Safety and efficacy of allogenic adipose tissue-derived mesenchymal stromal cells in patients with epidermolysis bullosa: Clinical trial phase I/II

Who can take part

• Age > 2 years (incl.)
• Clinical diagnosis of hereditary EB supported by histological diagnosis (electron microscopy, immunofluorescence antigen mapping) and/or DNA analysis
• The extent of the disease is at least 10% of the body surface
• Participants in a clinical trial of childbearing potential must agree to the use of prescribed contraceptive methods for the duration of the clinical study and for at least 6 months following the last dose of the study medication: a) Women - Proper use of a highly reliable method of contraception, ie combined hormonal contraception (in oral, vaginal or transdermal dosage form), gestagen hormonal contraception associated with ovulation inhibition (in oral or injectable dosage form), non-hormonal intrauterine device or intrauterine device releasing hormones, or the presence of bilateral tubal occlusion, a partner's vasectomy, or adherence to sexual abstinence as part of the patient's normal lifestyle. b) Men - Adherence to sexual abstinence or the use of an adequate contraceptive method (ie condom) in case of sexual intercourse
• Willingness and ability to adhere to restrictions in the care of study lesions and to adhere to the schedule of visits and examinations during the course of the study

Who cannot take part

• Systemic infection or sepsis 7 days before MSC administration
• Previous administration of a medicinal product in a clinical trial in the 30 days or 5 elimination half-lives (whichever is longer) before the enrolment to this trial (applies to clinical investigations of medical devices, as well)
• Treatment with blood transfusion (erythrocyte concentrate or whole blood transfusion) 7 days before MSC administration
• Clinical signs of infection in the study lesions 7 days before MSC administration
• Other severe somatic or psychiatric illnesses that are not adequately controlled and, in the opinion of the investigator, would not allow the study protocol to be followed
• Allergy/hypersensitivity to any component of Hypotermosol solution
• History of basal cell or squamous cell carcinoma of the skin in the last 5 years
• History of other malignancies of any type at any time during life
• Severe lung disease that requires home oxygenation
• Severe kidney or liver disease
• Life expectancy less than 90 days
• Severe immune response to allogeneic human cells
• Pregnancy or breastfeeding
• Inability to tolerate repeated skin injections (especially in pediatric patients)
• Dilatation cardiomyopathy