Limbal Stem Cell Deficiency – Allogeneic
We are recruiting patients with limbal stem cell deficiency (LSCD) for an international multicentric phase I/IIa clinical trial.
The aim of the trial is to assess the efficacy and safety of allogeneic ABCB5-positive limbal stem cells (ABCB5+ LSCs) in patients suffering from LSCD. It is examined whether application of ABCB5+ LSCs can result in regeneration of an intact cornea free from blood vessels. Primary safety endpoint is the occurrence of adverse events.
The trial has been approved by the German Paul Ehrlich Institute (PEI) and the U.S. Food and Drug Administration (FDA) as the competent national regulatory authorities. The European Medicines Agency (EMA) has granted RHEACELL’s LSC product EU Orphan Drug Designation for the treatment of LSCD. The product has also received Fast-Track-Designation in the USA granted by the FDA.
For this trial 16 patients will be recruited in Germany and the USA.
For the manufacturing process of the allogeneic ABCB5+ LSCs we are using a patent hold by the Brigham and Women’s Hospital, Boston/USA, and the Children’s Hospital, Boston/USA, in cooperation with our scientific partner Prof. Dr. Markus Frank.
More information about the trial can be found HERE (ClinicalTrials.gov).
Background information about the use of ABCB5+ LSCs in LSCD can be found HERE.