Searching Blood Markers in ALS Using Nerve Cell Vesicles
Looks for blood markers in adults with suspected sporadic ALS compared with healthy volunteers.
Plain-language title generated from the registry record
Registry title: Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles
Key facts
- Where
- Paris, FranceSee the site
Where in France
- Hopital Fondation Adolphe de RothschildParis, FranceTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Fondation Ophtalmologique Adolphe de Rothschild
- Trial code
- NCT07334743
- Record updated
- 12 January 2026
- Original description
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.Read full original descriptionShow less
Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles
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 suspected sporadic ALS; healthy volunteers without neurological disease.
Key procedures and timeline. Baseline (M0, inpatient): ALSFRS-R, MRC, hand dynamometry, eye-movement recording (MOC); NCS/EMG (NUMIX), TMS/MEP 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).
Follow-up: M6 clinic visit (repeat clinical/electrophysiology/neuropsychology/PFTs as per care) with blood (15 mL); additional routine follow-ups at M12, M18, M24 (clinical; MOC at M12 and M24).
Controls: single visit with blood (3×5 mL EDTA) and cortical excitability; brain MRI for targeting.
Sample size. 60 participants total (30 ALS, 30 controls).
Inclusion: For patients :; Age ≥ 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 ≥ 18 years. Explicit written informed consent to participate in the study. Affiliation with or beneficiary of a social security system.
Exclusion: Active infection within the 4 weeks prior to biological sampling; Diagnosed neurological disease; Self-reported cognitive impairment; Current diagnosis and management of cancer; Patient under legal protection/guardianship; Pregnant or breastfeeding woman.