Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Open, Prospective, Historic-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Infusion of Liver Cell Suspension (HHLivC) in Children With Urea Cycle Disorders.
1 other identifier
interventional
10
2 countries
5
Brief Summary
Treatment with liver cell infusion for children with urea cycle disorders (UCD).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Dec 2010
Longer than P75 for phase_2
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 2, 2010
CompletedFirst Posted
Study publicly available on registry
September 6, 2010
CompletedStudy Start
First participant enrolled
December 1, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2015
CompletedFebruary 8, 2016
February 1, 2016
5 years
March 2, 2010
February 5, 2016
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Changes in 13C urea formation from baseline to 2 and 4 months after first HHLivC infusion
Baseline to 2 and 4 months
Secondary Outcomes (1)
Frequency and severity of metabolic crises
6 months
Study Arms (1)
Liver Cell Infusion
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- Age: birth up to 5 years of age
- Ornithine transcarbamylase deficiency \[OTCD\], Carbamyl phosphate synthetase I deficiency \[CPSD\], Argininosuccinate synthetase deficiency \[ASSD, Citrullinaemia\]
- Written Informed Consent
You may not qualify if:
- Weight ≤ 3.5 kg
- Severe chronic or systemic disease other than study indication
- Structural liver disease (eg, cirrhosis, portal hypertension)
- Required valproate therapy
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (5)
Stanford University
Palo Alto, California, 94304, United States
University of California
San Diego, California, 92103, United States
Yale University
New Haven, Connecticut, 06250, United States
Children's Memorial Hospital
Chicago, Illinois, 60614, United States
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8, Canada
Related Publications (1)
Meyburg J, Das AM, Hoerster F, Lindner M, Kriegbaum H, Engelmann G, Schmidt J, Ott M, Pettenazzo A, Luecke T, Bertram H, Hoffmann GF, Burlina A. One liver for four children: first clinical series of liver cell transplantation for severe neonatal urea cycle defects. Transplantation. 2009 Mar 15;87(5):636-41. doi: 10.1097/TP.0b013e318199936a.
PMID: 19295306BACKGROUND
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 2, 2010
First Posted
September 6, 2010
Study Start
December 1, 2010
Primary Completion
December 1, 2015
Study Completion
December 1, 2015
Last Updated
February 8, 2016
Record last verified: 2016-02