Blood RNA biomarkers for ALS using neuron-derived extracellular vesicles
Official title: Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles
Run by Fondation Ophtalmologique Adolphe de Rothschild · NCT07334743 · Data updated 2026-11-12
This will try to see if RNA signatures in neuron-derived extracellular vesicles from blood can identify biomarkers in adults with suspected sporadic ALS compared with healthy volunteers.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 60 (estimated) |
| Sponsor | Fondation Ophtalmologique Adolphe de Rothschild Research network |
| Locations | 1 site (Paris) |
| Status | Recruiting |
| Started | September 17, 2025 |
| Main results expected | September 2029 |
| Study ends | September 2029 |
| Registry record updated | January 12, 2026 |
| Trial ID | NCT07334743 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are adults (≥18) undergoing diagnostic work-up for suspected sporadic ALS who can give informed consent and attend inpatient evaluations at the study center, with matched healthy adults without neurological disease serving as controls.
✕Probably not a fit
People with familial/genetic ALS, active infection within 4 weeks, ongoing cancer treatment, pregnant or breastfeeding individuals, or those unable to provide informed consent are excluded and are unlikely to benefit from participation.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
Researchers will collect blood from 30 adults with suspected sporadic ALS and 30 age- and sex-matched healthy volunteers and immuno-isolate neuron-derived extracellular vesicles for RNA sequencing. They will compare RNA expression patterns to discover NVEC-borne biomarkers that differ between patients and controls. Candidate biomarkers will be tested for EN1 modulation in En1+/- mouse models and in induced pluripotent stem cell–derived motor neurons from ALS patients to seek Prentice-style surrogate endpoints. The prospective cohort design aims to identify circulating markers that could enable and de-risk early-phase EN1 therapy trials.
Why it matters
Potential benefit: If successful, identified biomarkers could enable earlier, less invasive monitoring and speed up development of EN1-based therapies for ALS.
How similar studies have gone: Prior studies using neuron-derived extracellular vesicles and RNA profiling have reported promising biomarker signals in neurodegenerative disorders, but applying EN1-responsive NVEC biomarkers specifically to ALS remains relatively novel.
Who can join: the full rules
Show full inclusion / exclusion 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.
Where it's running
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Hopital Fondation Adolphe de Rothschild
Paris, France Recruiting
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.