NCT07334743 Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles
| NCT ID | NCT07334743 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Fondation Ophtalmologique Adolphe de Rothschild |
| Condition | Amyotrophic Lateral Sclerosis |
| Study Type | OBSERVATIONAL |
| Enrollment | 60 participants |
| Start Date | 2025-09-17 |
| Primary Completion | 2029-09 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 60 participants in total. It began in 2025-09-17 with a primary completion date of 2029-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
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.
Frequently Asked Questions
Who can join the NCT07334743 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Amyotrophic Lateral Sclerosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07334743 currently recruiting?
Yes, NCT07334743 is actively recruiting participants. Visit ClinicalTrials.gov or contact Fondation Ophtalmologique Adolphe de Rothschild to inquire about joining.
Where is the NCT07334743 trial being conducted?
This trial is being conducted at Paris, France.
Who is sponsoring the NCT07334743 clinical trial?
NCT07334743 is sponsored by Fondation Ophtalmologique Adolphe de Rothschild. The trial plans to enroll 60 participants.
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