Ex Vivo Expanded Corneal Limbal Stem Cell Transplantation

• Research type

  Research Study

• Full title

  Pilot Clinical Assessment of Ex Vivo Expanded Corneal Limbal Stem Cell Transplantation in Patients with Severe Ocular Surface Diseases (OSD) Arising from Limbal Stem Cell Deficiency

• IRAS ID

  65461

• Contact name

  B Dhillon

• Contact email

  bal.dhillon@luht.scot.nhs.uk

• Sponsor organisation

  R&D Office

• Eudract number

  2010-024409-11

• ISRCTN Number

  To be applied for

• Research summary

  The cornea is the clear front of the eye and its clarity is vital for the transmission of light to the retina for visual perception. The surface of the cornea is made up of a multi-layered epithelium, which is maintained by adult stem cells located in the periphery of the cornea, in a region known as the limbus.

  Limbal stem cell deficiency (LSCD) is an irreversible disease resulting from the loss of these corneal epithelial stem cells, or limbal stem cells (LSC), and results in severe ocular surface disease (OSD) characterised by a thickened, irregular and unstable eplthelium. A variety of conditions can cause OSD from LSCD and patients face a very poor prognosis resulting in blindness, redness and pain. Aniridia is a primary stem cell disorder, but secondary disorders of the LSC are more common e.g. chemical or thermal burns, Stevens-Johnson syndrome or severe contact lens induced keratopathy.

  A novel way of treating total limbal stem cell deficiency is to expand putative limbal stem cells in culture and transplant them on amniotic membrane (AM) to the ocular surface. This is a pilot study to examine the safety and efficacy of this procedure. Patients will undergo an initial surgery to remove the diseased cornea and replace it with the investigational product. The effectiveness of the treatment will be evaluated over an 18 month follow-up period.

  The study aims to:-
  •Generate the data required for reliable sample size calculations for subsequent studies
  •Evaluate the practicalities and logistics of the study including recruitment process, follow-up, data collection and analysis

  It also aims to obtain preliminary answers to the following questions:-
  •Is the procedure feasible, efficient and safe and does it lead to improvements in vision and quality of the ocular surface?
  •How does immunosuppression and limbal stem cell transplantation compare with using immunosuppression and amniotic membrane alone?

• REC name

  East of Scotland Research Ethics Service REC 2

• REC reference

  14/ES/1008

• Date of REC Opinion

  22 Jun 2011

• REC opinion

  Further Information Favourable Opinion