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

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

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.

Study Overview

• Status: Completed
• Conditions: Epidermolysis Bullosa
• Intervention / Treatment: Device: Cellutome Epidermal Harvesting System

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota Masonic Cancer Center and Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Graft from HCT donor; 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
Experimental: Self donor from intact skin patch; 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

What is the study measuring?

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
Participants With Lesion Free Skin	Participants without Squamous cell carcinoma (SCC) at graft site. Wound reassessments will be performed via photographs and follow-up visits.	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. Wound reassessments will be performed via photographs and follow-up visits.	1 year after grafting
Percentage Change of a Patient's IScorEB Assessment Score	Measure percent of changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life.	Baseline and 6 weeks
Percentage Change of a Patient's IScoreEB Assessment Score	Measure percent of changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life.	Baseline and 12 weeks
Scar-free Healing of the Body Sites of the Donor	Percentage of donors with no evidence of non-healed skin. Wound reassessments will be performed via photographs and follow-up visits.	1 year after grafting

Collaborators and Investigators

• Sponsor: Masonic Cancer Center, University of Minnesota
• Investigators: Principal Investigator: Christen Ebens, MD, MPH, Masonic Cancer Center, University of Minnesota

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): August 4, 2016
• Primary Completion (Actual): March 16, 2023
• Study Completion (Actual): April 3, 2024

Study Registration Dates

• First Submitted: January 8, 2016
• First Submitted That Met QC Criteria: January 29, 2016
• First Posted (Estimated): February 2, 2016

Study Record Updates

• Last Update Posted (Actual): April 9, 2024
• Last Update Submitted That Met QC Criteria: April 5, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Epidermolysis Bullosa
• Other Study ID Numbers: 2015LS154

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: No