Actively Recruiting
Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis (STEMALS)
Led by Casa Sollievo della Sofferenza IRCCS · Updated on 2024-04-03
30
Participants Needed
4
Research Sites
187 weeks
Total Duration
On this page
Sponsors
C
Casa Sollievo della Sofferenza IRCCS
Lead Sponsor
M
Ministry of Health, Italy
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis (STEMALS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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.
You will not qualify if you...
- Psychiatric disease or other neurological diseases different from ALS;
- 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;
- 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;
- 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.
AI-Screening
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Trial Site Locations
Total: 4 locations
1
Casa Sollievo Della Sofferenza IRCCS
San Giovanni Rotondo, Foggia, Italy, 71013
Not Yet Recruiting
2
Centro SLA Azienda Ospedaliera Università Maggiore della Carità
Novara, Italy, 28100
Actively Recruiting
3
Azienda Ospedaliera di Padova
Padua, Italy, 35128
Not Yet Recruiting
4
Azienza Ospedaliera Universitaria - Policlinico "P. Giaccone" Università degli Studi di Palermo
Palermo, Italy, 90129
Not Yet Recruiting
Research Team
M
Massimo Carella, PhD
CONTACT
C
CTO Clinical Trial Office
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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