Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis: A multicenter, randomized placebo controlled and biological endpoints clinical Trial
Key facts
- Where
- Novara, Padova, Palermo és San Giovanni Rotondo, OlaszországSee the 4 sites
- Phase
- 2. fázis
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 64 years old?
Where in Olaszország
- Azienda Ospedaliero Universitaria Maggiore della CaritàNovara, OlaszországTaking people
- Azienda Ospedaliera di PadovaPadova, OlaszországTaking people
- Azienda Ospedaliera Universitaria Policlinico Paolo GiacconePalermo, OlaszországTaking people
- Casa Sollievo della SofferenzaSan Giovanni Rotondo, OlaszországTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Casa Sollievo Della Sofferenza
- Trial code
- EU 2024-518888-35-00
- Record updated
- 2025. december 17.
- Original description
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.Read full original descriptionShow less
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.
Inclusion: 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.
Exclusion: 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; 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; Contraindications to perform MRI scans, CSF withdrawal and Skin biopsy; Patient unable to understand informed consent form; Pregnancy and breast feeding.