NCT07268833Recruiting
Translating Single-cell Vulnerability Into Novel ALS Biomarkers and Therapeutic Targets: Towards a Liquid Nerve Biopsy
Source: ClinicalTrials.gov · Last updated 8 Dec 2025
Brief summary (registry, in English)
The progress of ALS research and clinical practice is hampered by lack of effective biomarkers to monitor disease onset and progression. In response to this urgent need, we will integrate single-cell system biology approaches, histopathological and clinical data from precious human nerve biopsies collected from living ALS patients during the diagnostic workup and findings from innovative preclinical mouse models to unmask cell-specific molecular alterations that arise in the PNS tissue during the course of ALS pathology. This information will be used to select protein biomarkers of dysfunctional states associated with pre-manifest or early symptomatic stages of the disease, which will be further screened and validated in patient biofluids. Altogether, this project will lead to the discovery of novel, reliable and specific ALS biomarkers while providing insights into ALS mechanisms by leveraging an original "PNS perspective" on disease pathogenesis.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 400 (estimated)
- First submitted
- 25 Nov 2025
- Start date
- 24 Oct 2024
- Primary completion date
- Apr 2027
- Lead sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT07268833
What you commit to
- Study type: Observational
- Planned participants: 400
- From 24 Oct 2024 to the primary completion date, Apr 2027
- 4 sites 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.
Other illness
- nutritional or respiratory failure;
- significant hepatic or chronic renal failure or any interveninginfective or metabolic conditions potentially influencing CBs levels.
- significant hepatic or chronic renal failure or any interveninginfective or metabolic conditions potentially influencing CBs levels.
Other
- FVC \<60%;
Who the study is looking for
- Age equal or over 18 years old
- ALS patients, diagnosed accordingly to the revised El Escorial Criteria
- Disease duration \<24 months from symptom onset.
- Age equal or over 18 years old
- Patients with genetic defined susceptibility to ALS and one or more strict relative affected from ALS Exclusion criteria for ALS pre-symptomatic patients
- significant hepatic or chronic renal failure or any interveninginfective or metabolic conditions potentially influencing CBs levels.
- Age equal or over 18 years old
- Subjects without a diagnosis of neurodegenerative disease or neuromuscular disorder.
- Age equal or over 18 years old
- For AD: Diagnosis according to 2018 NIA-AA Framework for Alzheimer's Disease
- For FTD: Diagnosis according to 2011 International Behavioural Variant FTD Criteria Consortium
- For PD: Diagnosis according to 2015 Movement Disorder Society criteria
- For DLB: Diagnosis according to 2017 Fourth Consensus Report of the DLB Consortium Exclusion criteria for non ALS neurodegenerative patients
- significant hepatic or chronic renal failure or any interveninginfective or metabolic conditions potentially influencing CBs levels.
- Age equal or over 18 years old
- Presence of axonal or demyelinating neuropathy Exclusion criteria for neuromuscular patients
- significant hepatic or chronic renal failure or any interveninginfective or metabolic conditions potentially influencing CBs levels.
Age in the registry: 18 years to - · Sex: All · Accepts healthy volunteers
Sites (4)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Fondazione IRCCS Istituto Neurologico Carlo Besta Coordinator of this site? Confirm its status | Milan | Italy | Recruiting |
| IRCCS Ospedale San Raffaele SRL Coordinator of this site? Confirm its status | Milan | Italy | Recruiting |
| Azienda Ospedaliero-Universitaria Di Cagliari Coordinator of this site? Confirm its status | Monserrato | Italy | Recruiting |
| Azienda Ospedaliera Universitaria Federico II Coordinator of this site? Confirm its status | Naples | Italy | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.