Your journey
1What's a trial
4Get in touch
More info
You can access this
clinical trial
if you have
and you are
between 14 and 55
years old
This is a medium phase trial determining
the safety and dosage of the new treatment, as well as its efficacy.
Show me locations

The purpose

Human phenylketonuria (PKU) results from phenylalanine hydroxylase (PAH) deficiency, and represents one of the most common and extensively studied single-gene Mendelian disorders in humans. Unfortunately, optimum clinical outcome demands lifelong dietary restriction through adherence to an unpalatable and expensive artificial diet. Challenges in maintaining traditional therapy lead to increasing phenylalanine (Phe) levels in patients as they approach adulthood with an incumbent severe burden of psychosocial and intellectual difficulties. The recent introduction of the new medication Sapropterin for treatment of PKU has improved Phe control and dietary tolerance in some patients, but at enormous cost to patients and insurers for the FDA designated orphan product. Thus, there is an unmet need for novel therapies to correct PKU. PAH is almost exclusively expressed in the liver in humans. The main objective of the current proposal is to examine the safety and efficacy of hepatocyte transplantation in patients with PKU.

Provided treatments

  • Radiation: Preparative Radiation Therapy
  • Procedure: Hepatocyte Transplant
  • Drug: Immunosuppression
  • Other: Liver Evaluation
  • Behavioral: Neuro-psychological Assessment
  • DiagnosticTest: Whole body Phe oxidation testing
  • Procedure: Liver Biopsy
Wondering what is the locations section for?
We will connect you to the doctor responsible for this trial and help you get more information.

Locations near you

Unfortunately, there are no recruiting locations near you. Please check the list with all locations below.
Tris trial is registered with FDA with number: NCT01465100. The sponsor of the trial is Ira Fox and it is looking for 10 volunteers for the current phase.
Official trial title:
Hepatocyte Transplantation for Phenylketonuria