A Phase 1 interventional study of Human Neural Stem Cells in Secondary-progressive Multiple Sclerosis, sponsored by Casa Sollievo della Sofferenza IRCCS. Completed at 3 sites in 2 countries. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2021-07-12.
Sponsored by Casa Sollievo della Sofferenza IRCCS · Phase 1, Interventional, and Treatment
This will be a phase I, open, multicenter, international study performed by 3 participating centres across two countries (Italy and Switzerland). Fifteen to 24 patients affected by SPMS will be enrolled, according to a "standard" phase I design over 18 months. All patients will enter a 3 months run in phase. Thereafter they will receive one of four different doses of allogenic hNSCs (dose A=5 millions hNSCs; dose B=10 millions hNSCs; dose C=16 millions hNSCs; dose D=24 millions hNSCs). Following hNSCs injection, all SPMS patients will receive immunosuppression with tacrolimus for 6 months. Patients will be clinically followed monthly for 1 year and then every 6 months for the 5 years following the study completion (possibly all life long).
MRI assessments will be performed monthly for the first 6 months and then every 3 months for 5 years following the study completion.
Exclusion Criteria:
Intraventricular injections of Allogenic human Neural Stem Cells (hNSCs) in four different dosages (5, 10,16 or 24 millions)
Biological: Human Neural Stem Cells
Allogenic human Neural Stem Cells (hNSCs) in four different dosages (5, 10, 16 or 24 millions). hNSCs are produced by the Laboratorio Cellule Staminali of Terni according to GMP guidelines and are obtained from brain specimens of several fetal human donors from spontaneous miscarriages occurred after the 8th week after conception.
Incidence of Treatment Emergent AE
To Evaluate the Feasibility, Safety and Tolerability of intracerebroventricular injection of allogenic hNSCs
Time frame: 1 year
Percentage of Mortality in treated patients
Percentage of subjects (%) with death due to procedure (mortality correlated to treatment)
Time frame: 1 year
Change in Functional disability
this will be measured by the change of the Expanded Disability Scale (EDSS-disability score about pyramidal, cereberral, brainstem, sensory, bowel and bladder, visual, cerebral Functional Systems) during the study period.
Time frame: Up to 1 year
Change in Functional disability
this will be measured by the change of the the Multiple Sclerosis Functional Composite (MSFC-scores about upper extremity function, ambulation and cognitive function) during the study period.
Time frame: Up to 1 year
Activity of Cognitive function
This will be measured as the mean change of the score of the RAO Brief Repeatable Battery of Neuropsychological Test, during the study period.
Time frame: Up to 1 year
Relapses Rate
Relapses will be measured by the change in EDSS scale
Time frame: Up to 1 year
Relapses Rate
Relapses will be measured by the Imaging evaluations
Time frame: Up to 1 year
MS Biomarkers
Investigation of potential candidate biomarkers able to monitor disease activity and predict clinical course in MS (Neurofilaments)
Time frame: Up to 1 year
Alteration in Neurophysiological parameters
Assessed by Evoked Potentials.
Time frame: Up to 1 year
Plan to share: Yes — All IPD that underlie results in a publication
Supporting information: Csr
This study is completed, as verified in Jul 2021. You cannot join it, but the record below documents what was studied.
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Casa Sollievo della Sofferenza IRCCS