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Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders (SELICAIII)

This study is currently recruiting participants.
Verified November 2012 by Cytonet GmbH & Co. KG
Sponsor:
Information provided by (Responsible Party):
Cytonet GmbH & Co. KG
ClinicalTrials.gov Identifier:
NCT01195753
First received: March 2, 2010
Last updated: November 13, 2012
Last verified: November 2012
History of Changes
  Purpose

Treatment with liver cell infusion for children with urea cycle disorders (UCD).


Condition Intervention Phase
Urea Cycle Disorders
 Biological: HHLivC
 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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.

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Cytonet GmbH & Co. KG:

Primary Outcome Measures:
  • Changes in 13C urea formation from baseline to 2 and 4 months after first HHLivC infusion [ Time Frame: Baseline to 2 and 4 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Frequency and severity of metabolic crises [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 40
Study Start Date: December 2010
Estimated Study Completion Date: October 2013
Estimated Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Liver Cell Infusion Biological: HHLivC
multiple infusion of liver cells

Detailed Description:

Urea cycle disorders are rare inherited diseases that generally have a poor outcome, especially with onset of the disease in the neonatal period. UCDs are caused by a deficiency of one of six enzymes responsible for removing ammonia from the bloodstream. Instead of being converted into urea which is removed from the body with the urine, ammonia accumulates in UCD patients leading to brain damage or death. In the light of a mortality rate of > 50% at the age of 10 years the current pharmacological and dietary therapy is of modest success. Furthermore, mental retardation, cerebral palsy and other neurological sequelae are common among surviving patients.

In the last years, orthotopic liver transplantation (OLT) has become the best therapeutic option for UCD with long-term survival rates of about 90%. However, in the first weeks of life OLT still is technically demanding and prone to complications. With larger size of the recipient, the technical problems with OLT decrease considerably. The increased body weight usually achieved at the age of more than 8 weeks is related to a major reduction in transplantation related morbidity. Stabilization of metabolism until the patient can undergo OLT is essential.

In this study, young children with UCD will be treated by repetitive application of human liver cells. In the last consequence, the aim of this new therapy option is to supply a sufficient amount of healthy liver cells to compensate for the metabolic defect and to reduce the risk of neurological deterioration while awaiting OLT.

  Eligibility

Ages Eligible for Study:   up to 5 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age: birth up to 5 years of age
  • Ornithine transcarbamylase deficiency [OTCD], Carbamyl phosphate synthetase I deficiency [CPSD], Argininosuccinate synthetase deficiency [Citrullinaemia]
  • Written Informed Consent

Exclusion Criteria:

  • Weight ≤ 3.5 kg
  • Presence of acute infection at the time of inclusion
  • Severe chronic or systemic disease other than study indication
  • Structural liver disease (eg, cirrhosis, portal hypertension)
  • Required valproate therapy
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01195753

Contacts
Contact: Rodney Monroy, PhD 1-443-910-2441 rodney.monroy@cytonetllc.com
Contact: Jörg Schommer, PhD +49 6201 259 8232 joerg.schommer@cytonet.de

Locations
United States, California
David Geffen Schoool of Medicine at UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Gerald Lipshutz, MD         GLipshutz@mednet.ucla.edu    
Principal Investigator: Gerald Lipshutz, MD            
Stanford University Recruiting
Palo Alto, California, United States, 94304
Contact: Andrew Bonham, MD         cbonham@stanford.org    
Principal Investigator: Andrew Bonham, MD            
University of California Recruiting
San Diego, California, United States, 92103
Contact: Bruce Barshop, MD         bbarshop@ucsd.edu    
Principal Investigator: Bruce Barshop, MD            
United States, Connecticut
Yale University Recruiting
New Haven, Connecticut, United States, 06250
Contact: Sukru Emre, MD         sukru.emre@yale.edu    
Principal Investigator: Sukru Emre, MD            
United States, District of Columbia
Children's National Medical Center and Georgetown University Medical Center Recruiting
Washington, District of Columbia, United States, 20010
Contact: Uta Lichter, MD         ulichter@cnmc.org    
Principal Investigator: Uta Lichter, MD            
United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Miriam Vos, MD         mvos@emory.edu    
Principal Investigator: Miriam Vos, MD            
United States, Illinois
Children's Memorial Hospital Recruiting
Chicago, Illinois, United States, 60614
Contact: Barbara Burton, MD         bburton@childrensmemorial.org    
Principal Investigator: Barbara Burton, MD            
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Susan Berry, MD         berry002@umn.edu    
Principal Investigator: Susan Berry, MD            
United States, New York
Mount Sinai School of Medicine Recruiting
New York, New York, United States, 10029
Contact: George Diaz, MD         george.diaz@mssm.edu    
Principal Investigator: George Diaz, MD            
United States, Oregon
Oregon Health Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Cary Harding, MD         hardingc@ohsu.edu    
Principal Investigator: Cary Harding, MD            
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: John Lawrence Merritt III, MD         lawrence.merritt@seattlechildrens.org    
Principal Investigator: John Lawrence Merritt III, MD            
United States, Wisconsin
Children's Hospital of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: David Dimmock, MD         ddimmock@mcw.edu    
Principal Investigator: David Dimmock, MD            
Canada, Alberta
Alberta Children's Hospital Recruiting
Calgary, Alberta, Canada, T3B 6A8
Contact: Aneal Khan, MD         khaa@ucalgary.ca    
Principal Investigator: Aneal Khan, MD            
Sponsors and Collaborators
Cytonet GmbH & Co. KG
  More Information

Publications:

Responsible Party: Cytonet GmbH & Co. KG
ClinicalTrials.gov Identifier: NCT01195753     History of Changes
Other Study ID Numbers: CCD05
Study First Received: March 2, 2010
Last Updated: November 13, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Cytonet GmbH & Co. KG:
Urea cycle Disorders

Additional relevant MeSH terms:
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases

ClinicalTrials.gov processed this record on May 23, 2013