In this study, we propose, for the first time, to test the safety and the potential efficacy of repeated allogenic Adipose tissue-derived Mesenchymal Stromal Cells IT injections in Primary Progressive Multiple Sclerosis patients.
Patient with Primary Progressive MS according to the criteria of Mc Donald 2017 (Thompson et al Lancet neurol, 2017)
Age between 18 and 55 years
EDSS score: 3 to 6 at inclusion
Documented evidence of disability progression independent of relapse activity at any point in time over the 2 years prior to the screening visit
Inflammatory activity during the past year (relapses or new T2 MRI lesions)
Disease Modifying Drugs during the past year
Treatment with high dose corticosteroids during the 30 days preceding the inclusion
Contra indication to lumbar puncture/intrathecal infusion: intracranial hypertension, puncture site infections, severe thrombocytopenia (<50 G/L), anticoagulant or fibrinolytic treatment
Single group
Adipose tissue-derived Mesenchymal Stromal Cells
Drugrepeated allogenic ASCs IT injections
Percentage of patients experiencing at least one adverse effect
Percentage of patients experiencing at least one adverse effect (AE) due to ASC infusion qualified as CTCAE V5 grade ≥ 3 or qualified as "serious" AE using the MeDRA terminology, and occurring between the first infusion and the end of the follow-up. The severity of the AE and the causality of the ASC infusion will be validated by a DSMB
Description of Adverse events related to experimental product
Number and description of AE (related to the experimental product) using MedDRA terminology and CTCAE V5 at each time point during the study
Clinical disease evolution at Week24 and Week48 after treatment : Confirmed Disability Progression using Expanded Disability Status Scale score
Percentage of patients with 3-months Confirmed Disability Progression (CDP) between W0 and W24-W48. Disability progression will be defined as an increase of 1pt Expanded Disability Status Scale (EDSS). The Expanded Disability Status Scale (EDSS) is a method of quantifying disability in multiple sclerosis and monitoring changes in the level of disability over time. It is widely used in clinical trials and in the assessment of people with MS. (Disability progression will be defined as an increase of 1.5 pt if baseline EDSS=0, 1pt EDSS (if baseline 1 ≤ EDSS\&lt;6), or an increase of 0.5pt if baseline EDSS is ≥ 6; confirmed at 6 months.) EDSS : Minimum Score 1, Maximum score 10, higher scores mean a worse outcome.
Clinical disease evolution at Week24 and Week48 after treatment : Expanded Disability Status Scale score
Mean change in EDSS score between W0 and W24-W48. Expanded Disability Status Scale (EDSS). The Expanded Disability Status Scale (EDSS) is a method of quantifying disability in multiple sclerosis and monitoring changes in the level of disability over time. It is widely used in clinical trials and in the assessment of people with MS.(Disability progression will be defined as an increase of 1.5 pt if baseline EDSS=0, 1pt EDSS (if baseline 1 ≤ EDSS\&lt;6), or an increase of 0.5pt if baseline EDSS is ≥ 6; confirmed at 6 months.) EDSS : Minimum Score 1, Maximum score 10, higher scores mean a worse outcome.
Clinical disease evolution at Week24 and Week48 after treatment : Expanded Disability Status Scale score
Percentage of patients with No Evidence of Progression (NEP): Evidence of Progression is defined by (1) EDSS progression confirmed at 3 months, The Expanded Disability Status Scale (EDSS) is a method of quantifying disability in multiple sclerosis and monitoring changes in the level of disability over time. It is widely used in clinical trials and in the assessment of people with MS. EDSS : Minimum Score 1, Maximum score 10, higher scores mean a worse outcome.
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Rennes University Hospital
Lead sponsor
Etablissement Français du Sang
Collaborator
