Fibroblast cell therapy for RDEB - a phase II clinical trial

  • Research type

    Research Study

  • Full title

    A prospective placebo controlled phase II study to evaluate the use of allogeneic fibroblasts for the treatment of skin erosions in recessive dystrophic epidermolysis bulllosa.

  • IRAS ID

    64567

  • Contact name

    John Alexander McGrath

  • Eudract number

    2010-023121-38

  • ISRCTN Number

    Pending

  • Research summary

    This research proposal concerns individuals with the inherited blistering skin disease known as recessive dystrophic epidermolysis bullosa (RDEB). This is a very severe condition in which skin injury leads to blisters and wounds that remain inflamed and do not heal. The entire skin and internal organs can be affected. The increased skin fragility can lead to scarring (especially of the hands and feet) and an increased risk of skin cancer at a young age. Currently we have no effective treatments for RDEB and we are unable to prevent the disease complications. We have recently shown that cultured skin cells called fibroblasts from unrelated donors (allogeneic) have the potential to improve skin strength and function when injected into the skin of individuals with RDEB. We have also discovered that injecting the same fibroblasts into the edges of chronic wounds in people with RDEB can stimulate wound healing, lessen the need for dressings and improve quality of life. Nevertheless, use of a potential ''growth factor bullet'' to speed up wound healing and skin function in RDEB is not yet ready or necessary appropriate for clinical application and therefore a phase II clinical trial of allogeneic fibroblast therapy in RDEB represents the next feasible option in driving forward research for this inherited disease. We plan to recruit 25 people with RDEB (up to 40 may be needed) in order to treat 20 and study an average of 6 wounds per individual with a single treatment of allogeneic fibroblasts or placebo (inactive solution) injections into the skin. The clinical trial will take place within King’s College London and The Guy’s and St Thomas’ NHS Foundation Trust, which is home to the UK’s National Epidermolysis Bullosa clinical and diagnostic services. The trial is expected to last 7 months.

  • REC name

    London - Dulwich Research Ethics Committee

  • REC reference

    10/H0808/146

  • Date of REC Opinion

    2 Dec 2010

  • REC opinion

    Favourable Opinion