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    Summary
    EudraCT Number:2020-000308-12
    Sponsor's Protocol Code Number:P150941J
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2020-05-28
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2020-000308-12
    A.3Full title of the trial
    « Mesenchymal stromal cells treatment in Lyell syndrome: A pilot phase 1-2 open trial”
    Cellules stromales mésenchymateuses dans le traitement du syndrome du Lyell : une étude de phase pilote I-II en ouvert
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    NA
    A.3.2Name or abbreviated title of the trial where available
    LYSYME
    A.4.1Sponsor's protocol code numberP150941J
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorASSISTANCE PUBLIQUE-HOPITAUX DE PARIS
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAP-HP_PHRC N_2015
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameexpanded allogeneic adipose derived stromal cells
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product Yes
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients: Adults diagnosed with SJS-TEN with at least 10% of body surface area involved.
    Patients : Adultes avec diagnostic de SJS-TEN avec 10% ou plus de la surface corporelle décollée-décollable
    E.1.1.1Medical condition in easily understood language
    Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) are muco-cutaneous diseases characterized by more or less extensive cutaneous-mucosal detachments
    Stevens-Johnson syndrome (SJ ) et toxic epidermal necrolysis (TEN) sont des maladie cutanéo-muqueuse grave caractérisées par des décollements cutanéo-muqueux plus ou moins étendus.
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10025166
    E.1.2Term Lyell syndrome
    E.1.2System Organ Class 100000004858
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    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. Indeed, usual mean time to epithelialization is 14 days, thus, considering the results of the survey about MCID described above, we decided to evaluate the rate of complete or almost complete healing at D7 post-infusion (i.e 10 days after inclusion).
    E.2.2Secondary objectives of the trial
    To evaluate the impact of ASCs treatment on SJS-TEN clinical course and immunological markers.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients:
    - Patients aged from 18 to 75 years-old
    - Admission less than 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)
    - Written consent from patient or trustworthy person or legal representant or family member
    - Affiliated to a social security scheme

    Donors:
    - Donors aged from 18 to 55 years old
    - Admission for a programmed plastic surgery of liposuction or aspiration in the abdominal wall under general anesthesia
    - Selection criteria according to stem cell donor health history questionnaire from Agence de la Biomédecine
    - Written consent
    - Affiliated to a social security scheme
    E.4Principal exclusion criteria
    Patients:
    - 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 or allogenic bone marrow transplantation
    - Participation in other interventional drug research
    - Patient deprived of freedom
    - Any psychological, familial, sociological or geographical condition potentially hampering compliance with the research protocol and follow-up schedule

    Donors:
    - Positive viral serology (HBV, HCV, HIV, HEV, syphilis, HTLV, active infection with IgM+ for toxoplasmosis, EBV, CMV)
    - Deprived of freedom
    - Significant comorbidities according to donor health history or authenticated risk factors for viral infections in the past 12 months:
    • Multiple sexual partners between the donor or his or her usual partner
    • Intravenous addiction to the donor or regular partner
    • Accident of exposure to blood or derivatives suspected of being contaminated
    - Treatment with extractive pituitary hormones (including growth hormones)
    - Human dura mater transplant
    - Surgical history of the central nervous system
    - Dementia or neurological disease that may evoke subacute spongiform encephalopathy
    - Family history as part of subacute spongiform encephalopathy
    - Hematological malignancies
    - Active or cured cancer
    - System disease
    - Active generalized infection (viral, parasitic, tuberculosis, leprosy...)
    - Multiple adenopathy, splenomegaly, hepatomegaly
    - Icterus
    - Chemotherapy history, irradiation
    - Long-term steroids ( > 90 days)
    E.5 End points
    E.5.1Primary end point(s)
    Safety :
    The toxicity is defined as the observation of at least one of the following adverse events:
    - Persisting fever (> 38.5°C between D5 and D10) independently of a sepsis
    - Local acute infusional toxicity: phlebitis, hematoma, local infection between D0 and D3
    - Anaphylaxis or severe systemic reaction following ASCs administration at D0
    - Rash between D0 and D3, involving ≥30% of BSA
    - Cardiac complications in absence of previous cardiologic disease between D0 and D3
    - Sustained ventricular arrhythmia between D0 and D3
    - ST-elevation myocardial infarction between D0 and D3
    - Acute pulmonary edema with PaO2 < 60 mm Hg or signs of congestion (RR> 25, laboured breathing, cyanosis, dyspnoea NYHA Grade IV) in absence of infectious pneumopathy and specific SJS-TEN lung involvement between D0 and D3
    - Cardiogenic shock with signs of hypoperfusion between D0 and D3
    - Thrombo-embolic event between D0 and D3
    - Cerebral infarction between D0 and D3
    - Acute renal failure or doubling of creatinine between D0 and D3
    - Epilepsia between D0 and D3
    - Sepsis between D0 and D3
    - Death between D0 and D3

    If one of these outcome is observed, then it will be considered as a toxicity for the patient.

    Efficacy:
    The efficacy is defined as the cutaneous re-epithelialization at D7 post-infusion according to the percentage of cutaneous BSA re-epithelialized in comparison to maximal cutaneous detachable-detached BSA observed previous or after infusion.

    Complete response, almost complete response : At least 90% of detached-detachable cutaneous BSA re-epithelialized.
    Partial response : Between 50% and 90% of detached-detachable cutaneous BSA re-epithelialized.
    Failure : Less than 50% of detached-detachable cutaneous BSA re-epithelialized.

    For simplicity, regarding the small sample size of the trial, complete (or almost complete) response and partial response will be considered as procedure success. In case of patient’s death, his(her) response will be considered as a failure of the procedure.
    Cutaneous detachable-detached BSA will be assessed according to the international standardized Wallace rule of Nines: 9% of body surface area involved is given for involvement of the head, each arm, chest and abdomen, 18% for each leg and the back, and 1% for the groin. Also the patient's palm can serve a reference point roughly equivalent to 1% of the body surface area for total assessment.
    E.5.1.1Timepoint(s) of evaluation of this end point
    at Day 7.
    E.5.2Secondary end point(s)
    - Rate of observed and predicted death at one month by the SCORTEN .
    - Duration of hospitalization according to historical cohort related to BSA involved, onset of the disease and SCORTEN.
    - Duration of each mucous membranes healing ie.(buccal, nasal, genital, eyes).
    - Rate of sepsis at one month.
    - Rate of intensive care transfer at one month.
    - Rate of sequelae at M12
    - Th1/Th2 immune response in the peripheral blood of the patients before injection at D0, D7 and M1
    - Evaluation of expression profile of Th1/Th2 associated chemokines and anti-inflammatory chemokines in the peripheral blood after injection at D0, D7 and M1.
    - Epidermal chimerism research on healed skin biopsy and peripheral blood 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.
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 Month
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers Yes
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    -Patient with confirmed SJS-TEN diagnosis hospitalized in the department of Dermatology or intensive care médicine.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-09-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-06
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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