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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 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: March 25, 2014
Last verified: March 2014

March 2, 2010
March 25, 2014
December 2010
December 2014   (final data collection date for primary outcome measure)
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 ]
Changes in 13C urea formation from baseline to 3 and 6 months after first HHLivC infusion [ Time Frame: Baseline to 3 and 6 months ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT01195753 on ClinicalTrials.gov Archive Site
Frequency and severity of metabolic crises [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Same as current
Not Provided
Not Provided
 
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.

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

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.

Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Urea Cycle Disorders
Biological: HHLivC
multiple infusion of liver cells
Experimental: Liver Cell Infusion
Intervention: Biological: HHLivC
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
40
December 2014
December 2014   (final data collection date for primary outcome measure)

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
Both
up to 5 Years
No
Contact: Rodney Monroy, PhD 1-443-910-2441 rodney.monroy@cytonetllc.com
Contact: Jörg Schommer, PhD +49 6201 259 8232 joerg.schommer@cytonet.de
United States,   Canada
 
NCT01195753
CCD05
Yes
Cytonet GmbH & Co. KG
Cytonet GmbH & Co. KG
Not Provided
Not Provided
Cytonet GmbH & Co. KG
March 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP