HepaStem
( DrugBank: - / KEGG DRUG: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 251 | 尿素サイクル異常症 | 12 |
251. 尿素サイクル異常症
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 12 trials
| 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 | NCT03884959 (ClinicalTrials.gov) | July 12, 201820180712 | 3/3/201920190303 | A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients | A Prospective, Open Label, Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients A Prospective, Open Label, Safety and Efficacy Study of Infusions of HepaStemin Urea Cycle Disorders ... | Urea Cycle Disorder | Biological: HepaStem Infusion | HLB Cell Co., Ltd. | NULL | Withdrawn | N/A | 12 Years | All | 0 | Phase 2 | Korea, Republic of |
| 2 | EUCTR2014-000650-11-PL (EUCTR) | 11/12/201520151211 | 01/10/201520151001 | 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 ... | HEP002: A prospective, open label, multi-country, efficacy and safety study of several infusions of HepaStem in Urea Cycle Disorders pediatric patients. - HEP002 HEP002: A prospective, open label, multi-country, efficacy and safety study of several infusions of ... | The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are five disorders to be investigated: carbamoylphosphate synthetase I deficiency [CPS ID], ornithine transcarbamylase deficiency [OTCD], argininosuccinic acid synthetase deficiency [ASSD], argininosuccinic acid lyase deficiency [ASLD], arginase deficiency [ARGD]. MedDRA version: 19.0;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: Heterologous Human Adult Liver-derived Progenitor Cells | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | France;Spain;Belgium;Poland | ||
| 3 | EUCTR2014-000650-11-ES (EUCTR) | 13/04/201520150413 | 20/10/201420141020 | 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 | ||
| 4 | EUCTR2014-000650-11-FR (EUCTR) | 18/02/201520150218 | 26/06/201520150626 | 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, multicenter, efficacy and safety study of several infusions of Promethera HepaStem in Urea Cycle Disorders pediatric patients - HEP002 A prospective, open label, multicenter, efficacy and safety study of several infusions of Promethera ... | The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are seven disorders to be investigated: carbamoylphosphate synthetase I deficiency [CPS ID], ornithine transcarbamylase deficiency [OTCD], argininosuccinic acid synthetase deficiency [ASSD], argininosuccinic acid lyase deficiency [ASLD], arginase deficiency [ARGD], N-acetylglutamate synthase deficiency [NAGSD], and citrine deficiency. 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: Heterologous Human Adult Liver-derived Progenitor Cells | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | France;Poland;Spain;Belgium | ||
| 5 | EUCTR2014-000650-11-BE (EUCTR) | 15/09/201420140915 | 01/07/201420140701 | 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, multicountry, efficacy and safety study of several infusions of HepaStem in Urea Cycle Disorders pediatric patients. - HEP002 A prospective, open label, multicountry, efficacy and safety study of several infusions of HepaStemi ... | The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are seven disorders to be investigated: carbamoylphosphate synthetase I deficiency [CPS ID], ornithine transcarbamylase deficiency [OTCD], argininosuccinic acid synthetase deficiency [ASSD], argininosuccinic acid lyase deficiency [ASLD], arginase deficiency [ARGD], N-acetylglutamate synthase deficiency [NAGSD], and citrine deficiency. 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: Heterologous Human Adult Liver-derived Progenitor Cells | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | France;Poland;Spain;Belgium | ||
| 6 | EUCTR2013-001045-14-GB (EUCTR) | 30/07/201420140730 | 28/04/201420140428 | 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 | Phase 1;Phase 2 | Portugal;Belgium;Italy;United Kingdom | ||
| 7 | EUCTR2013-001045-14-PT (EUCTR) | 04/04/201420140404 | 02/10/201320131002 | 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 | ||
| 8 | EUCTR2013-001045-14-IT (EUCTR) | 14/02/201420140214 | 31/12/201320131231 | 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 | ||
| 9 | EUCTR2013-001045-14-BE (EUCTR) | 22/04/201320130422 | 19/03/201320130319 | 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 | ||
| 10 | EUCTR2011-004074-28-IT (EUCTR) | 19/12/201220121219 | 14/01/201320130114 | 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 |