurons. Design. Prospective cohort, N=60 (30 sporadic ALS; 30 healthy controls matched on age/sex). Population. Adults undergoing diagnostic work-up for

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๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: For patients : Age โ‰ฅ 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/EMG, motor evoked potentials (TMS/MEP), brain and spinal MRI, pulmonary function testing, lumbar puncture, and standard blood tests. Explicit written informed consent to participate in the study. Affiliation with or beneficiary of a social security system. Non-inclusion criteria: Patient under legal protection/guardianship. Pregnant or breastfeeding woman. Active infection within the 4 weeks preceding biological sampling. Ongoing diagnosis and management of cancer. Secondary exclusion criterion: ALS diagnosis not confirmed. For Healthy Controls : Age โ‰ฅ 18 years. Explicit written informed consent to participate in the study. Affiliation with or beneficiary of a social security system. Exclusion Criteria: * Patient under legal protection/guardianship. * Pregnant or breastfeeding woman. * Diagnosed neurological disease. * Self-reported cognitive impairment. * Active infection within the 4 weeks prior to biological sampling. * Current diagnosis and management of cancer.
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What it tests

  • Not listed
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Where

1 site
Hopital Fondation Adolphe de Rothschild
Paris, France
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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