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

Primary Purpose

Epidermolysis Bullosa

Status
Active
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Cellutome Epidermal Harvesting System
Sponsored by
Masonic Cancer Center, University of Minnesota
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Epidermolysis Bullosa

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Minnesota Masonic Cancer Center and Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Graft from HCT donor

Self donor from intact skin patch

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Safety 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.
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.
Functionality 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.
Percentage change of a patient's IScorEB assessment score
Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire
Percentage change of a patient's IScoreEB assessment score
Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire
Seamless, scar-free healing of the body sites of the donor
Percentage of donors with no evidence of non-healed skin
Safety during healing of the body sites of the donor
Percentage of donors with no evidence of non-healed skin

Full Information

First Posted
January 8, 2016
Last Updated
June 2, 2023
Sponsor
Masonic Cancer Center, University of Minnesota
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1. Study Identification

Unique Protocol Identification Number
NCT02670837
Brief Title
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

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 4, 2016 (Actual)
Primary Completion Date
March 16, 2023 (Actual)
Study Completion Date
November 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Masonic Cancer Center, University of Minnesota

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Epidermolysis Bullosa

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
35 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Graft from HCT donor
Arm Type
Experimental
Arm Description
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.
Arm Title
Self donor from intact skin patch
Arm Type
Experimental
Arm Description
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.
Intervention Type
Device
Intervention Name(s)
Cellutome Epidermal Harvesting System
Primary Outcome Measure Information:
Title
Percentage of grafts successfully treated
Description
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.
Time Frame
12 weeks after grafting
Secondary Outcome Measure Information:
Title
Safety of grafted skin
Description
Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting.
Time Frame
1 year after grafting
Title
Longevity of grafted skin
Description
Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting.
Time Frame
1 year after grafting
Title
Functionality of grafted skin
Description
Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting.
Time Frame
1 year after grafting
Title
Percentage change of a patient's IScorEB assessment score
Description
Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire
Time Frame
6 weeks after grafting
Title
Percentage change of a patient's IScoreEB assessment score
Description
Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire
Time Frame
12 weeks after grafting
Title
Seamless, scar-free healing of the body sites of the donor
Description
Percentage of donors with no evidence of non-healed skin
Time Frame
1 year after grafting
Title
Safety during healing of the body sites of the donor
Description
Percentage of donors with no evidence of non-healed skin
Time Frame
1 year after grafting

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Christen Ebens, MD, MPH
Organizational Affiliation
Masonic Cancer Center, University of Minnesota
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Minnesota Masonic Cancer Center and Medical Center
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
32866988
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.
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Study of Cellutome System for Treatment of Individual Lesions in EB Pts

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