Neural stem cell treatment for ALS

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

This study is testing whether a new treatment using human neural stem cells can help people with ALS feel better and stay safe compared to a placebo.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Casa Sollievo della Sofferenza IRCCS (other)
• Locations: 4 sites (San Giovanni Rotondo, Foggia and 3 other locations)
• Trial ID: NCT06344260 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of human neural stem cell transplantation in patients with amyotrophic lateral sclerosis (ALS). It consists of a 30-day screening period followed by a 12-month enrollment and follow-up phase. The study includes a dose-escalation phase to determine the toxicity of two different doses of stem cells, administered via an Ommaya reservoir. Patients will be randomized to receive either the stem cells or a placebo, with ongoing safety monitoring by an independent board.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-65 with a confirmed diagnosis of ALS, showing fast disease progression and a specific range of functional scores.

Not a fit: Patients with slow-progressing ALS or those outside the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could potentially slow disease progression and improve quality of life for ALS patients.

How similar studies have performed: While there have been studies on stem cell therapies for ALS, this specific approach using human neural stem cells is relatively novel.

Eligibility criteria

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.

Where this trial is running

San Giovanni Rotondo, Foggia and 3 other locations

• Casa Sollievo Della Sofferenza IRCCS — San Giovanni Rotondo, Foggia, Italy (NOT_YET_RECRUITING)
• Centro SLA Azienda Ospedaliera Università Maggiore della Carità — Novara, Italy (RECRUITING)
• Azienda Ospedaliera di Padova — Padua, Italy (NOT_YET_RECRUITING)
• Azienza Ospedaliera Universitaria - Policlinico "P. Giaccone" Università degli Studi di Palermo — Palermo, Italy (NOT_YET_RECRUITING)

Study contacts

• Study coordinator: Massimo Carella, PhD

Conditions: Amyotrophic Lateral Sclerosis, Neural Stem Cells, ALS, Biomarkers, ICV Surgery