UKHEP001

• Research type

  Research Study

• Full title

  UKHEP 001 UKHEP001: A prospective, open label, multicenter, partially randomized, safety study of one cycle of Promethera HepaStem in Urea Cycle Disorders and Crigler-Najjar Syndrome patients

• IRAS ID

  91039

• Contact name

  Patrick Mckiernan

• Sponsor organisation

  Promethera Biosciences

• Eudract number

  2011-004074-28

• ISRCTN Number

  n/a

• Research summary

  Crigler Najjar Syndrome (CN), and urea cycle disorders (UCD) are metabolic diseases, where a specific enzyme is missing in the liver. Waste products that are normally removed from the blood, and converted in less toxic substances in the liver, build up in the blood. This lead to severe symptoms such as brain-, muscle-, and nerve damage, and is the cause of severe brain damage and death among newborns and infants. There is no cure. Adult stem cells are found in a variety of tissues in the body, and also in the liver. They are basic, immature cells which act as a repair and replacement system for the body. In this study we give stem cells isolated from a donated liver that could not be used for transplantation. We expect that these cells, called HepaStem, will multiply in the liver of the patient. Since the new cells do have all the enzymes that the liver needs to remove waste products from the blood, the waste products can be removed from the body. We will first assess if the cell infusion is safe in children, and what cell number is needed to be safe, but improve the condition of the patients. We will infuse the cells directly into the blood vessel of the liver. We have already seen in three patients with metabolic diseases that the technique is safe. However, not enough patients have been treated to tell what the optimal amount of cells is. The safety will be assessed at several intervals: weekly, biweekly, and monthly at 3, 4, 5, 6, 8, 10, and 12 months. The study will include enough patients to tell whether the cell infusion is safe, and at which number. It will also already indicate if HepaStem can improve the health of the children.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  12/LO/0564

• Date of REC Opinion

  20 Jul 2012

• REC opinion

  Further Information Favourable Opinion