Recessive dystrophic epidermolysis bullosa Skin and Connective Tissue Diseases Epidermolysis bullosa
Conditions
Interventions
Children with RDEB from Great Ormond Street NHS Trust will be invited to take part in the study. Each subject will undergo an initial screening including physical examination, vital signs and disease
Sponsors
King's College London (UK)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subjects who have a diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) characterised by partial or complete collagen VII deficiency 2. Subjects who are = 12 months and = 17 years of age at the time of enrolment 3. Subjects whose responsible relative/guardian has voluntary signed and dated an Informed Consent Form (ICF) prior to the first study intervention. Whenever the minor child is able to give consent, the minor's assent will be obtained in addition to the signed consent of the minor's legal guardian
Exclusion criteria
Exclusion criteria: Current exclusion criteria as of 10/09/2013: 1. Subjects who have had other investigational medicinal products within 90 days prior to screening or during the treatment phase 2. Subjects who have received immunotherapy including oral corticosteroids for more than 1 week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study 3. Subjects with a known allergy to any of the constituents of the investigational product 4. Subjects with signs of active infection 5. Subjects with a medical history or evidence of malignancy, including cutaneous squamous cell carcinoma 6. Subjects with both positive C7 ELISA and a positive indirect immunofluorescence (IIF) with binding to the base of salt split skin 7. Subjects who are pregnant or of child-bearing potential who are not abstinent or practicing an acceptable means of contraception, as determined by the Investigator, for the duration of the treatment phase Previous exclusion criteria: 1. Subjects who have had other investigational medicinal products within 90 days prior to screening or during the treatment phase 2. Subjects who have received immunotherapy including oral corticosteroids for more than 1 week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study 3. Subjects with a known allergy to any of the constituents of the investigational product 4. Subjects with signs of active infection 5. Subjects with a medical history or evidence of malignancy, including cutaneous squamous cell carcinoma 6. Subjects with positive serum antibodies to C7 confirmed by ELISA 7. Subjects who are pregnant or of child-bearing potential who are not abstinent or practicing an acceptable means of contraception, as determined by the Investigator, for the duration of the treatment phase
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Current primary outcome measures as of 15/01/2015: The safety of allogeneic intravenously administered MSCs in children with RDEB over a 12-month period Previous primary outcome measures: The safety of allogeneic intravenously administered MSCs in children with RDEB over a 24-month period | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Increase in collagen VII deposition at the DEJ post treatment 2. Quantitative analysis of the donor cells dermal chimerism 3. Improvement of haematological and serological markers of generalised inflammation 4. General clinical appearance of the skin based on medical photographs, generalised severity score and BEBSS score 5. Improved quality of life 6. Pain scoring 7. Reduction in blister numbers 8. Increase in skin strength measured by increased time to blister formation after skin suction at screening and D120 | — |
Countries
United Kingdom
Outcome results
None listed