2024-518888-35-00RecruitingPhase 2
Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis: A multicenter, randomized placebo controlled and biological endpoints clinical Trial
Source: EU CTIS (euclinicaltrials.eu) · Last updated 17 Dec 2025
Brief summary (registry, in English)
The primary trial outcome is safety, assessed with respect to the incidence of treatment-emergent AEs (TEAEs) and serious AEs (SAEs) over the whole study period. AEs are coded using MedDRA and they should be reported to competent authorities only if unexpected and related to Intracerebroventricular administration of human Neural Stem Cells.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 30 (estimated)
- First submitted
- 15 Oct 2024
- Start date
- 24 Jan 2024
- Lead sponsor
- Casa Sollievo Della Sofferenza
- Diseases
- ALS (motor neurone disease)
- EU CT number
- 2024-518888-35-00
- EudraCT number
- 2018-002373-22
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 30
- 4 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of EU CTIS (euclinicaltrials.eu). It describes the study design.
AI-kivonat: who the study is looking for
Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with definite or probable ALS whose disease began within the last 24 months and is getting worse quickly. The treatment is neural stem cells given into the brain fluid spaces.
- Age
- 18-65 years in the criteria (the registry lists 18 to 64 years)
- Sex
- All sexes can join.
- Disease stage
- Definite or probable ALS by the revised El Escorial criteria. Onset 24 months ago or less. FVC 70% or more. ALSFRS-R score of at least 26. Fast progression: a drop of 1 point or more per month during a 12-week run-in period.
- Earlier treatment
- Must be on a stable dose of Riluzole for more than 30 days, or not take Riluzole at all and not plan to start it. Must not have had any stem cell or somatic cell therapy. Must not have joined another treatment trial or taken experimental drugs in the last 1 month.
Main reasons for not taking part
- Psychiatric disease or other neurological diseases besides ALS
- Cancer within the previous 10 years
- Immunosuppressive therapy within 12 weeks of screening, or active autoimmune disease or infection (hepatitis B, hepatitis C, HIV)
- Cognitive impairment
- Pregnancy or breastfeeding
Read by machine on 9 Nov 2026.
Who cannot take part
Copied verbatim from EU CTIS (euclinicaltrials.eu) and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study.
- Evidence of any concurrent illness or treatments limiting the safety to participate or any condition that the neurosurgeon feels may pose complications for the surgery;
- Cancer within the previous 10 years;
- Patient has been treated previously with any stem cell or somatic cells therapy;
Other illness
- Psychiatric disease or other neurological diseases different from ALS
- Immunosuppressive therapy within 12 weeks of screening; active autoimmune disease or infection (including hepatitis B, hepatitis C, or HIV);
- Cognitive impairment;
Other
- Contraindications to perform MRI scans, CSF withdrawal and Skin biopsy
- Patient unable to understand informed consent form;
- Pregnancy and breast feeding;
Who the study is looking for
- Patient provides written informed consent, informed consent signature collection prior to any study procedure (patient has good acceptance and understanding of the informed consent);
- Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines.
- Definite, probable diagnosis according to the revised El Escorial criteria;
- Age: 18-65 years;
- FVC ≥ 70%;
- Onset ≤ 24 months;
- Patients with an ALSFRS-R score of at least 26; overall, including a score of at least 2 on each of the 1-9 ALSFRS-R individual component items and of at least 3 of the 10-12 individual components items;
- Evidence of fast progression of the disease. We exclude slow progressors at the time of screening defined as Patient with an ALSFRS-R total score progression between onset of the disease and screening of < 0.3 per month. We document the fast progression of the disease defined as ALSFRS-R total score decrease of ≥ 1 point per month during a 12 week run-in period between screening and randomization;
- Patient should be on a stable dose of Riluzole for > 30 days from pre-screening visit or not taking riluzole at all, nor plan to begin riluzole during the study period;
- Patient is medically able to tolerate transient immunosuppression regimen;
Age in the registry: 18 years to 64 years · Sex: All · Does not accept healthy volunteers
Sites (4)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Azienda Ospedaliero Universitaria Maggiore della Carità | Novara | Italy | Recruiting |
| Azienda Ospedaliera di Padova | Padova | Italy | Recruiting |
| Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone | Palermo | Italy | Recruiting |
| Casa Sollievo della Sofferenza | San Giovanni Rotondo | Italy | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.