2024-516404-42-00招募中2 期
Mesenchymal stromal cells treatment in Lyell syndrome: A pilot phase 1-2 open trial (LYSYME)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Safety: The toxicity is defined as the observation of at least one adverse effect
研究概览
简要总结
To evaluate the safety and the efficacy i.e., complete almost complete cutaneous re-epithelialization at D7 after infusion of 2×106/kg ASCs in SJS-TEN patients.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •For patients : Patient ≥18 and ≤ 75 years-old
- •For donors : Written consent
- •For donors : Affiliated to a social security scheme
- •For patients : Admission ≤ 10 days after the index date (date of the first symptoms of the disease)
- •For patients : Patient with confirmed SJS-TEN diagnosis hospitalized in the department of Dermatology or intensive care medicine
- •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)
- •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
- •For patients : Affiliated to a social security scheme
- •For donors : Donor ≥ 18 and ≤ 55 years old
- •For donors : Admission for a programmed plastic surgery of liposuction or aspiration in the abdominal wall under general anesthesia
- •For donors : Health history questionnaire in appendix completed
排除标准
- •For patients : Pregnant or breastfeeding woman
- •For donors : Treatment with extractive pituitary hormones (including growth hormones)
- •For donors : Human dura mater transplant
- •For patients : History of malignant disease within the past ten years and or presence of metastasis
- •For donors : Surgical history of the central nervous system
- •For donors : Dementia or neurological disease that may evoke subacute spongiform encephalopathy
- •For donors : Family history as part of subacute spongiform encephalopathy
- •For donors : Hematological malignancies
- •For donors : Active or cured cancer
- •For donors : System or connective tissue disease
- •For donors : Active generalized infection (bacterial, viral, parasitic, tuberculosis, leprosy...)
- •For patients : Patient deprived of liberty by a judicial or administrative decision or under the protection of justice
- •For donors : Multiple adenopathy, splenomegaly, hepatomegaly at clinical examination
- •For donors : Icterus
- •For donors : Chemotherapy history, irradiation
- •For patients : Positive serology for HIV
- •For donors : Long-term systemic steroids (duration > 30 days within the 5 past years or within 1 month before inclusion, at any dose)
- •For patients : Active infection for hepatitis B or C
- •For patients : Detection of Coronavirus SARS CoV-2 RNA on admission (positive RTPCR), if performed in the usual care
- •For patients : Decompensated cardiac failure
- •For patients : Uncontrolled epilepsia
- •For patients : Previous history or allogenic bone marrow transplantation
- •For patients : Any psychological, familial, sociological or geographical condition potentially hampering compliance with the research protocol and followup schedule
- •For patients : Participation in other interventional drug research
- •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
- •For donors : Topical steroids on abdominal wall within 1 month before inclusion
- •For donors : Contraindication to general anesthesia
- •For donors : Body mass index <18.5 or >30 kg/m2
- •For donors : Active smoking
- •For donors : Unbalanced diabetes or treated by insuline
- •For donors : Donors who present a risk according to current health alerts and recommendations from ANSM, ABM and SECPROCH at the time of inclusion
- •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
- •For patients : Patient under tutorship or curatorship
- •For patients : Patient under psychiatric care according to art. L1121-6 CSP
- •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
- •For donors : Detection of Coronavirus SARS CoV-2 RNA on screening (positive RTPCR), if performed in the usual care
- •For donors : Deprived of freedom
- •For donors : Significant comorbidities according to donor health history, if considered to be clinically significant by the investigator
结局指标
主要结局
Safety: The toxicity is defined as the observation of at least one adverse effect
Safety: The toxicity is defined as the observation of at least one adverse effect
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.
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.
次要结局
- Rate of observed and predicted death at one month by the SCORTEN scale
- Duration of hospitalisation according to our historical cohort
- Duration of each mucous membrane healing i.e. (buccal, nasal, genital, eyes).
- Rate of sepsis
- Rate of intensive care transfer
- Rate of sequelae at M12
- Th1/Th2 immune response in the peripheral blood of the patients after injection at D0, D10, M1
- Evaluation of expression profile of Th1/Th2 associated chemokines and anti-inflammatory chemokines in the peripheral blood after injection at D0, D10, M1.
- Epidermal chimerism research on healed skin biopsy and peripheral blood at 1 month.
- Rate of complete or almost complete reepithelialisation at D5, D10 and D15 after infusion.
研究者
Dr Saskia ORO
Scientific
Assistance Publique Hopitaux De Paris
研究点 (1)
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