Tests neural stem cell treatment in people with amyotrophic lateral sclerosis
Tests injected human neural stem cells against saline placebo in adults with ALS.
Key facts
- Who runs it
- Casa Sollievo della Sofferenza IRCCS
- Where
- Novara, Padua, Palermo, and San Giovanni Rotondo, ItalySee the 4 sites
- Trial code
- Registry title
- Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis (STEMALS)
- Status
- Recruiting
- Phase
- Phase 2
- People taking part
- 30 people, planned
- Started
- 25 Jan 2024
- Expected to end
- Sept 2027
- Type of study
- Interventional, 3 groupsOne group gets a placebo (a dummy treatment), for comparison.
- Treatments tested
- human Neural Stem Cells (hNSC)
- Saline (Placebo)
- Record updated
- 3 April 2024
Who can take part
AI summary
From the trial's eligibility criteria. 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 aged 18 to 65 with definite or probable ALS whose disease is progressing fast and who can safely take a short course of immune-suppressing medicine.
- Age
- 18 to 65 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 above 70%. ALSFRS-R score of at least 26. Fast progression, with 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. Cannot have had stem cell or somatic cell therapy before.
Main reasons for not taking part
- Psychiatric disease or other neurological disease besides ALS
- Cancer within the previous 10 years
- Immunosuppressive therapy within 12 weeks of screening, or active autoimmune disease or infection (including hepatitis B, hepatitis C or HIV)
- Cognitive impairment
- Pregnancy or breast feeding
The registry criteria, word for word
Show all 20 criteriaShow less
Who can take part
- 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
Who cannot take part
- 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
Check if you might fit
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?
Where in Italy
- Centro SLA Azienda Ospedaliera Università Maggiore della CaritàNovara, ItalyContact: Letizia Mazzini, MDTaking people
- Azienda Ospedaliera di PadovaPadua, ItalyContact: Gianni Sorarù, MDNot yet recruiting
- Azienza Ospedaliera Universitaria - Policlinico "P. Giaccone" Università degli Studi di PalermoPalermo, ItalyContact: Vincenzo La Bella, MDNot yet recruiting
- Casa Sollievo Della Sofferenza IRCCSSan Giovanni Rotondo, ItalyContact: Massimo Carella, PhDNot yet recruiting
About the trial
What the trial wants to find out
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
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.
The 3 groups in the trial
- hNSC 20*10^6Treatment being tested
Inoculation of 20\*10\^6 neural stem cells into the right lateral ventricle through a silicone catheter
Treatment: human Neural Stem Cells (hNSC)
- hNSC 40*10^6Treatment being tested
Inoculation of 40\*10\^6 neural stem cells into the right lateral ventricle through a silicone catheter
Treatment: human Neural Stem Cells (hNSC)
- Saline Solution (placebo)Placebo, for comparison
Inoculation of saline (placebo) into the right lateral ventricle through a silicone catheter The reservoir will be placed to all recruited patients in a first surgical session, after randomization half of the subjects will receive the drug product (further randomized for doses) and half the placebo. After three months (period of time considered the minimum for a correct clinical evaluation that does not risk having to exclude patients treated with placebo from subsequent treatment with cells due to excessive physical decline), patients that already received cells will also be infused with placebo while the others will be randomized and transplanted with one of the two cell doses previously mentioned
Treatment: Saline (Placebo)
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?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis: A Multicenter, Randomized Placebo Controlled and Biological Endpoints Clinical Trial
- Plain-language title
- Tests neural stem cell treatment in people with amyotrophic lateral sclerosisGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Fit questions
- Built from NCT06344260, who can take part
- Record updated
- 3 April 2024