251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details
臨床試験数 : 54 / 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
Showing 1 to 10 of 10 diseases
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05412160 (ClinicalTrials.gov) | January 18, 2023 | 6/6/2022 | Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD: a Multicenter, Randomized, Controlled, Proof of Concept Study Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD: a Multic ... | Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD: a Multicenter, Randomized, Controlled, Proof of Concept Study Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD: a Multic ... | COPD;Dyspnea;Argininemia | Drug: Arginine;Drug: Placebo | University of Milan | NULL | Recruiting | 40 Years | N/A | All | 152 | N/A | Italy |
| 2 | EUCTR2014-000650-11-ES (EUCTR) | 13/04/2015 | 20/10/2014 | HEP002 is a clinical study designed for paediatric patients with urea cycle disorders (UCD). UCD patients will receive several infusions of HepaStem. The efficacy as well as the safety of the medicinal product will be assessed during the year following infusions. HEP002 is a clinical study designed for paediatric patients with urea cycle disorders (UCD). UCD pat ... | A prospective, open label, multi-country, multicenter, efficacy and safety study of several infusions of Promethera HepaStem in Urea Cycle Disorders pediatric patients A prospective, open label, multi-country, multicenter, efficacy and safety study of several infusion ... | The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are eight disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia type I, argininosuccinic aciduria, argininemia, HHH syndrme and citrullinemia type II.Mutations in each of the enzymes have been identified, resulting in failed production of urea, the end product of the urea cycle. MedDRA version: 18.0;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into u ... | Product Name: HepaStem INN or Proposed INN: Celulas hepatocitos troncales adultas alogenicas de higado expandidas Product Name: HepaStem INN or Proposed INN: Celulas hepatocitos troncales adultas alogenicas de higad ... | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | France;Poland;Belgium;Spain | ||
| 3 | EUCTR2013-001045-14-GB (EUCTR) | 30/07/2014 | 28/04/2014 | A long-term safety follow-up study of patients having received infusions of HepaStem. | SAF 001: A long-term safety follow-up study of patients having received infusions of HepaStem. - SAF001 SAF 001: A long-term safety follow-up study of patients having received infusions of HepaStem. - SAF ... | Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The UCD are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are 8 disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia I&II, argininosuccinic aciduria, HHH syndrome & argininemia MedDRA version: 19.0;Level: LLT;Classification code 10021601;Term: Inborn error of metabolism NOS;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucur ... | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Ot ... | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 19 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ... | Portugal;Belgium;Italy;United Kingdom | ||
| 4 | EUCTR2013-001045-14-PT (EUCTR) | 04/04/2014 | 02/10/2013 | A clinical study for evaluating long-term safety of patients suffering from Urea Cycle disorders (UCD) or Crigler-Najjar Syndrome (CN) having received infusions of HepaStem. A clinical study for evaluating long-term safety of patients suffering from Urea Cycle disorders (UC ... | SAF 001: A long-term safety follow-up study of patients suffering from Urea Cycle disorders (UCD) or Crigler-Najjar Syndrome (CN) having received infusions of HepaStem. - SAF001 SAF 001: A long-term safety follow-up study of patients suffering from Urea Cycle disorders (UCD) or ... | Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The UCD are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are 8 disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia I&II, argininosuccinic aciduria, HHH syndrome & argininemia MedDRA version: 16.0;Level: LLT;Classification code 10021601;Term: Inborn error of metabolism NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucur ... | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Ot ... | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 1;Phase 2 | Portugal;Belgium;Israel;United Kingdom;Italy | ||
| 5 | EUCTR2013-001045-14-IT (EUCTR) | 14/02/2014 | 31/12/2013 | A clinical study for evaluating long-term safety of patients having received infusions of HepaStem. | SAF 001: A long-term safety follow-up study of patients having received infusions of HepaStem. - SAF001 SAF 001: A long-term safety follow-up study of patients having received infusions of HepaStem. - SAF ... | Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The UCD are inborn errors of metabolism that affect the transfer of nitrogen into urea. Thereare 8 disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia I&II, argininosuccinic aciduria, HHH syndrome & argininemia MedDRA version: 16.1;Level: LLT;Classification code 10021601;Term: Inborn error of metabolism NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucur ... | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Ot ... | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 1;Phase 2 | Portugal;Belgium;Israel;United Kingdom;Italy | ||
| 6 | EUCTR2013-001045-14-BE (EUCTR) | 22/04/2013 | 19/03/2013 | A clinical study for evaluating long-term safety of patients suffering from Urea Cycle disorders (UCD) or Crigler-Najjar Syndrome (CN) having received infusions of HepaStem. A clinical study for evaluating long-term safety of patients suffering from Urea Cycle disorders (UC ... | SAF 001: A long-term safety follow-up study of patients suffering from Urea Cycle disorders (UCD) or Crigler-Najjar Syndrome (CN) having received infusions of HepaStem. - SAF001 SAF 001: A long-term safety follow-up study of patients suffering from Urea Cycle disorders (UCD) or ... | Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are six disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia, argininosuccinic aciduria and argininemia MedDRA version: 15.1;Level: LLT;Classification code 10021601;Term: Inborn error of metabolism NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucur ... | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Ot ... | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 1;Phase 2 | Portugal;Belgium;United Kingdom;Italy | ||
| 7 | EUCTR2011-004074-28-IT (EUCTR) | 19/12/2012 | 14/01/2013 | A phase I/II trial for evaluating both safety and preliminary efficacy of one cycle of Promethera Hepastem in Urea Cycle Disorders and Crigler-Najjar Syndrome patients A phase I/II trial for evaluating both safety and preliminary efficacy of one cycle of Promethera He ... | A prospective, open label, multicenter, partially randomized, safety study of one cycle of Promethera HepaStem in Urea Cycle Disorders and Crigler-Najjar Syndrome Patients - HEP 001 A prospective, open label, multicenter, partially randomized, safety study of one cycle of Promether ... | Crigle-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucoronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are six desorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia, argininosuccinic aciduria and argininemia. MedDRA version: 15.1;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism;System Organ Class: 100000004861 MedDRA version: 15.1;Level: PT;Classification code 10011386;Term: Crigler-Najjar syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 15.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;Classification code 10021601;Term: Inborn error of metabolism NOS;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Crigle-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucoro ... | Product Name: Promethera HepaStem Product Code: HHALPC INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells (HHALPC) Product Name: Promethera HepaStem Product Code: HHALPC INN or Proposed INN: Heterologous Human Adult L ... | PROMETHERA BIOSCIENCES | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 1/2 | Belgium;United Kingdom;Italy | ||
| 8 | EUCTR2011-004074-28-GB (EUCTR) | 20/07/2012 | 28/09/2011 | A phase I/II trial for evaluating both safety and preliminary efficacy of one cycle of Promethera HepaStem® in Urea Cycle Disorders and Crigler-Najjar Syndrome patients A phase I/II trial for evaluating both safety and preliminary efficacy of one cycle of Promethera He ... | A prospective, open label, multicenter, randomized, safety and preliminary efficacy study of one cycle of Promethera HepaStem® in Urea Cycle Disorders and Crigler-Najjar Syndrome patients - UKHEP001 A prospective, open label, multicenter, randomized, safety and preliminary efficacy study of one cyc ... | Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are six disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia, argininosuccinic aciduria and argininemia MedDRA version: 17.0;Level: LLT;Classification code 10021601;Term: Inborn error of metabolism NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucur ... | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Ot ... | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 1/2 | Belgium;Italy;United Kingdom | ||
| 9 | EUCTR2011-004074-28-BE (EUCTR) | 13/02/2012 | 21/02/2012 | A phase I/II trial for evaluating safety of one cycle of Promethera HepaStem in Urea Cycle Disorders and Crigler-Najjar Syndrome patients A phase I/II trial for evaluating safety of one cycle of Promethera HepaStem in Urea Cycle Disorders ... | HEP 001: A prospective, open label, multicenter, partially randomized, safety study of one cycle of Promethera HepaStem in Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) paediatric patients. - HEP 001 HEP 001: A prospective, open label, multicenter, partially randomized, safety study of one cycle of ... | Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are six disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia, argininosuccinic aciduria and argininemia MedDRA version: 14.1;Level: LLT;Classification code 10021601;Term: Inborn error of metabolism NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucur ... | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Ot ... | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 1/2 | Belgium;Italy;United Kingdom | ||
| 10 | NCT00345605 (ClinicalTrials.gov) | February 2008 | 26/6/2006 | Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder | A Randomized, Double-Blind, Crossover Study of Sodium Phenylbutyrate and Low-Dose Arginine Compared to High-Dose Arginine Alone on Liver Function, Ureagenesis and Subsequent Nitric Oxide Production in Patients With Argininosuccinic Aciduria A Randomized, Double-Blind, Crossover Study of Sodium Phenylbutyrate and Low-Dose ArginineCompared t ... | Argininosuccinic Aciduria;Amino Acid Metabolism, Inborn Errors;Urea Cycle Disorders | Drug: Sodium Phenylbutyrate;Drug: Arginine | Brendan Lee | Office of Rare Diseases (ORD);Rare Diseases Clinical Research Network;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Office of Rare Diseases (ORD);Rare Diseases Clinical Research Network;Eunice Kennedy Shriver Nationa ... | Completed | 5 Years | N/A | All | 12 | Phase 2 | United States |