Hepatocyte Transplantation for Liver Based Metabolic Disorders
|ClinicalTrials.gov Identifier: NCT01345578|
Recruitment Status : Suspended (seeking additional funding)
First Posted : May 2, 2011
Last Update Posted : January 19, 2018
|Condition or disease||Intervention/treatment||Phase|
|Metabolic Diseases||Drug: human hepatocyte transplantation Radiation: Preparative Radiation Therapy||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Hepatocyte Transplantation for Liver Based Metabolic Disorders|
|Study Start Date :||March 2011|
|Estimated Primary Completion Date :||October 2018|
|Estimated Study Completion Date :||January 2019|
Experimental: Hepatocyte Transplantation
Drug: human hepatocyte transplantation
Transplantation of hepatocytes into the liver will be through the portal vein. The portal vein will be accessed transhepatically, by umbilical vein, or surgically by a peripheral mesenteric vein.
The subject will be evaluated de novo and if they are a candidate for orthotopic liver transplantation they will receive the transplant. Even if the subject receives the hepatocyte transplant and it does not work, they will be evaluated for orthotopic liver transplantation as if they never received the hepatocyte transplant.
If at 6 months we see an improvement in disease, we will recommend a re-transplantation with a goal of complete correction of disease and until the subject is no longer required to be a candidate for organ transplantation. Subjects will be re-evaluated every 6 months for re-transplantation. Subjects will remain on the waiting list for organ transplantation. Further radiation therapy will not be needed prior to re-transplantation.
Other Name: hepatocyte transplantRadiation: Preparative Radiation Therapy
Just prior to the hepatocyte transplant, a portion of the right hepatic lobe comprising between 35-50% of the entire liver volume will be irradiated to a dose of 7.5-10 Gy in a single fraction using a linear accelerator-based stereotactic radiosurgery system with intensity-modulated radiation therapy planning (IMRT).
- Improvement in enzyme physiologic function at 6 months [ Time Frame: 6 months post hepatocyte transplant ]After infusing donor allogeneic hepatocytes through the portal vein following preparative hepatic irradiation, improvement in enzyme physiologic function will be assessed at 6 months.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01345578
|United States, Pennsylvania|
|Children's Hospital of Pittsburgh of UPMC|
|Pittsburgh, Pennsylvania, United States, 15201|
|Principal Investigator:||Ira J Fox, MD||University of Pittsburgh|