Study of Cellutome System for Treatment of Individual Lesions in EB Pts

Study Overview
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Brief Summary
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Few but persistent wounds often remain even after successful hematopoietic cell transplantation for systemic genodermatosis epidermolysis bullosa (EB). The investigators propose local wound therapy using epidermal skin grafting from the same donor that provided the hematopoietic graft, or from the same EB individual with a mosaic (naturally gene corrected) skin. In both cases permissive immune system and skin chimerism is expected to enable long-term epidermal engraftment and wound healing. The investigators will use FDA approved vacuum device (CelluTome®, Regulation number 878.4820) that enables scar-free harvesting of epidermis and its transfer on a square of surgical tape (Tegaderm®) to the recipient as a wound dressing.

Official Title
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Study of Epidermal Grafting Using the CelluTome Epidermal Harvesting System for the Treatment of Individual Lesions in Persons With Epidermolysis Bullosa [MT2015-36]

Conditions
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Epidermolysis Bullosa

Intervention / Treatment
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* Device: Cellutome Epidermal Harvesting System


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient (Recipient) Diagnosis of Dystrophic Epidermolysis Bullosa (DEB) or Junctional Epidermolysis Bullosa (JEB) with at least one wound, visibly free from infection (or previously treated) and meets the eligibility for Arm A or Arm B based on the skin graft source: Cell harvest from previous hematopoietic cell transplantation (HCT) donor (Arm A) - not applicable if Arm B At least 6 months after hematopoietic cell transplantation with donor chimerism Peripheral blood donor chimerism should be measured within 21 days of grafting and be >/= 5% and stable. Stability of chimerism will be determined by the protocol team and based on 3 peripheral blood chimerism values at least 1 month apart. No history of pre-BMT autoimmune cytopenias Off immune suppressive therapy Original transplant donor is available and willing to be the epidermis donor Self-donation (Arm B) - not applicable if Arm A Proven somatic reversion Site for skin grafting free of cellulitis and any other clinically evident abnormalities Meets donor eligibility Insurance pre-authorization for procedure, if applicable Voluntary written consent (patient or parent/guardian for minors with assent) prior to any research related procedures or treatment. Skin Graft Donor (either hematopoietic cell transplantation donor for the EB patient [Arm A] or EB patient herself/himself [Arm B]) Age > 2 years (based on prior safety testing of the device) Healthy on physical examination in the opinion of the evaluating provider Negativity for Hepatitis B and C, HIV, and HTLV1/2 within 30 days of donation Voluntary written consent (donor or parent/guardian for minors with assent) prior to any research related procedures
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Graft from HCT donor<br>Cells are harvested from a donor using Cellutome, then transferred via Adaptic dressing to the recipient's wound with up to 3 donor harvest sites/treated wound sites on day 0. | Device: Cellutome Epidermal Harvesting System<br> <br> |
| Experimental: Self donor from intact skin patch<br>Cells are harvested from the subject using Cellutome, then transferred via Adaptic dressing to that subject's wound with up to 3 harvest sites/treated wound sites on day 0. | Device: Cellutome Epidermal Harvesting System<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of grafts successfully treated | If the body surface area affected by the wound is at least 50% lower at 12 weeks relative to baseline, the graft will be considered successful. | 12 weeks after grafting | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of grafted skin | Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting. | 1 year after grafting | 
| Longevity of grafted skin | Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting. | 1 year after grafting | 
| Functionality of grafted skin | Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting. | 1 year after grafting | 
| Percentage change of a patient's IScorEB assessment score | Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire | 6 weeks after grafting | 
| Percentage change of a patient's IScoreEB assessment score | Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire | 12 weeks after grafting | 
| Seamless, scar-free healing of the body sites of the donor | Percentage of donors with no evidence of non-healed skin | 1 year after grafting | 
| Safety during healing of the body sites of the donor | Percentage of donors with no evidence of non-healed skin | 1 year after grafting |