LYell SYndrome MEsenchymal Stromal Cells Treatment (LYSYME)

May 2, 2022 updated by: Assistance Publique - Hôpitaux de Paris

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.

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 Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Patients aged from 18 to 75 years-old
  • Admission less than 10 days after onset of the reaction
  • 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)
  • Written consent from patient or trustworthy person or legal representant or family member
  • 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
  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Adipose derived stromal cells intravenously injected
Drug: Adipose 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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety : Observation of at least one adverse effect
Time Frame: Day 10
Day 10
Efficacy : Rate of complete or almost complete reepithelialisation
Time Frame: Day 7 after infusion
Day 7 after infusion

Secondary Outcome Measures

Outcome Measure
Time Frame
Rate of observed and predicted death by the SCORTEN
Time Frame: at one month
at one month
Duration of hospitalisation according to our historical cohort related to BSA involved
Time Frame: Month 12
Month 12
Duration of hospitalisation according to our historical cohort related to onset of the disease
Time Frame: Month 12
Month 12
Duration of hospitalisation according to our historical cohort related to SCORTEN
Time Frame: Month 12
Month 12
Duration of each mucous membranes healing ie.(buccal, nasal, genital, eyes)
Time Frame: at Month 12
at Month 12
Rate of sepsis
Time Frame: at Month 12
at Month 12
Rate of intensive care transfer
Time Frame: at Month 12
at Month 12
Rate of sequelae
Time Frame: at Month 12
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
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.
after injection at Day 0, Day 10, Month 1.
Epidermal chimerism study on healed skin biopsy
Time Frame: at 1 month
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
at Day 5, Day 10 and Day15

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Investigators

  • Principal Investigator: Saskia Oro, MD, saskia.oro@aphp.fr

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ANTICIPATED)

September 1, 2022

Primary Completion (ANTICIPATED)

September 1, 2025

Study Completion (ANTICIPATED)

September 1, 2025

Study Registration Dates

First Submitted

November 30, 2020

First Submitted That Met QC Criteria

January 12, 2021

First Posted (ACTUAL)

January 15, 2021

Study Record Updates

Last Update Posted (ACTUAL)

May 3, 2022

Last Update Submitted That Met QC Criteria

May 2, 2022

Last Verified

April 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • P150941J

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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