Hepatocyte Transplantation for Liver Based Metabolic Disorders

• Status: Suspended
• Trial end date: 2022-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this research study is to determine whether partial irradiation of the liver and liver cell transplantation can provide help for patients with life-threatening liver-based metabolic diseases who are unlikely to survive without extensive medical therapy or transplant. The goal of this research study is to determine if liver cell transplants can be effective as an alternative to organ transplantation. At the present time, liver cell transplants are experimental and have been done in a limited number of human subjects.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ira Fox; University of Pittsburgh

Criteria

Inclusion Criteria:

• Patients will have life-threatening liver-based metabolic disorders who are candidates for organ transplantation where hepatocyte transplantation is considered theoretically curative.

• In addition to child subjects less than 18 years of age, for purposes of this protocol, adults up to age 21 years will be enrolled since this is the upper age limit of patients which are seen at Children's Hospital of Pittsburgh.

Exclusion Criteria:

• Patients with liver based metabolic disorders not theoretically treatable with organ transplantation.

• Subjects who meet any of the following criteria will be excluded from participation in this protocol:

1. Subject has active malignancy.

2. Subject has allergy to immunosuppression medications that are required post transplant procedure for the prevention of rejection.

3. Subject has sepsis, pneumonia, other active infection or other secondary life-threatening organ dysfunction.

4. Significant liver fibrosis determined by biopsy (if clinically indicated). Significant liver fibrosis will be defined by the Ishak Staging, Stage 5: bridges with occasional nodules.

5. Subject is pregnant or breastfeeding.