NCT07334743Recruiting
Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles
Source: ClinicalTrials.gov · Last updated 12 Jan 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 60 (estimated)
- First submitted
- 31 Dec 2025
- Start date
- 17 Sept 2025
- Primary completion date
- Sept 2029
- Lead sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT07334743
What you commit to
- Study type: Observational
- Planned participants: 60
- From 17 Sept 2025 to the primary completion date, Sept 2029
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Active infection within the 4 weeks prior to biological sampling.
Other illness
- Diagnosed neurological disease.
- Self-reported cognitive impairment.
- Current diagnosis and management of cancer.
Other
- Patient under legal protection/guardianship.
- Pregnant or breastfeeding woman.
Who the study is looking for
- 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.
Age in the registry: 18 years to - · Sex: All · Accepts healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Hopital Fondation Adolphe de Rothschild Coordinator of this site? Confirm its status | Paris | France | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.