Clinical trial NCT02670837: Study of Cellutome System for Treatment of Individual Lesions in EB Pts
Official title: Study of Epidermal Grafting Using the CelluTome Epidermal Harvesting System for the Treatment of Individual Lesions in Persons With Epidermolysis Bullosa [MT2015-36]
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Epidermolysis Bullosa
Lead sponsor: Masonic Cancer Center, University of Minnesota
Sponsor class: OTHER
First posted: 2016-02-02
Enrollment: 34
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: 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 non-adherent silicone dressing (Adaptic) to the recipient as a wound dressing.


Eligibility criteria: 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

Primary outcomes:
measure: Percentage of Grafts Successfully Treated
timeFrame: 12 weeks after grafting
Secondary outcomes:
measure: Participants With Lesion Free Skin
timeFrame: 1 year after grafting
measure: Longevity of Grafted Skin
timeFrame: 1 year after grafting
measure: Percentage Change of a Patient's IScorEB Assessment Score
timeFrame: Baseline and 6 weeks
measure: Percentage Change of a Patient's IScoreEB Assessment Score
timeFrame: Baseline and 12 weeks
measure: Scar-free Healing of the Body Sites of the Donor
timeFrame: 1 year after grafting
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: University of Minnesota Masonic Cancer Center and Medical Center
state: Minnesota
Study officials:
affiliation: Masonic Cancer Center, University of Minnesota
name: Christen Ebens, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References:
citation: Ebens CL, McGrath JA, Riedl JA, Keith AR, Lilja G, Rusch S, Keene DR, Tufa SF, Riddle MJ, Shanley R, Van Heest AE, Tolar J. Immune tolerance of allogeneic haematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds. Br J Dermatol. 2021 Jun;184(6):1161-1169. doi: 10.1111/bjd.19503. Epub 2020 Dec 14.