Safety Study of Human Neural Stem Cells Injections for Secondary Progressive Multiple Sclerosis Patients (NSC-SPMS)
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|ClinicalTrials.gov Identifier: NCT03282760|
Recruitment Status : Unknown
Verified January 2019 by Casa Sollievo della Sofferenza IRCCS.
Recruitment status was: Active, not recruiting
First Posted : September 14, 2017
Last Update Posted : January 14, 2019
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.
|Condition or disease||Intervention/treatment||Phase|
|Secondary-progressive Multiple Sclerosis||Biological: Human Neural Stem Cells||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Sequential Assignment|
|Intervention Model Description:||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 millionshNSCs; dose C=16 millions hNSCs; dose D=24 millions hNSCs).|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Multicenter Study of Allogenic, Intracerebroventricular Human Neural Stem Cells Transplantation for the Experimental Treatment of Secondary Progressive Multiple Sclerosis Patients|
|Actual Study Start Date :||September 9, 2017|
|Estimated Primary Completion Date :||April 30, 2020|
|Estimated Study Completion Date :||February 28, 2021|
Experimental: Human Neural Stem Cells Suspension
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 [ Time Frame: 1 year ]To Evaluate the Feasibility, Safety and Tolerability of intracerebroventricular injection of allogenic hNSCs
- Percentage of Mortality in treated patients [ Time Frame: 1 year ]Percentage of subjects (%) with death due to procedure (mortality correlated to treatment)
- Change in Functional disability [ Time Frame: Up to 1 year ]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.
- Change in Functional disability [ Time Frame: Up to 1 year ]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.
- Activity of Cognitive function [ Time Frame: Up to 1 year ]This will be measured as the mean change of the score of the RAO Brief Repeatable Battery of Neuropsychological Test, during the study period.
- Relapses Rate [ Time Frame: Up to 1 year ]Relapses will be measured by the change in EDSS scale
- Relapses Rate [ Time Frame: Up to 1 year ]Relapses will be measured by the Imaging evaluations
- MS Biomarkers [ Time Frame: Up to 1 year ]Investigation of potential candidate biomarkers able to monitor disease activity and predict clinical course in MS (Neurofilaments)
- Alteration in Neurophysiological parameters [ Time Frame: Up to 1 year ]Assessed by Evoked Potentials.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03282760
|Casa Sollievo della Sofferenza - IRCCS|
|San Giovanni Rotondo, Foggia, Italy, 71013|
|Azienda Ospedaliera Santa Maria di Terni|
|Terni, Italy, 05100|
|Neurocentro della Svizzera Italiana, Istituto di Neurosienze cliniche della svizzera italiana, Centro sclerosi Multipla|
|Lugano, Switzerland, 6900|
|Study Director:||Angelo L Vescovi, PhD||Casa Sollievo della Sofferenza IRCCS|