Epidermolysis bullosa congenita
Conditions
Interventions
Product Name: allogeneic adipose tissue-derived MSC
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: Human allogeneic adipose tissue-derived mesenchymal stem cells expanded
Other des
Sponsors
Masarykova univerzita
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Age > 1 year (incl.) 2) Clinical diagnosis of hereditary EB supported by histological diagnosis (electron microscopy, immunofluorescence antigen mapping) and DNA analysis 3) The extent of the disease is at least 10% of the body surface 4) 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. 5) 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. Are the trial subjects under 18? yes Number of subjects for this age range: 11 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 4 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1) Systemic infection or sepsis 7 days before MSC administration 2) Treatment with blood transfusion (erythrocyte concentrate or whole blood transfusion) 7 days before MSC administration 3) Clinical signs of infection in the study lesions 7 days before MSC administration 4) Inability to tolerate repeated skin injections (especially in pediatric patients) 5) History of basal cell or squamous cell carcinoma of the skin in the last 5 years 6) History of other malignancies of any type at any time during life 7) Severe lung disease that requires home oxygenation 8) Severe kidney or liver disease 9) Dilatation cardiomyopathy 10) Life expectancy less than 90 days 11) Severe immune response to allogeneic human cells 12) 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 13) 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) 14) Pregnancy or breastfeeding 15) Allergy/hypersensitivity to any component of Hypotermosol solution
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to assess the safety of human allogeneic mesenchymal stromal cells administered intradermally;Secondary Objective: The secondary objective of the study is to obtain pilot data on the efficacy of the treatment;Primary end point(s): Incidence of adverse events of special interest (AESI): severe local reaction at the injection site other than blistering, severe systemic allergic reaction at any time after MSC administration, severe systemic or local infectious complications at any time after MSC administration (combined primary safety endpoint);Timepoint(s) of evaluation of this end point: Cohort 1: - Day 90 after 2nd administration of the 4th adult patient - End of the trial (Day 290 after the 2nd administration) Cohort 2: - Day 7 after 1st administration of the 1st patient aged 12-17 years - Day 7 after 1st administration of the 1st patient aged 2-11 years - Day 7 after 1st administration of the 1st patient aged 12-23 months - End of the trial (Day 290 after the 2nd administration of all pediatric patients) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary safety endpoints: - Incidence, type, seriousness, intensity of AEs and their relation to study medication Efficacy endpoints: - Non-healed part in the area marked in the study active and control lesions - Change in the area of non-healed part from baseline - Estimation of the total area of lesions on the patient's body (% of body surface area);Timepoint(s) of evaluation of this end point: Secondary safety endpoints: End of the trial (Day 290 after the 2nd administration) Efficacy endpoints: a) Day 4, 15, 30, and 75 after the 1st administration and Day 4, 15, 30 and 90 after the 2nd administration b) Day 30 after the 1st administration and Day 30 after the 2nd administration c) Day 1 vs. Day 30 after the 1st and 2nd administration | — |
Countries
Czech Republic
Contacts
Public ContactFarmÚ- Centrum klinických hodnocení
Masarykova univerzita - LF
Outcome results
None listed