Actively Recruiting
Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles
Led by Fondation Ophtalmologique Adolphe de Rothschild · Updated on 2026-01-12
60
Participants Needed
1
Research Sites
206 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 years or older
- Diagnostic suspicion of amyotrophic lateral sclerosis (ALS) for patients
- Planned inpatient admission for standard diagnostic work-up including clinical evaluation, neuropsychological assessment, nerve conduction studies/EMG, motor evoked potentials (TMS/MEP), brain and spinal MRI, pulmonary function testing, lumbar puncture, and blood tests
- Explicit written informed consent to participate
- Affiliation with or beneficiary of a social security system
- For healthy controls: adults aged 18 years or older, explicit written informed consent, and social security affiliation
You will not qualify if you...
- Patient under legal protection or guardianship
- Pregnant or breastfeeding women
- Active infection within 4 weeks prior to biological sampling
- Ongoing diagnosis and management of cancer
- ALS diagnosis not confirmed (secondary exclusion for patients)
- Diagnosed neurological disease (for healthy controls)
- Self-reported cognitive impairment (for healthy controls)
AI-Screening
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Trial Site Locations
Total: 1 location
1
Hopital Fondation Adolphe de Rothschild
Paris, France, 75019
Actively Recruiting
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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