Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders (SELICAIII)
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| First Received Date ICMJE | March 2, 2010 | ||||||||
| Last Updated Date | November 13, 2012 | ||||||||
| Start Date ICMJE | December 2010 | ||||||||
| Estimated Primary Completion Date | July 2013 (final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
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 ] | ||||||||
| Original Primary Outcome Measures ICMJE |
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 ] | ||||||||
| Change History | Complete list of historical versions of study NCT01195753 on ClinicalTrials.gov Archive Site | ||||||||
| Current Secondary Outcome Measures ICMJE |
Frequency and severity of metabolic crises [ Time Frame: 6 months ] [ Designated as safety issue: Yes ] | ||||||||
| Original Secondary Outcome Measures ICMJE | Same as current | ||||||||
| Current Other Outcome Measures ICMJE | Not Provided | ||||||||
| Original Other Outcome Measures ICMJE | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title ICMJE | Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders | ||||||||
| Official Title ICMJE | 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. | ||||||||
| Brief Summary | Treatment with liver cell infusion for children with urea cycle disorders (UCD). |
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| 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. |
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| Study Type ICMJE | Interventional | ||||||||
| Study Phase | Phase 2 | ||||||||
| Study Design ICMJE | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
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| Condition ICMJE | Urea Cycle Disorders | ||||||||
| Intervention ICMJE | Biological: HHLivC
multiple infusion of liver cells |
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| Study Arm (s) | Experimental: Liver Cell Infusion
Intervention: Biological: HHLivC |
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| Publications * | 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. | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status ICMJE | Recruiting | ||||||||
| Estimated Enrollment ICMJE | 40 | ||||||||
| Estimated Completion Date | October 2013 | ||||||||
| Estimated Primary Completion Date | July 2013 (final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | ||||||||
| Ages | up to 5 Years | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts ICMJE |
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| Location Countries ICMJE | United States, Canada | ||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT01195753 | ||||||||
| Other Study ID Numbers ICMJE | CCD05 | ||||||||
| Has Data Monitoring Committee | Yes | ||||||||
| Responsible Party | Cytonet GmbH & Co. KG | ||||||||
| Study Sponsor ICMJE | Cytonet GmbH & Co. KG | ||||||||
| Collaborators ICMJE | Not Provided | ||||||||
| Investigators ICMJE | Not Provided | ||||||||
| Information Provided By | Cytonet GmbH & Co. KG | ||||||||
| Verification Date | November 2012 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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