EBSTEM

• Research type

  Research Study

• Full title

  A prospective phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in children with recessive dystrophic epidermolysis bullosa.

• IRAS ID

  99899

• Contact name

  John Alexander McGrath

• Sponsor organisation

  King's College London

• Eudract number

  2012-001394-87

• Research summary

  Recessive dystrophic epidermolysis bullosa (RDEB) is an inherited blistering skin disease. This is a very severe condition in which skin injury leads to blisters and wounds that may be slow to heal or which leave non-healing erosions (open wounds). The increased skin fragility can lead to scarring (especially of the hands and feet) and an increased risk of skin cancer (squamous cell carcinoma). Currently, there is no effective treatment for RDEB and we are unable to prevent the disease complications. The cause of RDEB is a lack of a skin protein called collagen VII (C7), the major component of velcro-like structures (anchoring fibrils) that help stick the outer skin layer (epidermis) to the underlying inner layer (dermis). A previous study of whole bone marrow transplantation in RDEB has shown some benefit in children, as this leads to new C7 in the skin and fewer blisters. More recent studies have also indicated that a specific sub-population of bone marrow cells has the capacity to be recruited into damaged skin where they can change into skin cells and generate new C7. The type of cells that can do this are known as mesenchymal stromal cells (MSCs) which reside primarily in bone marrow. In other trials, these cells have been given in many other diseases and they appear to be safe to use. We propose to give these cells in children with RDEB to find out if they are safe in this genetic condition and whether they have an impact on disease severity and quality of life. We plan to recruit 6-10 children with RDEB, administer three infusions of MSCs and primarily assess safety and efficacy in this context. The clinical trial will take place at Great Ormond Street Hospital and King??s College London is the academic sponsor. The trial will last 2 years.

• REC name

  London - Bloomsbury Research Ethics Committee

• REC reference

  12/LO/1258

• Date of REC Opinion

  16 Nov 2012

• REC opinion

  Further Information Favourable Opinion