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

NCT03884959 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2020-11-06

No results posted yet for this study

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.

Conditions

  • Urea Cycle Disorder

Interventions

BIOLOGICAL

HepaStem Infusion

HepaStem will be infused intravenously into the portal vein, either (1) via a permanent mesenteric PAC inserted surgically in an affluent of the portal vein; or (2) through a transient percutaneous transhepatic catheter inserted in to the portal vein under radio guidance.

Sponsors & Collaborators

  • HLB Cell Co., Ltd.

    lead INDUSTRY

Principal Investigators

  • Sanghoon Lee, MD. Ph.D · Samsung Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-07-12
Primary Completion
2020-11-04
Completion
2020-11-04

Countries

  • South Korea

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03884959 on ClinicalTrials.gov