ALLOGENEIC ADIPOSE-DERIVED MESENCHYMAL STROMAL CELLS, EX-VIVO EXPANDED for Stevens-Johnson syndrome / Toxic epidermal necrolysis (SJS-TEN) | Lyell syndrome

Inclusion criteria

• {"criterion_text":"- For patients : Patient ≥18 and ≤ 75 years-old"}
• {"criterion_text":"- For donors : Written consent"}
• {"criterion_text":"- For donors : Affiliated to a social security scheme"}
• {"criterion_text":"- For patients : Admission ≤ 10 days after the index date (date of the first symptoms of the disease)"}
• {"criterion_text":"- For patients : Patient with confirmed SJS-TEN diagnosis hospitalized in the department of Dermatology or intensive care medicine"}
• {"criterion_text":"- For patients : At least 10 % of detachable-detached body surface area at any time during the first 10 days after the index date (date of the first symptoms of the disease)"}
• {"criterion_text":"- For patients : Who, after the nature of the study has been explained to them or a support person (if applicable), and prior to any protocol specific procedures being performed, have given written consent according to local regulatory requirements"}
• {"criterion_text":"- For patients : Affiliated to a social security scheme"}
• {"criterion_text":"- For donors : Donor ≥ 18 and ≤ 55 years old"}
• {"criterion_text":"- For donors : Admission for a programmed plastic surgery of liposuction or aspiration in the abdominal wall under general anesthesia"}
• {"criterion_text":"- For donors : Health history questionnaire in appendix completed"}

Exclusion criteria

• {"criterion_text":"- For patients : Pregnant or breastfeeding woman"}
• {"criterion_text":"- For donors : Treatment with extractive pituitary hormones (including growth hormones)"}
• {"criterion_text":"- For donors : Human dura mater transplant"}
• {"criterion_text":"- For patients : History of malignant disease within the past ten years and or presence of metastasis"}
• {"criterion_text":"- For donors : Surgical history of the central nervous system"}
• {"criterion_text":"- For donors : Dementia or neurological disease that may evoke subacute spongiform encephalopathy"}
• {"criterion_text":"- For donors : Family history as part of subacute spongiform encephalopathy"}
• {"criterion_text":"- For donors : Hematological malignancies"}
• {"criterion_text":"- For donors : Active or cured cancer"}
• {"criterion_text":"- For donors : System or connective tissue disease"}
• {"criterion_text":"- For donors : Active generalized infection (bacterial, viral, parasitic, tuberculosis, leprosy...)"}
• {"criterion_text":"- For patients : Patient deprived of liberty by a judicial or administrative decision or under the protection of justice"}
• {"criterion_text":"- For donors : Multiple adenopathy, splenomegaly, hepatomegaly at clinical examination"}
• {"criterion_text":"- For donors : Icterus"}
• {"criterion_text":"- For donors : Chemotherapy history, irradiation"}
• {"criterion_text":"- For patients : Positive serology for HIV"}
• {"criterion_text":"- For donors : Long-term systemic steroids (duration > 30 days within the 5 past years or within 1 month before inclusion, at any dose)"}
• {"criterion_text":"- For patients : Active infection for hepatitis B or C"}
• {"criterion_text":"- For patients : Detection of Coronavirus SARS CoV-2 RNA on admission (positive RTPCR), if performed in the usual care"}
• {"criterion_text":"- For patients : Decompensated cardiac failure"}
• {"criterion_text":"- For patients : Uncontrolled epilepsia"}
• {"criterion_text":"- For patients : Previous history or allogenic bone marrow transplantation"}
• {"criterion_text":"- For patients : Any psychological, familial, sociological or geographical condition potentially hampering compliance with the research protocol and followup schedule"}
• {"criterion_text":"- For patients : Participation in other interventional drug research"}
• {"criterion_text":"- For donors : Authenticated risk factors for viral infections in the past 12 months : o Multiple sexual partners (donor or regular partner) o Intravenous addiction (donor or regular partner) o Accident of exposure to blood or derivatives suspected of being contaminated"}
• {"criterion_text":"- For donors : Topical steroids on abdominal wall within 1 month before inclusion"}
• {"criterion_text":"- For donors : Contraindication to general anesthesia"}
• {"criterion_text":"- For donors : Body mass index <18.5 or >30 kg/m2"}
• {"criterion_text":"- For donors : Active smoking"}
• {"criterion_text":"- For donors : Unbalanced diabetes or treated by insuline"}
• {"criterion_text":"- For donors : Donors who present a risk according to current health alerts and recommendations from ANSM, ABM and SECPROCH at the time of inclusion"}
• {"criterion_text":"- For donors : In addition, any donor patient presenting an exclusion criterion or any element considered to be clinically significant in appendix will not be included"}
• {"criterion_text":"- For patients : Patient under tutorship or curatorship"}
• {"criterion_text":"- For patients : Patient under psychiatric care according to art. L1121-6 CSP"}
• {"criterion_text":"- For donors : Positive viral serology: HIV, HBV (An anti HBc antibody isolated without detection of viral genome (HBV NAT) and HBsAg can be considered as acceptable), HCV, HEV, syphilis, HTLV, active infection with IgM+ for toxoplasmosis, EBV, CMV"}
• {"criterion_text":"- For donors : Detection of Coronavirus SARS CoV-2 RNA on screening (positive RTPCR), if performed in the usual care"}
• {"criterion_text":"- For donors : Deprived of freedom"}
• {"criterion_text":"- For donors : Significant comorbidities according to donor health history, if considered to be clinically significant by the investigator"}

Primary endpoints

• {"endpoint_text":"- Safety: The toxicity is defined as the observation of at least one adverse effect","definition_or_measurement_approach":"Toxicity defined as the observation of at least one adverse effect (as stated in the protocol)"}
• {"endpoint_text":"- Efficacy: Rate of complete almost complete reepithelialisation at D7 after infusion. This criterion is defined as at least 90% of cutaneous body surface area (BSA) healed at D7 in comparison to maximal cutaneous detachable-detached BSA observed.","definition_or_measurement_approach":"Re-epithelialisation measured as at least 90% of cutaneous BSA healed at Day 7 compared with maximal detachable-detached BSA observed"}

Secondary endpoints

• {"endpoint_text":"- Rate of observed and predicted death at one month by the SCORTEN scale","definition_or_measurement_approach":"Comparison of observed and predicted 1-month mortality using the SCORTEN scale"}
• {"endpoint_text":"- Duration of hospitalisation according to our historical cohort","definition_or_measurement_approach":"Duration of hospital stay compared with historical cohort data"}
• {"endpoint_text":"- Duration of each mucous membrane healing i.e. (buccal, nasal, genital, eyes).","definition_or_measurement_approach":"Time to healing for each mucous membrane site (buccal, nasal, genital, ocular)"}
• {"endpoint_text":"- Rate of sepsis","definition_or_measurement_approach":"Incidence rate of sepsis events during follow-up"}
• {"endpoint_text":"- Rate of intensive care transfer","definition_or_measurement_approach":"Incidence of transfer to intensive care"}
• {"endpoint_text":"- Rate of sequelae at M12","definition_or_measurement_approach":"Proportion of patients with sequelae at month 12"}
• {"endpoint_text":"- Th1/Th2 immune response in the peripheral blood of the patients after injection at D0, D10, M1","definition_or_measurement_approach":"Assessment of Th1/Th2 immune response in peripheral blood at Day 0, Day 10 and Month 1"}
• {"endpoint_text":"- Evaluation of expression profile of Th1/Th2 associated chemokines and anti-inflammatory chemokines in the peripheral blood after injection at D0, D10, M1.","definition_or_measurement_approach":"Expression profiling of Th1/Th2-associated and anti-inflammatory chemokines in peripheral blood at Day 0, Day 10 and Month 1"}
• {"endpoint_text":"- Epidermal chimerism research on healed skin biopsy and peripheral blood at 1 month.","definition_or_measurement_approach":"Assessment of epidermal chimerism on healed skin biopsy and peripheral blood at 1 month"}
• {"endpoint_text":"- Rate of complete or almost complete reepithelialisation at D5, D10 and D15 after infusion.","definition_or_measurement_approach":"Proportion of patients with complete or near-complete re-epithelialisation at Days 5, 10 and 15 post-infusion"}

France

Earliest CTIS Part Ii Submission Date

28-10-2024

Latest Decision Or Authorization Date

19-06-2025

Processing Time Days

234

Number Of Sites

4

Number Of Participants

30

Primary sponsor

Full Name

Assistance Publique Hopitaux De Paris

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France