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| Sponsor: | Cytonet GmbH & Co. KG |
|---|---|
| Information provided by: | Cytonet GmbH & Co. KG |
| ClinicalTrials.gov Identifier: | NCT01195753 |
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. |
| Estimated Enrollment: | 40 |
| Study Start Date: | December 2010 |
| Estimated Study Completion Date: | October 2013 |
| Estimated Primary Completion Date: | July 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Liver Cell Infusion |
Biological: HHLivC
multiple infusion of liver cells
|
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 |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Rodney Monroy, PhD | 1-443-910-2441 | rodney.monroy@cytonetllc.com |
| Contact: Heinz Kriegbaum, PhD | +49 6201 259 816 | heinz.kriegbaum@cytonet.de |
| 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, 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 | |
More Information
| Responsible Party: | Dr. Heinz Kriegbaum/Head of R&D, Cytonet GmbH & Co.KG |
| ClinicalTrials.gov Identifier: | NCT01195753 History of Changes |
| Other Study ID Numbers: | CCD05 |
| Study First Received: | March 2, 2010 |
| Last Updated: | February 13, 2012 |
| Health Authority: | United States: Food and Drug Administration |
|
Urea cycle Disorders |
|
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 |
