Neural stem cell treatment for ALS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Centro SLA Azienda Ospedaliera Università Maggiore della Carità
  Novara, Italy Recruiting
• Casa Sollievo Della Sofferenza IRCCS
  San Giovanni Rotondo, Foggia, Italy Not yet 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
• Email: m.carella@operapadrepio.it
• Phone: +390882835928

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.