Treatment of Systemic Lupus Erythematosus refractory to standard treatments. MedDRA version: 20.0 Level: PT Classification code 10042945 Term: Systemic lupus erythematosus System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
Conditions
Interventions
Product Name: ALLOGENEIC MESENCHYMAL STROMAL CELLS DERIVED FROM THE UMBILICAL CORD
Product Code: 0
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: 0
CAS Number: 0
Current Sponsor co
Sponsors
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects: 1- Pregnancy, breastfeeding or lack of appropriate contraception during study duration 2- Presence of: a) Renal failure: calculated creatinine clearance of 2 x normal) not related to disease activity. d) Respiratory disease: mean PAP> 50 mmHg (echocardiography), respiratory failure defined by a resting blood pressure of oxygen at PaO 2 50 mmHg without oxygen 3- Severe psychiatric disorders, including severe psychosis related to SLE, which would prevent to give informed consent or to undergo the procedure. 4- Active neoplasia or concomitant myelodysplasia, except for basal cell carcinoma or squamous cell carcinoma or in situ cervix carcinoma. 5- Bone marrow failure defined by neutropenia =65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: Subjects: 1- Pregnancy, breastfeeding or lack of appropriate contraception during study duration 2- Presence of: a) Renal failure: calculated creatinine clearance of 2 x normal) not related to disease activity. d) Respiratory disease: mean PAP> 50 mmHg (echocardiography), respiratory failure defined by a resting blood pressure of oxygen at PaO 2 50 mmHg without oxygen 3- Severe psychiatric disorders, including severe psychosis related to SLE, which would prevent to give informed consent or to undergo the procedure. 4- Active neoplasia or concomitant myelodysplasia, except for basal cell carcinoma or squamous cell carcinoma or in situ cervix carcinoma. 5- Bone marrow failure defined by neutropenia <0.5.109/L, thrombocytopenia <30. 109 / L, anemia < 8 g / dL, lymphopenia CD4 + <200 x 106 / L caused by another disease than SLE. 6- Acute or chronic uncontrolled infection: HIV 1/2, HTLV-1/2, Hepatitis B (HBsAg surface antigen), Hepatitis C with positive PCR 7- Patient having received belimumab within 2 months of belimumab within 2 months of Baseline, or having received rituximab or other B cell depleting biologic therapy within 6 months of Baseline 8- Comorbidities requiring corticosteroid therapy 9- Current substance abuse or recent (within 60 days) history of substance abuse 10- Patient in periods of exclusion from the national roster of researchers 11- Patient with Linguistic or psychological incapacity to sign informed consent 12- Patient who has already been included in this or another study at the same time. 13- Poor patient compliance. 14- Patient under legal protection.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To analyze the tolerance of the administration of allogeneic CSM in the treatment of systemic Lupus Erythematous System (SLE) refractory to conventional treatments (Cyclophosphamide (CY), MMF or corticosteroids with or without corticosteroids);Secondary Objective: 1- Feasibility of the administration of allogeneic CSM in the treatment of refractory patients with refractory severe prior conventional treatments 2- Tolerance at more than three months after injection, taking into account the morbidity observed and the overall survival of patients with SLE treated up to 1 year after the procedure (with 1 year of follow-up after the end of the study ). 3- Analysis of clinical and biological response according to clinico-biological criteria for routine surveillance of lupus by functional scores (SELENA-SLEDAI, BILAG, SF36, EQ5D, SRI, SLICC-SLE) 4- Analysis of the efficacy at 3 months of injection of allogeneic MSCs by evaluating the proportion of patients presenting a Major Clinical Response (MCR). 5- Evaluate the percentage of patients with Partial Clinical Response (CPR) during the 12-month follow-up period of the study. 6- Evaluation of the immunomodulatory action;Primary end point(s): Immediate tolerance assessed during the injection and the first 10 days after the injection of the allogeneic MSCs, according to the side effects defined by the CTCAE standards.;Timepoint(s) of evaluation of this end point: No | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: No;Secondary end point(s): 1-Feasibility of administering allogeneic MSCs in the treatment of refractory patients with refractory severe prior conventional treatments 2-Tolerance at more than three months after injection, taking into account the morbidity observed and the overall survival of SLE patients treated up to 1 year after the procedure (with 1 year of follow-up after the end of the study ). 3-Analysis of clinical and biological response according to clinico-biological criteria for routine lupus surveillance by functional scores (SELENA-SLEDAI, BILAG, SF36, EQ5D, SRI, SLICC-SLE) 4-Analysis of the efficacy at 3 months of injection of allogeneic MSCs by evaluating the proportion of patients presenting a Major Clinical Response (MCR). 5-Evaluate the percentage of patients presenting a Partial Clinical Response (CPR) during the 12 months of study follow-up. 6-Evaluation of immunomodulatory action | — |
Countries
France
Contacts
Public ContactDRCI Hôpital St Louis
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
Outcome results
None listed