Epidermal NecrolysisPhase 1/2LYSYMENCT04711200
LYell SYndrome MEsenchymal Stromal Cells Treatment
A Phase 1/2 study testing Adipose derived stromal cells intravenously injected in people with Epidermal Necrolysis and Lyell Syndrome, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Epidermal Necrolysis, Lyell Syndrome, Toxic Epidermal Necrolysis
- What is being tested?
- Adipose derived stromal cells intravenously injected
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteFrance
- Study dates
- 15 Apr 2024to Apr 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Mesenchymal Stromal Cells Treatment in Lyell Syndrome: A Pilot Phase 1-2 Open Trial
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.
Read more
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.
- Conditions
- Epidermal Necrolysis, Lyell Syndrome, Toxic Epidermal Necrolysis, Overlap Syndrome, Mesenchymal Stromal Cells, Adipose Derived Stromal Cells
- Being tested
- Adipose derived stromal cells intravenously injected
- Keywords
- Epidermal necrolysis, Lyell syndrome, toxic epidermal necrolysis, Overlap syndrome, Mesenchymal stromal cells, Adipose derived stromal cells
- Medical subject headings
- Stevens-Johnson Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adipose derived stromal cells intravenously injectedDrug
2×10^6/kg of Adipose derived stromal cells A single injection at D0 (performed maximum three days post-admission).
| Study group | Role | What they receive |
|---|---|---|
| Adipose derived stromal cells intravenously injected | Experimental | Not described Receives: Drug: Adipose derived stromal cells intravenously injected |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety : Observation of at least one adverse effect
Measured: Day 10
Efficacy : Rate of complete or almost complete reepithelialisation
Measured: Day 7 after infusion
Secondary outcomes (12)
Rate of observed and predicted death by the SCORTEN
Measured: at one month
Duration of hospitalisation according to our historical cohort related to BSA involved
Measured: Month 12
Duration of hospitalisation according to our historical cohort related to onset of the disease
Measured: Month 12
Duration of hospitalisation according to our historical cohort related to SCORTEN
Measured: Month 12
Duration of each mucous membranes healing ie.(buccal, nasal, genital, eyes)
Measured: at Month 12
Rate of sepsis
Measured: at Month 12
Rate of intensive care transfer
Measured: at Month 12
Rate of sequelae
Measured: at Month 12
Th1/Th2 immune response in the peripheral blood of the patients
Measured: after injection at Day 0, Day 10, Month 1
Evaluation of expression profile of Th1/Th2 associated chemokines and anti-inflammatory chemokines in the peripheral blood
Measured: after injection at Day 0, Day 10, Month 1.
Epidermal chimerism study on healed skin biopsy
Measured: at 1 month
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.
Measured: at Day 5, Day 10 and Day15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2021
- Study start15 Apr 2024
- Primary completionApr 2027 (estimated)
- Study completionApr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Epidermal NecrolysisStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Saskia Oro, PhD, Principal investigator, saskia.oro@aphp.fr
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04711200
- Sponsor's protocol ID
- P150941J
- Other IDs
- 2024-516404-42-00
- Acronym
- LYSYME
- Status last verified
- Jul 2025
- First submitted
- 30 Nov 2020
- Last updated
- 1 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
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 17805350Creamer 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 27317286Roux 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 21367635Chung 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 19780713Duong 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
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mesenchymal Stromal Cells Treatment in Lyell Syndrome: A Pilot Phase 1-2 Open Trial
- ClinicalTrials.gov ID
- NCT04711200
- Acronym / sponsor ID
- LYSYME / P150941J
- Conditions
- 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
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 15 Apr 2024 (actual)
- Primary completion
- Apr 2027 (estimated)
- Study completion
- Apr 2027 (estimated)
- First posted
- 15 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adipose derived stromal cells intravenously injected | Experimental | Drug: Adipose derived stromal cells intravenously injected |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Adipose derived stromal cells intravenously injected2×10^6/kg of Adipose derived stromal cells A single injection at D0 (performed maximum three days post-admission). | Drug | Adipose derived stromal cells intravenously injected |
Primary outcome
Safety : Observation of at least one adverse effect
Time frame: Day 10
Efficacy : Rate of complete or almost complete reepithelialisation
Time frame: Day 7 after infusion
12 secondary outcomes
Rate of observed and predicted death by the SCORTEN
Time frame: at one month
Duration of hospitalisation according to our historical cohort related to BSA involved
Time frame: Month 12
Duration of hospitalisation according to our historical cohort related to onset of the disease
Time frame: Month 12
Duration of hospitalisation according to our historical cohort related to SCORTEN
Time frame: Month 12
Duration of each mucous membranes healing ie.(buccal, nasal, genital, eyes)
Time frame: at Month 12
Rate of sepsis
Time frame: at Month 12
Rate of intensive care transfer
Time frame: at Month 12
Rate of sequelae
Time frame: at Month 12
Th1/Th2 immune response in the peripheral blood of the patients
Time frame: after injection at Day 0, Day 10, Month 1
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.
Epidermal chimerism study on healed skin biopsy
Time frame: at 1 month
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Aug 2025.
- Background: PubMed 17805350Mockenhaupt 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.
- Background: PubMed 27317286Creamer 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.
- Background: PubMed 21367635Roux 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.
- Background: PubMed 19780713Chung 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.
- Results publication: PubMed 28476287Duong 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Saskia Oro, PhD principal investigator
saskia.oro@aphp.fr
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Adipose derived stromal cells intravenously injected in people with Epidermal Necrolysis and Lyell Syndrome, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Saskia Oro, MD (0149812536 ext. +33, saskia.oro@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety : Observation of at least one adverse effect (time frame: Day 10). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04711200, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Aug 2025.