Q81.2 Q81.8
Conditions
Interventions
Group 1: Defect coverage by autologous transplantation of in-vitro cultured keratinocyte sheets
Sponsors
Klinik für Plastische und Handchirurgie, Universitätsklinikum Freiburg
Eligibility
Sex/Gender
All
Age
18 Years to 60 Years
Inclusion criteria
Inclusion criteria: 1. Patient’s assent 2. Molecularly-confirmed diagnosis of JEB due to mutations in the laminin-332 genes or RDEB 3. Presence of an easy-accessible, clinically and immunohistologically-proved revertant skin patch with a growth of >6mm2 4. Ability of patient to understand the nature of the trial and trial-related procedures and to comply with them 5. Able to travel to trial site for all clinic visits
Exclusion criteria
Exclusion criteria: 1. Simultaneous participation or participation in any other interventional trial within the past three months before entering this trial (at least 6 months for cell therapy trials) 2. Patients with molecularly-confirmed EB simplex 3. Reasons to assume reduced patient compliance for the course of the trial 4. Evidence of any other medical conditions (such as psychiatric illness or active infection) based on physical examination, or laboratory findings that may interfere with the planned treatment, affect the patient’s compliance, or place the patient at high risk of complications related to the treatment; at investigators discretion 5. Clinically significant or unstable concurrent disease or other clinical contraindications (e.g. inability to tolerate general anaesthesia) 6. Contraindications to the local or systemic antibiotics and/ or corticosteroids foreseen by the protocol 7. Patients anticipated to be unwilling or unable to comply with the requirements of the protocol 8. Patients with over 60 years of age 9. For female patients of child-bearing age: Current pregnancy or nursing period or unwillingness to use adequate contraception to achieve a Pearl index < 1 or to stay sexually abstinent during the course of the trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Demonstrate the feasibility of the therapeutic intervention | — |
Secondary
| Measure | Time frame |
|---|---|
| local cure of EB with associated improvement in quality of life Follow-up examinations at 3, 6 and 12 months postoperative: - clinical check-up and local biopsies to immunohistochemically and genetically confirm the revertant mosaicism in the transplanted areal - in addition evaluation of the EBDASI and QOLEB scores to correlate subjective parameters of the patients | — |
Countries
Germany
Contacts
Public ContactAlexander Runkel
Klinik für Plastische- und Handchirurgie Universitätsklinikum Freiburg
Outcome results
None listed