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Clinical Trials in France / NCT07334743
Recruiting Observational

Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles

NCT07334743 · tracked via the Priya Life Science France tracker
Sponsor
Fondation Ophtalmologique Adolphe de Rothschild
Phase
Observational
Started
2025-09-17
Last updated
2026-01-12

Condition(s) studied

Amyotrophic Lateral SclerosisENGRAILED1

Study summary

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).

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
Accepted
Inclusion Criteria: 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 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.

Primary outcome measure(s)

  • Identification by RNA-seq of biomarkers carried by neuron-derived extracellular vesicles (NVEC) present in the blood of ALS patients — at inclusion visit, month 6
    Identification by RNA-seq of biomarkers carried by neuron-derived extracellular vesicles (NVEC) present in the blood of ALS patients

Trial sites (1)

FacilityCityRegionStatus
Hopital Fondation Adolphe de Rothschild Paris France Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07334743 on ClinicalTrials.gov ↗ ← All trials in France