Amyotrophic Lateral Sclerosis (ALS)
Conditions
Interventions
Product Name: hNSC
Product Code: [hNSC]
Pharmaceutical Form: Solution for injection/infusion
Current Sponsor code: hNSC
Other descriptive name: hNSC
Concentration unit: Other
Concentration type: up to
Sponsors
FONDAZIONE CASA SOLLIEVO DELLA SOFFERENZA OPERA DI SAN PIO DA PIETRELCINA OSPEDALE IRCCS
Eligibility
Sex/Gender
All
Inclusion 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 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 30 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary trial outcome is safety, assessed with respect to the incidence of treatment-emergent AEs (TEAEs) and serious AEs (SAEs) over the whole study period.;Secondary Objective: The secondary aim is to measure the effects of the treatment on the progression of the disease;Primary end point(s): safety (AEs incidence);Timepoint(s) of evaluation of this end point: over the whole study period | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): To compare the change in slopes from the pre-transplantation period to the post transplantation period in FVC (=25% to =100% change for responders) between the treatment and placebo groups through 3 months post first transplantation.; To compare the slope of the rate of decline in the ALSFRS-R at 3 and 6 months following first transplantation relative to the 3-4 months baseline period before transplantation in all patients (both treatment and placebo groups); To compare the slope of the rate of decline in FVC at 3 and 6 months following first transplantation relative to the 3-4 months baseline period before transplantation in all patients (both treatment and placebo groups).; To collect data on the impact of hNSC transplantation on quality of life as measured by ALSAQ-40 scale.; compare the change in slopes in points per month (=0.5 to =2.5 for responders) from the pre-transplantation period to the post transplantation period in ALSFRS-R between the treatment and placebo groups through 3 months post first transplantation;Timepoint(s) of evaluation of this end point: through 3 months post first transplantation; 3 and 6 months following first transplantation; 3 and 6 months following first transplantation; during the conduct of the study; through 3 months post first transplantation | — |
Countries
Italy
Contacts
Public ContactClinical Trial Office
Fondazione IRCCS Casa Sollievo della Sofferenza
Outcome results
None listed