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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]

Status
Completed
Phases
NA
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT02670837
Enrollment
34
Registered
2016-02-02
Start date
2016-08-04
Completion date
2024-04-03
Last updated
2024-04-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Epidermolysis Bullosa

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.

Interventions

Sponsors

Masonic Cancer Center, University of Minnesota
Lead SponsorOTHER

Study design

Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SUPPORTIVE_CARE
Masking
NONE

Eligibility

Sex/Gender
ALL
Healthy volunteers
No

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

Design outcomes

Primary

MeasureTime frameDescription
Percentage of Grafts Successfully Treated12 weeks after graftingIf 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

MeasureTime frameDescription
Participants With Lesion Free Skin1 year after graftingParticipants without Squamous cell carcinoma (SCC) at graft site. Wound reassessments will be performed via photographs and follow-up visits.
Longevity of Grafted Skin1 year after graftingPercentage 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.
Percentage Change of a Patient's IScorEB Assessment ScoreBaseline and 6 weeksMeasure 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.
Percentage Change of a Patient's IScoreEB Assessment ScoreBaseline and 12 weeksMeasure 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.
Scar-free Healing of the Body Sites of the Donor1 year after graftingPercentage of donors with no evidence of non-healed skin. Wound reassessments will be performed via photographs and follow-up visits.

Countries

United States

Participant flow

Participants by arm

ArmCount
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
17
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
17
Total34

Withdrawals & dropouts

PeriodReasonFG000FG001FG002
Overall StudyDeath200

Baseline characteristics

CharacteristicGraft From HCT DonorDonorTotal
Age, Categorical
<=18 years
15 Participants10 Participants25 Participants
Age, Categorical
>=65 years
0 Participants0 Participants0 Participants
Age, Categorical
Between 18 and 65 years
2 Participants7 Participants9 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants1 Participants2 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
16 Participants16 Participants32 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Asian
1 Participants1 Participants2 Participants
Race (NIH/OMB)
Black or African American
0 Participants0 Participants0 Participants
Race (NIH/OMB)
More than one race
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants4 Participants5 Participants
Race (NIH/OMB)
White
15 Participants12 Participants27 Participants
Region of Enrollment
United States
17 participants17 participants34 participants
Sex: Female, Male
Female
8 Participants15 Participants23 Participants
Sex: Female, Male
Male
9 Participants2 Participants11 Participants

Adverse events

Event typeEG000
affected / at risk
EG001
affected / at risk
deaths
Total, all-cause mortality
2 / 170 / 17
other
Total, other adverse events
0 / 170 / 17
serious
Total, serious adverse events
0 / 170 / 17

Outcome results

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.

Time frame: 12 weeks after grafting

ArmMeasureValue (NUMBER)
Graft From HCT DonorPercentage of Grafts Successfully Treated78 Percentage of grafts
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.

Time frame: 1 year after grafting

ArmMeasureValue (NUMBER)
Graft From HCT DonorLongevity of Grafted Skin34 Percentage of participants
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.

Time frame: 1 year after grafting

ArmMeasureValue (NUMBER)
Graft From HCT DonorParticipants With Lesion Free Skin100 Percentage of participants
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.

Time frame: Baseline and 6 weeks

ArmMeasureGroupValue (MEAN)
Graft From HCT DonorPercentage Change of a Patient's IScorEB Assessment ScoreChange in pain QOL score-2.1 Percent change
Graft From HCT DonorPercentage Change of a Patient's IScorEB Assessment ScoreChange in itch QOL score-1.9 Percent change
Graft From HCT DonorPercentage Change of a Patient's IScorEB Assessment ScoreChange in General QOL score3.1 Percent change
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.

Time frame: Baseline and 12 weeks

ArmMeasureGroupValue (MEAN)
Graft From HCT DonorPercentage Change of a Patient's IScoreEB Assessment ScoreChange in general QOL score-0.4 Percent change
Graft From HCT DonorPercentage Change of a Patient's IScoreEB Assessment ScoreChange in pain QOL score-8.1 Percent change
Graft From HCT DonorPercentage Change of a Patient's IScoreEB Assessment ScoreChange in itch QOL score-10.4 Percent change
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.

Time frame: 1 year after grafting

ArmMeasureValue (NUMBER)
Graft From HCT DonorScar-free Healing of the Body Sites of the Donor100 Percentage of participants

Source: ClinicalTrials.gov · Data processed: Feb 24, 2026