Tests neural stem cell treatment in people with amyotrophic lateral sclerosis
Tests injected human neural stem cells against saline placebo in adults with ALS.
Plain-language title generated from the registry record
Registry title: Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis (STEMALS)
Key facts
- Where
- Novara, Padua, Palermo, and San Giovanni Rotondo, ItalySee the 4 sites
- Phase
- Phase 2
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 65 years old?
Source: NCT06344260, who can take part
Where in Italy
- Centro SLA Azienda Ospedaliera Università Maggiore della CaritàNovara, ItalyTaking people
- Azienda Ospedaliera di PadovaPadua, ItalyNot yet recruiting
- Azienza Ospedaliera Universitaria - Policlinico "P. Giaccone" Università degli Studi di PalermoPalermo, ItalyNot yet recruiting
- Casa Sollievo Della Sofferenza IRCCSSan Giovanni Rotondo, ItalyNot yet recruiting
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 IRCCS
- Trial code
- NCT06344260
- Record updated
- 3 April 2024
- Original description
A Not for Profit Phase II Study to Evaluate Safety, Efficacy and Biomarkers secondary endpoints of Human Neural Stem cell intracerebroventricular transplantation in amyotrophic lateral sclerosis patients: a randomized, placebo controlled, triple blind study.Read full original descriptionShow less
Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis: A Multicenter, Randomized Placebo Controlled and Biological Endpoints Clinical Trial
A Not for Profit Phase II Study to Evaluate Safety, Efficacy and Biomarkers secondary endpoints of Human Neural Stem cell intracerebroventricular transplantation in amyotrophic lateral sclerosis patients: a randomized, placebo controlled, triple blind study.
This is an approximate 24-months study (PHASE B) consisting, per patient, of a 30-day screening period, 12-month enrollment and follow up period. A preliminary 3+3 dose-escalation open-label phase (PHASE A) will be performed in order to test the toxicity of the two proposed cell doses. The study will be stopped when all the subjects included in the treatment period complete the study visits. The study uses an ATMP, for that reason all the patients follow up will be prosecuted long life.
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); 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; Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines.
Exclusion: 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; 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; 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.