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

Epidermolysis Bullosa Clinical Trial

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]

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02670837
• Other study ID #: 2015LS154
• Status: Completed
• Phase: N/A
• Start date: August 4, 2016
• Est. completion date: April 3, 2024

Study information

• Verified date: April 2024
• Source: Masonic Cancer Center, University of Minnesota
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 34
• Est. completion date: April 3, 2024
• Est. primary completion date: March 16, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Epidermolysis Bullosa

Intervention

Device:
• Cellutome Epidermal Harvesting System

Locations

Country	Name	City	State
United States	University of Minnesota Masonic Cancer Center and Medical Center	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Masonic Cancer Center, University of Minnesota

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
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.	12 weeks after grafting
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.	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.	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.	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.	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.	1 year after grafting