Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cellutome Epidermal Harvesting System | Device |
|
| 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 |
| 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 |
Not provided
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
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
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])
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Christen Ebens, MD, MPH | Masonic Cancer Center, University of Minnesota | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Minnesota Masonic Cancer Center and Medical Center | Minneapolis | Minnesota | 55455 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32866988 | Derived | 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. |
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | 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. Cellutome Epidermal Harvesting System |
| FG001 | Donor | Cells are harvested from a donor using Cellutome, then transferred via Adaptic dressing to the recipient's wound. Cellutome Epidermal Harvesting System |
| FG002 | Self-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. Cellutome Epidermal Harvesting System |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | 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. Cellutome Epidermal Harvesting System |
| BG001 | Donor |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Number | 95% Confidence Interval | Percentage of grafts | 12 weeks after grafting | grafts | grafts |
|
|
1 year
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Christen Ebens | University of Minnesota, Masonic Cancer Center | (612) 626-5654 | ebens012@umn.edu |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 7, 2021 | Dec 8, 2023 | Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Sep 24, 2021 | Dec 8, 2023 | ICF_001.pdf |
Not provided
| ID | Term |
|---|---|
| D004820 | Epidermolysis Bullosa |
| ID | Term |
|---|---|
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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 |
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. Cellutome Epidermal Harvesting System |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| grafts |
|
|
| Secondary | 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. | Posted | Number | 95% Confidence Interval | Percentage of participants | 1 year after grafting |
|
|
|
| Secondary | 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. | Posted | Number | 95% Confidence Interval | Percentage of participants | 1 year after grafting |
|
|
|
| Secondary | 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. | Posted | Mean | 95% Confidence Interval | Percent change | Baseline and 6 weeks |
|
|
|
| Secondary | 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. | Posted | Mean | 95% Confidence Interval | Percent change | Baseline and 12 weeks |
|
|
|
| Secondary | 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. | Posted | Number | 95% Confidence Interval | Percentage of participants | 1 year after grafting |
|
|
|
| 2 |
| 17 |
| 0 |
| 17 |
| 0 |
| 17 |
| EG001 | Donor | Cells are harvested from a donor using Cellutome, then transferred via Adaptic dressing to the recipient's wound. | 0 | 17 | 0 | 17 | 0 | 17 |
Not provided
Not provided
Not provided
| D030342 | Genetic Diseases, Inborn |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
|
|