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

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ClinicalTrials.gov Identifier: NCT02670837
Recruitment Status : Recruiting
First Posted : February 2, 2016
Last Update Posted : September 26, 2018
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

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 square of surgical tape (Tegaderm®) to the recipient as a wound dressing.

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Device: Cellutome Epidermal Harvesting System Not Applicable

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
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]
Actual Study Start Date : August 4, 2016
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2021


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



Primary Outcome Measures :
  1. Percentage of grafts successfully treated [ Time Frame: 12 weeks after grafting ]

    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.



Secondary Outcome Measures :
  1. Safety of grafted skin [ Time Frame: 1 year after grafting ]
    Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting.

  2. Longevity of grafted skin [ Time Frame: 1 year after grafting ]
    Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting.

  3. Functionality of grafted skin [ Time Frame: 1 year after grafting ]
    Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting.

  4. Percentage change of a patient's IScorEB assessment score [ Time Frame: 6 weeks after grafting ]
    Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire

  5. Percentage change of a patient's IScoreEB assessment score [ Time Frame: 12 weeks after grafting ]
    Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire

  6. Seamless, scar-free healing of the body sites of the donor [ Time Frame: 1 year after grafting ]
    Percentage of donors with no evidence of non-healed skin

  7. Safety during healing of the body sites of the donor [ Time Frame: 1 year after grafting ]
    Percentage of donors with no evidence of non-healed skin



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

Patient (Recipient)

  • Diagnosis of Recessive 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 by clinical exam and immune fluorescence (antigen mapping)
    • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02670837


Contacts
Contact: Kim Nelson, RN 612-273-2925 knelso62@fairview.org

Locations
United States, Minnesota
University of Minnesota Masonic Cancer Center and Medical Center Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Kim Nelson    612-273-2925    knelso62@fairview.org   
Principal Investigator: Jakub Tolar, MD, PhD         
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Principal Investigator: Jakub Tolar, MD, PhD Masonic Cancer Center, University of Minnesota

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT02670837     History of Changes
Other Study ID Numbers: 2015LS154
First Posted: February 2, 2016    Key Record Dates
Last Update Posted: September 26, 2018
Last Verified: September 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: No
Pediatric Postmarket Surveillance of a Device Product: No
Product Manufactured in and Exported from the U.S.: No

Additional relevant MeSH terms:
Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous