Study of Epidermal Grafting Using the CelluTome Epidermal Harvesting System for the Treatment of Individual Lesions in Persons With Epidermolysis Bullosa [MT2015-36]
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 34
- 试验地点
- 2
- 主要终点
- Percentage of Grafts Successfully Treated
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- 未提供
结局指标
主要结局
Percentage of Grafts Successfully Treated
时间窗: 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.
次要结局
- Participants With Lesion Free Skin(1 year after grafting)
- Percentage Change of a Patient's IScoreEB Assessment Score(Baseline and 12 weeks)
- Scar-free Healing of the Body Sites of the Donor(1 year after grafting)
- Longevity of Grafted Skin(1 year after grafting)
- Percentage Change of a Patient's IScorEB Assessment Score(Baseline and 6 weeks)