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N/A N=34 Supportive Care

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

Epidermolysis Bullosa

Enrolled (actual)
34
Serious AEs
0.0%
Results posted
Mar 2024
Primary outcome: Primary: Percentage of Grafts Successfully Treated — 78 Percentage of grafts

Study Design & Population

Study type
Interventional
Phase
N/A
Interventions
Cellutome Epidermal Harvesting System (Device)
Age
Pediatric, Adult, Older Adult
Sex
All
Sponsor
Masonic Cancer Center, University of Minnesota
Primary completion
Mar 2023

Outcome Measures

OutcomeResultp-value
PRIMARY
Percentage of Grafts Successfully Treated
78
SECONDARY
Participants With Lesion Free Skin
100
SECONDARY
Longevity of Grafted Skin
34
SECONDARY
Percentage Change of a Patient's IScorEB Assessment Score
-2.1; -1.9; 3.1
SECONDARY
Percentage Change of a Patient's IScoreEB Assessment Score
-8.1; -10.4; -0.4
SECONDARY
Scar-free Healing of the Body Sites of the Donor
100

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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02670837). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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