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| Sponsor: | Cytonet GmbH & Co. KG |
|---|---|
| Information provided by (Responsible Party): | Cytonet GmbH & Co. KG |
| ClinicalTrials.gov Identifier: | NCT00718627 |
Purpose
Urea cycle disorders are rare inherited diseases that generally have a poor outcome. In this study, neonates and infants with UCD will be included within the first 3 months of life and will be treated by repetitive application of human liver cells to reduce the risk of neurological deterioration while awaiting OLT.
| Condition | Intervention | Phase |
|---|---|---|
|
Urea Cycle Disorders Carbamoylphosphate Synthetase I Deficiency Disease Ornithine Transcarbamylase Deficiency Disease Citrullinemia |
Biological: Human Heterologous Liver Cells |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children With Urea Cycle Disorders (UCDs) |
| Estimated Enrollment: | 15 |
| Study Start Date: | July 2008 |
| Estimated Study Completion Date: | September 2012 |
| Estimated Primary Completion Date: | September 2012 (Final data collection date for primary outcome measure) |
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, neonates and infants with UCD will be included within the first 3 months of life and 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 |
Inclusion Criteria:
Biochemically proven urea cycle disorder with deficiency of either:
Exclusion Criteria:
Contacts and Locations| Contact: Martin Lindner, MD | +49 6221 56 ext 2311 | Martin.Lindner@med.uni-heidelberg.de |
| Germany | |
| Allgemeine Pädiatrie, Pädiatrische Stoffwechselmedizin | Recruiting |
| Charité Universitätsmedizin Berlin, Berlin, Germany, 13353 | |
| Contact: Julia Hennermann, MD julia.hennermann@charite.de | |
| Principal Investigator: Julia Hennermann, MD | |
| University Children's Hospital, Heinrich-Heine University | Recruiting |
| Düsseldorf, Germany, 40225 | |
| Principal Investigator: Ute Spiekerkötter, Prof., MD | |
| Hannover Medical School, Children's Hospital | Recruiting |
| Hannover, Germany, 30625 | |
| Principal Investigator: Anibh M. Das, Prof., MD | |
| University Children's Hospital | Recruiting |
| Heidelberg, Germany, D-69120 | |
| Principal Investigator: Georg F. Hoffmann, Prof., MD | |
| Universitätsklinikum Münster | Recruiting |
| Münster, Germany, 48149 | |
| Contact: Thorsten Marquardt, Prof. Dr. med. +49251-83 ext 48002 thorsten.marquardt@ukmuenster.de | |
| Principal Investigator: Thorsten Marquardt, Prof., MD | |
| Principal Investigator: | Georg F. Hoffmann, MD | University Children's Hospital, Heidelberg |
More Information
| Responsible Party: | Cytonet GmbH & Co. KG |
| ClinicalTrials.gov Identifier: | NCT00718627 History of Changes |
| Other Study ID Numbers: | CCD02, SELICA V, 2006-000136-27 |
| Study First Received: | July 16, 2008 |
| Last Updated: | January 25, 2012 |
| Health Authority: | Germany: Paul-Ehrlich-Institut |
|
Urea cycle Disorders Carbamoylphosphate synthetase I deficiency Ornithine transcarbamoylase deficiency Argininosuccinate synthase deficiency Citrullinemia |
newborns infants liver cell transplantation liver cell infusion |
|
Deficiency Diseases Carbamoyl-Phosphate Synthase I Deficiency Disease Urea Cycle Disorders, Inborn Citrullinemia Ornithine Carbamoyltransferase Deficiency Disease Malnutrition Nutrition Disorders 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 Mitochondrial Diseases Genetic Diseases, X-Linked |
