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Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles

NCT07334743 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 60

Last updated 2026-01-12

No results posted yet for this 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).

Conditions

Sponsors & Collaborators

  • Fondation Ophtalmologique Adolphe de Rothschild

    lead NETWORK

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2025-09-17
Primary Completion
2029-09-30
Completion
2029-09-30

Countries

  • France

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07334743 on ClinicalTrials.gov