Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis (STEMALS)

ConditionAmyotrophic Lateral Sclerosis

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18-65

SponsorCasa Sollievo della Sofferenza IRCCS

About this trial

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.

Eligibility criteria

Qualifiers

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%;

Disqualifiers

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);

Trial design

Treatments tested in this trial

human Neural Stem Cells (hNSC)
Saline (Placebo)

Treatment groups

30 Participants

are divided into 3 treatment groups

Sponsors and collaborators

Casa Sollievo della Sofferenza IRCCS

Lead sponsor

Ministry of Health, Italy

Collaborator

Source ClinicalTrials.gov