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Cell Therapy

Hepatocyte Transplant for Phenylketonuria

Phase 1 & 2
Waitlist Available
Led By Ira J Fox, MD
Research Sponsored by Ira Fox
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

Summary

This trial uses hepatocyte transplantation to help patients with PKU. Healthy liver cells from donors are transplanted into the patient's liver to help it process phenylalanine properly, which could improve their health. This method has been proposed as an alternative to traditional liver transplantation to treat metabolic liver diseases.

Eligible Conditions
  • Phenylketonuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Improvement/reversal of characteristics of PKU
Secondary outcome measures
Adaptive Behavior Assessment System-third edition (ABAS-III)
Beck Depression Inventory
Behavior Assessment System for Children-second edition (BASC-II)
+7 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Hepatocyte TransplantationExperimental Treatment7 Interventions
See Below.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Liver Biopsy
2015
N/A
~270

Find a Location

Who is running the clinical trial?

Ira FoxLead Sponsor
2 Previous Clinical Trials
5 Total Patients Enrolled
Ira J Fox, MDPrincipal InvestigatorUniversity of Pittsburgh
2 Previous Clinical Trials
5 Total Patients Enrolled
~0 spots leftby Sep 2025