CClinicalTrials.gg
Sign In
RecruitingNCT04711200LYSYMEUpdated Aug 1, 2025

LYell SYndrome MEsenchymal Stromal Cells Treatment

A Phase 1/2 interventional study of Adipose derived stromal cells intravenously injected in Epidermal Necrolysis, Lyell Syndrome and Toxic Epidermal Necrolysis, sponsored by Assistance Publique - Hôpitaux de Paris. Recruiting at 1 site in France. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2025-08-01.

Sponsored by Assistance Publique - Hôpitaux de Paris · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
15
Allocation
Not applicable
Ages
18 Years to 75 Years
Sex
All
01

Study summary

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.

02

Conditions studied

  • Epidermal Necrolysis
  • Lyell Syndrome
  • Toxic Epidermal Necrolysis
  • Overlap Syndrome
  • Mesenchymal Stromal Cells
  • Adipose Derived Stromal Cells

Keywords

  • Epidermal necrolysis
  • Lyell syndrome
  • toxic epidermal necrolysis
  • Overlap syndrome
  • Mesenchymal stromal cells
  • Adipose derived stromal cells
03

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
15 participants (estimated)

Study arms

  • Experimental
    Adipose derived stromal cells intravenously injected

    Drug: Adipose derived stromal cells intravenously injected

Interventions

  • DrugAdipose derived stromal cells intravenously injected

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

05

What researchers measure

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

06

Study locations

1 of 1 sites recruiting
  • Henri Mondor
    Créteil, France
    Recruiting
07

References and documents

Publications

  • Mockenhaupt M, Viboud C, Dunant A, Naldi L, Halevy S, Bouwes Bavinck JN, Sidoroff A, Schneck J, Roujeau JC, Flahault A. Stevens-Johnson syndrome and toxic epidermal necrolysis: assessment of medication risks with emphasis on recently marketed drugs. The EuroSCAR-study. J Invest Dermatol. 2008 Jan;128(1):35-44. doi: 10.1038/sj.jid.5701033. Epub 2007 Sep 6. PubMed 17805350 ↗
  • Creamer D, Walsh SA, Dziewulski P, Exton LS, Lee HY, Dart JK, Setterfield J, Bunker CB, Ardern-Jones MR, Watson KM, Wong GA, Philippidou M, Vercueil A, Martin RV, Williams G, Shah M, Brown D, Williams P, Mohd Mustapa MF, Smith CH. U.K. guidelines for the management of Stevens-Johnson syndrome/toxic epidermal necrolysis in adults 2016. Br J Dermatol. 2016 Jun;174(6):1194-227. doi: 10.1111/bjd.14530. No abstract available. PubMed 27317286 ↗
  • Roux S, Leotot J, Chevallier N, Bierling P, Rouard H. [Mesenchymal stromal cells: Biological properties and clinical prospects]. Transfus Clin Biol. 2011 Feb;18(1):1-12. doi: 10.1016/j.tracli.2011.01.001. Epub 2011 Mar 1. French. PubMed 21367635 ↗
  • Chung HM, Won CH, Sung JH. Responses of adipose-derived stem cells during hypoxia: enhanced skin-regenerative potential. Expert Opin Biol Ther. 2009 Dec;9(12):1499-508. doi: 10.1517/14712590903307362. PubMed 19780713 ↗
  • Duong TA, Valeyrie-Allanore L, Wolkenstein P, Chosidow O. Severe cutaneous adverse reactions to drugs. Lancet. 2017 Oct 28;390(10106):1996-2011. doi: 10.1016/S0140-6736(16)30378-6. Epub 2017 May 2. PubMed 28476287 ↗
08

Registry details

Key details

Study ID
NCT04711200
Lead sponsor
Assistance Publique - Hôpitaux de Paris
Responsible party
Sponsor
First posted
Jan 15, 2021
Start date
Apr 15, 2024
Primary completion
Apr 2027 (estimated)
Completion
Apr 2027 (estimated)
Last update
Aug 1, 2025

Study contacts

Saskia Oro, MD
Contact
saskia.oro@aphp.fr
0149812536 ext. +33
Charline Menanteau, MSc
Contact
myriem.carrier@aphp.fr
0144841752
Saskia Oro, PhD
principal investigator · saskia.oro@aphp.fr

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Interested in this study?

Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.

Contact study team

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Stevens-Johnson Syndrome→

Other studies from this sponsor

Assistance Publique - Hôpitaux de Paris

Start the discussion