Amyotrophic Lateral SclerosisBALNEVNCT07334743
Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles
An observational study of Amyotrophic Lateral Sclerosis and ENGRAILED1, run by Fondation Ophtalmologique Adolphe de Rothschild at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, ENGRAILED1
- How many people
- 60 participants
- Where
- 1 siteFrance
- Study dates
- 17 Sep 2025to Sep 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Study Population Description. Prospective cohort of 60 adults: 30 patients undergoing inpatient diagnostic work-up for suspected sporadic ALS at the HFAR neurology department and 30 age/sex-matched healthy controls.
Who may be able to join
- 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:
Show all 9Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Hopital Fondation Adolphe de Rothschild
Paris, 75019
Antoine Gueguen, MD, 01 48 03 65 65, neurologie@for.paris
About this study
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.
Read more
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
- Amyotrophic Lateral Sclerosis, ENGRAILED1
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| patients with ALS | Not described | |
| controls without ALS | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Identification by RNA-seq of biomarkers carried by neuron-derived extracellular vesicles (NVEC) present in the blood of ALS patients
Identification by RNA-seq of biomarkers carried by neuron-derived extracellular vesicles (NVEC) present in the blood of ALS patients
Measured: at inclusion visit, month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2026
- Study start17 Sep 2025
- Primary completionSep 2029 (estimated)
- Study completionSep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Fondation Ophtalmologique Adolphe de RothschildThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
- Sponsor type
- Research network
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07334743
- Sponsor's protocol ID
- AGN_2024_9
- Acronym
- BALNEV
- Status last verified
- Dec 2025
- First submitted
- 31 Dec 2025
- Last updated
- 12 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Identifying Biomarkers in ALS Patients Using Neuronal Derived Extracellular Vesicles
- ClinicalTrials.gov ID
- NCT07334743
- Acronym / sponsor ID
- BALNEV / AGN_2024_9
- Conditions
- Amyotrophic Lateral Sclerosis, ENGRAILED1
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Fondation Ophtalmologique Adolphe de RothschildResearch network
- Study start
- 17 Sep 2025 (actual)
- Primary completion
- Sep 2029 (estimated)
- Study completion
- Sep 2029 (estimated)
- First posted
- 12 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| patients with ALS | – | – |
| controls without ALS | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Study Population Description. Prospective cohort of 60 adults: 30 patients undergoing inpatient diagnostic work-up for suspected sporadic ALS at the HFAR neurology department and 30 age/sex-matched healthy controls.
Key inclusion criteria (9 listed)
- 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.
- Patient under legal protection/guardianship. Pregnant or breastfeeding woman. Active infection within the 4 weeks preceding biological sampling. Ongoing diagnosis and management of cancer.
- ALS diagnosis not confirmed.
- Age ≥ 18 years. Explicit written informed consent to participate in the study. Affiliation with or beneficiary of a social security system.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Identification by RNA-seq of biomarkers carried by neuron-derived extracellular vesicles (NVEC) present in the blood of ALS patients
Time frame: at inclusion visit, month 6
Identification by RNA-seq of biomarkers carried by neuron-derived extracellular vesicles (NVEC) present in the blood of ALS patients
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis and ENGRAILED1, run by Fondation Ophtalmologique Adolphe de Rothschild at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 12 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identification by RNA-seq of biomarkers carried by neuron-derived extracellular vesicles (NVEC) present in the blood of ALS patients (time frame: at inclusion visit, month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jan 2026; AzTrials last refreshed its copy on 6 Nov 2026.