A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients

Summary

This is a phase2, prospective, open label study designed to investigate the safety and efficacy of several infusions of HepaStem. This study will include 5 pediatric Urea Cycle Disorder (UCD) patients under 12 years old. Its assessment includes all safety parameters and an efficacy assessment based on 13C tracer tests, ammonia, medication and diet changes. HepaStem will be administered in addition to the conventional UCD treatments.

Timeline

Start

2018-07-12

Primary completion

2020-11-04

Completion

2020-11-04

Outcome

Outcome not reported

Stopped (Enrollment): “Sponsor decided to stop enrollment”

Drugs

Indications