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

NCT02670837 | Completed Results DMC FDA Device

• 1 other identifier: 2015LS154
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

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.

Conditions

Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• Participants With Lesion Free Skin

  1 year after grafting

• Longevity of Grafted Skin

  1 year after grafting

• Percentage Change of a Patient's IScorEB Assessment Score

  Baseline and 6 weeks

• Percentage Change of a Patient's IScoreEB Assessment Score

  Baseline and 12 weeks

• Scar-free Healing of the Body Sites of the Donor

  1 year after grafting

Study Arms (2)

Graft from HCT donor

EXPERIMENTAL

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

Self donor from intact skin patch

EXPERIMENTAL

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

Interventions

Cellutome Epidermal Harvesting System DEVICE

Graft from HCT donor; Self donor from intact skin patch

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 \[Arm B\])

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

University of Minnesota Masonic Cancer Center and Medical Center

Minneapolis, Minnesota, 55455, United States

Location

Related Publications (1)

• 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.

  PMID: 32866988 DERIVED

MeSH Terms

Conditions

Epidermolysis Bullosa

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Results Point of Contact

• Title: Dr. Christen Ebens
• Organization: University of Minnesota, Masonic Cancer Center

ebens012@umn.edu

(612) 626-5654

Study Officials

• Christen Ebens, MD, MPH

  Masonic Cancer Center, University of Minnesota

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 8, 2016
• First Posted: February 2, 2016
• Study Start: August 4, 2016
• Primary Completion: March 16, 2023
• Study Completion: April 3, 2024
• Last Updated: April 9, 2024
• Results First Posted: March 12, 2024

Record last verified: 2024-04

Locations

US (1)