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NCT Number: NCT04711200

LYell SYndrome MEsenchymal Stromal Cells Treatment

Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) are rare severe cutaneous adverse reactions (SCARs) to drugs.

To date, no curative drug has demonstrated with a good level of evidence its ability to promote SJS and TEN healing and could contribute to earlier reepithelialisation. Mesenchymal stroma cells (MSCs) therapy represents a new therapeutic approach. eg, in patients with cardiovascular diseases, neurological diseases, renal transplantation, lung diseases as acute respiratory distress syndrome.

Recently, MSCs have been proposed in both burn wound healing with a significantly decrease of the unhealed burn area and in cutaneous radiation.

Moreover, MSCs have immunomodulation properties potentially effective in refractory acute and chronic graft versus host disease (GVHD) by improving thymic function and induction of Tregs. Indeed, MSCs are able to migrate to inflamed tissues after stimulation by pro-inflammatory cytokines and to modulate the local inflammatory reactions. MSCs have also demonstrated their ability to promote tissue remodelling, angiogenesis and immunomodulation through either differentiation or secretion of several growth factors such as VEGF, basic FGF and various cytokines.

Therefore, combining their immunomodulation effect and secretion of soluble factors involved in wound repair, MSCs might be valuable as a cell therapy strategy for promoting cutaneous healing in SJS-TEN syndrome and subsequently decrease the morbi-mortality.

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Key information

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients ≥ 18 and ≤ 75 years-old
  • Admission ≤ 10 days after the index date (date of the first symptoms of the disease)
  • Patient with confirmed SJS-TEN diagnosis hospitalized in the department of Dermatology or intensive care medicine
  • 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)
  • 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
  • Affiliated to a social security scheme

Exclusion criteria

  • Pregnant or breastfeeding women
  • History of malignant disease within the past ten years and or presence of metastasis
  • Positive serology for HIV
  • Active infection for hepatitis B or C
  • Detection of Coronavirus SARS CoV-2 RNA on admission (positive RT-PCR), if performed in the usual care
  • Decompensated cardiac failure
  • Uncontrolled epilepsia
  • Previous history of allogenic bone marrow transplantation
  • Participation in other interventional drug research Patient deprived of liberty by a judicial or administrative decision or under the protection of justice
  • Any psychological, familial, sociological or geographical condition potentially hampering compliance with the research protocol and follow-up schedule
  • Patient under tutorship or curatorship
  • Patient under psychiatric care according to art. L1121-6 CSP

Treatment and study plan

Adipose derived stromal cells intravenously injected

Drug

2×10^6/kg of Adipose derived stromal cells A single injection at D0 (performed maximum three days post-admission).

Primary outcomes

  1. Safety : Observation of at least one adverse effect

    Time frame: Day 10

  2. Efficacy : Rate of complete or almost complete reepithelialisation

    Time frame: Day 7 after infusion

Secondary outcomes

  1. Rate of observed and predicted death by the SCORTEN

    Time frame: at one month

  2. Duration of hospitalisation according to our historical cohort related to BSA involved

    Time frame: Month 12

  3. Duration of hospitalisation according to our historical cohort related to onset of the disease

    Time frame: Month 12

  4. Duration of hospitalisation according to our historical cohort related to SCORTEN

    Time frame: Month 12

  5. Duration of each mucous membranes healing ie.(buccal, nasal, genital, eyes)

    Time frame: at Month 12

  6. Rate of sepsis

    Time frame: at Month 12

  7. Rate of intensive care transfer

    Time frame: at Month 12

  8. Rate of sequelae

    Time frame: at Month 12

  9. Th1/Th2 immune response in the peripheral blood of the patients

    Time frame: after injection at Day 0, Day 10, Month 1

  10. Evaluation of expression profile of Th1/Th2 associated chemokines and anti-inflammatory chemokines in the peripheral blood

    Time frame: after injection at Day 0, Day 10, Month 1.

  11. Epidermal chimerism study on healed skin biopsy

    Time frame: at 1 month

  12. Cutaneous re-epithelialization rate at D5, D10 and D15 post-infusion according to the percentage of cutaneous BSA re-epithelialized in comparison to maximal cutaneous detachable-detached BSA observed.

    Time frame: at Day 5, Day 10 and Day15

Study contacts

Contact information is provided by the study sponsor or research team.

Charline Menanteau, MSc

CONTACT

[email protected]

0144841752

Saskia Oro, MD

CONTACT

[email protected]

0149812536 ext. +33

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Registry information

Official study title

Mesenchymal Stromal Cells Treatment in Lyell Syndrome: A Pilot Phase 1-2 Open Trial

Acronym: LYSYME

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Jan 15, 2021
Registry last updated
Aug 1, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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