ADSTEM

• Research type

  Research Study

• Full title

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

• IRAS ID

  162879

• Contact name

  John McGrath

• Contact email

  john.mcgrath@kcl.ac.uk

• Sponsor organisation

  King's College London University

• Eudract number

  2014-004500-30

• Duration of Study in the UK

  2 years, 0 months, 1 days

• Research summary

  Introduction
  This is a research study to evaluate whether mesenchymal stromal (stem) cells can modify disease severity in patients with a rare severe inherited fragility skin disease called Recessive Dystrophic Epidermolysis Bullosa (RDEB). Mesenchymal stromal or stem cells (MSCs) come from the bone marrow of healthy donors and have the ability to self-renew and differentiate into various cell types. RDEB is caused by a genetic defect causing a lack in collagen VII. This protein ‘sticks’ the top and bottom layer of the skin together.
  This current research project aims to find out if using MSCs can modify disease by setting off an inflammatory response. It is hoped that this anti-inflammatory effect will help improve the skin in these patients by reducing the appearance of blisters and improve wound healing.

  Methods
  This is a DebRA (Dystrophic Epidermolysis Bullosa Research Association) funded study. Ten patients will be recruited from the Guy's and St Thomas' NHS Trust. These patients will have a diagnosis of RDEB. Participants will be screened to ensure suitability for the Clinical Trial. Once deemed suitable they will have a blood sample taken to screen for infections and perform routine blood tests. They will also have a skin biopsy taken. A skin biopsy involves taking a sample of skin under local anaesthetic to assess degree of collagen VII abnormality. Two infusions of MSCs will be given through the vein two weeks apart (Day 0 and Day 14) through a drip. The participant will be reviewed on Day 28, 60, 100, Month 6 and Month 12. At each of these 8 review dates, blood samples will be taken and skin biopsies taken at 6 visits. At each visit, except the days of MSC infusion, quality of life and skin severity scores will be completed.

• REC name

  North East - York Research Ethics Committee

• REC reference

  15/NE/0006

• Date of REC Opinion

  14 Jan 2015

• REC opinion

  Favourable Opinion