Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis: A Multicenter, Randomized Placebo Controlled and Biological Endpoints Clinical Trial

Trial ID: NCT06344260

Interventional - Phase 2 - Recruiting

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

• Amyotrophic Lateral Sclerosis

Interventions

• human Neural Stem Cells (hNSC)
• Saline (Placebo)

Eligibility

Inclusion criteria

• 1. Patient provides written informed consent, informed consent signature collection prior to any study procedure (patient has good acceptance and understanding of the informed consent);
• 2. Definite, probable diagnosis according to the revised El Escorial criteria;
• 3. Age: 18-65 years;
• 4. FVC \>70%;
• 5. Onset ≤ 24 months;
• 6. 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;
• 7. 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;
• 8. 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;
• 9. Patient is medically able to tolerate transient immunosuppression regimen;
• 10. Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines.

Exclusion criteria

• 1. Psychiatric disease or other neurological diseases different from ALS;
• 2. 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;
• 3. Cancer within the previous 10 years;
• 4. Immunosuppressive therapy within 12 weeks of screening; active autoimmune disease or infection (including hepatitis B, hepatitis C, or HIV);
• 5. Cognitive impairment;
• 6. Contraindications to perform MRI scans, CSF withdrawal and Skin biopsy;
• 7. Patient unable to understand informed consent form;
• 8. Pregnancy and breast feeding;
• 9. Patient has been treated previously with any stem cell or somatic cells therapy;
• 10. 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.

Sponsor

Casa Sollievo della Sofferenza IRCCS, Ministry of Health, Italy

Source: ClinicalTrials.gov

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