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
| 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 | EUCTR2020-003384-25-FR (EUCTR) | 16/12/2022 | 26/11/2021 | A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiency | A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-Onset OTC Deficiency | Late-onset Ornithine transcarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Acetate INN or Proposed INN: Sodium Ethanoate (1- 13 C) Other descriptive name: Sodium acetate (1-13C) | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Portugal;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;Germany;Netherlands;Japan | ||
| 2 | NCT05345171 (ClinicalTrials.gov) | October 18, 2022 | 18/4/2022 | Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency | A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency | OTC Deficiency | Genetic: DTX301;Other: Placebo;Drug: Oral Corticosteroids;Drug: Placebo for oral corticosteroids;Drug: Sodium Acetate | Ultragenyx Pharmaceutical Inc | NULL | Recruiting | 12 Years | N/A | All | 50 | Phase 3 | United States;Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom |
| 3 | EUCTR2020-003384-25-DE (EUCTR) | 02/08/2022 | 17/12/2021 | A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiency | A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-Onset OTC Deficiency | Late-onset Ornithine transcarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Acetate INN or Proposed INN: Sodium Ethanoate (1- 13 C) Other descriptive name: Sodium acetate (1-13C) | Ultragenyx Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 3 | United States;Portugal;Spain;United Kingdom;Italy;France;Canada;Argentina;Brazil;Croatia;Australia;Germany;Netherlands;Japan | ||
| 4 | NCT05526066 (ClinicalTrials.gov) | July 6, 2022 | 8/8/2022 | Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 | Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency;OTC Deficiency;OTCD | Biological: ARCT-810;Other: Placebo | Arcturus Therapeutics, Inc. | NULL | Recruiting | 12 Years | 65 Years | All | 24 | Phase 2 | Belgium;Spain;United Kingdom |
| 5 | EUCTR2020-003384-25-ES (EUCTR) | 04/04/2022 | 17/12/2021 | A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiency | A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-Onset OTC Deficiency | Late-onset Ornithine transcarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Acetate INN or Proposed INN: Sodium Ethanoate (1-13C) Other descriptive name: Sodium acetate (1-13C) | Ultragenyx Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 3 | Portugal;United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;Germany;Netherlands;Japan | ||
| 6 | NCT05092685 (ClinicalTrials.gov) | February 1, 2022 | 28/9/2021 | Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn | Phase I/II Open Label, Multicentre Clinical Trial to Assess Safety and Efficacy of AAVLK03hOTC for Paediatric Patients With Ornithine Transcarbamylase Deficiency. | Ornithine Transcarbamylase Deficiency | Genetic: AAVLK03hOTC | University College, London | NULL | Not yet recruiting | N/A | 16 Years | All | 12 | Phase 1/Phase 2 | NULL |
| 7 | EUCTR2021-001081-38-ES (EUCTR) | 12/01/2022 | 31/08/2021 | A Multi-dose Study of an Investigational Treatment to Evaluate Safety, Tolerability, and Ability to Improve Urea Cycle Function in Adolescents and Adults with Ornithine Transcarbamylase Deficiency | Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants with Ornithine Transcarbamylase Deficiency - Phase 2 nested single/multiple ascending dose study of ARCT-810 in Participants with OTC Deficiency | Ornithine transcarbamylase deficiency MedDRA version: 21.1;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism;System Organ Class: 200000003094;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ARCT-810 Product Code: ARCT-810 INN or Proposed INN: Not assigned Other descriptive name: mRNA encoding modified Ornithine transcarbamylase | Arcturus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | Belgium;Spain;United Kingdom | ||
| 8 | EUCTR2018-000156-18-FR (EUCTR) | 02/11/2020 | 31/01/2020 | A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term | A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency | Ornithine transcarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301 | Ultragenyx Pharmaceutical, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Phase 1;Phase 2 | United States;France;Canada;Spain;United Kingdom | ||
| 9 | NCT03767270 (ClinicalTrials.gov) | December 2019 | 28/11/2018 | Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency | A Phase 1/2 Single Ascending Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenously Administered MRT5201 in Subjects With Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency | Biological: MRT5201;Other: Placebo | Translate Bio, Inc. | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 1/Phase 2 | NULL |
| 10 | JPRN-jRCTs041190055 | 24/07/2019 | 24/07/2019 | Carglumic Acid Administration for OTC Deficienscy | A Clinical Study of Carglumic Acid in Patients with Late-Onset Ornithine Transcarbamylase Deficiency | ornitine transcarbamylase deficiency patients | administraton of carglumic acid | Ito Tetsuya | NULL | Complete | Not applicable | < 75age old | Both | 2 | Phase 4 | Japan |
| 11 | EUCTR2018-000156-18-GB (EUCTR) | 15/08/2018 | 02/05/2018 | A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term | A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency | Ornithine transcarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301 | Ultragenyx Pharmaceutical, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Phase 1;Phase 2 | United States;Canada;Spain;United Kingdom | ||
| 12 | EUCTR2018-000156-18-ES (EUCTR) | 18/06/2018 | 14/05/2018 | A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term | A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency | Ornithine transcarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301 | Ultragenyx Pharmaceutical, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 9 | Phase 1;Phase 2 | United States;Canada;Spain;United Kingdom | ||
| 13 | NCT02991144 (ClinicalTrials.gov) | July 31, 2017 | 9/12/2016 | Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency | A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency | Ornithine Transcarbamylase (OTC) Deficiency | Genetic: scAAV8OTC;Drug: Reactive Corticosteroid Taper Regimen;Drug: Prophylactic Corticosteroid Taper Regimen | Ultragenyx Pharmaceutical Inc | NULL | Completed | 18 Years | N/A | All | 16 | Phase 1/Phase 2 | United States;Canada;Spain;United Kingdom |
| 14 | EUCTR2016-001057-40-GB (EUCTR) | 14/03/2017 | 15/12/2016 | An early-phase clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency | A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency | Ornithine transcarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301 | Ultragenyx Pharmaceutical, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Spain;United Kingdom | ||
| 15 | EUCTR2016-001057-40-ES (EUCTR) | 08/03/2017 | 27/12/2016 | An early-phase clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency. | A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency. | Ornithine transcarbamylase deficiency MedDRA version: 19.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301 | Dimension Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 1;Phase 2 | France;United States;Canada;Spain;United Kingdom | ||
| 16 | NCT02670889 (ClinicalTrials.gov) | November 2016 | 14/1/2016 | Urease Inhibitor Drug Treatment for Urea Cycle Disorders | Manipulating the Gut Microbiome in Urea Cycle Disorders | Ornithine Transcarbamylase Deficiency;Argininosuccinate Synthetase Deficiency (Citrullinemia);Argininosuccinic Acid Lyase Deficiency (Argininosuccinic Aciduria);Carbamyl-Phosphate Synthase I Deficiency | Drug: Acetohydroxamic Acid;Drug: Isotopic Intravenous [13C]-Urea | Nicholas Ah Mew | Data Management and Coordinating Center (DMCC);Children's Hospital of Philadelphia | Not yet recruiting | 18 Years | 60 Years | Both | 16 | Phase 1/Phase 2 | NULL |
| 17 | EUCTR2014-000650-11-PL (EUCTR) | 11/12/2015 | 01/10/2015 | 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: A prospective, open label, multi-country, efficacy and safety study of several infusions of HepaStem in Urea Cycle Disorders pediatric patients. - HEP002 | 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] | 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 | ||
| 18 | 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. | A prospective, open label, multi-country, multicenter, efficacy and safety study of several infusions of Promethera HepaStem in Urea Cycle Disorders pediatric patients | 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] | Product Name: HepaStem INN or Proposed INN: Celulas hepatocitos troncales adultas alogenicas de higado expandidas | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | France;Poland;Belgium;Spain | ||
| 19 | EUCTR2014-000650-11-FR (EUCTR) | 18/02/2015 | 26/06/2015 | 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. | A prospective, open label, multicenter, efficacy and safety study of several infusions of Promethera HepaStem in Urea Cycle Disorders pediatric patients - HEP002 | 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] | 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 | ||
| 20 | EUCTR2014-000650-11-BE (EUCTR) | 15/09/2014 | 01/07/2014 | 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. | A prospective, open label, multicountry, efficacy and safety study of several infusions of HepaStem in Urea Cycle Disorders pediatric patients. - HEP002 | 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] | 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 | ||
| 21 | 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 | 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] | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA | 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 | Portugal;Belgium;Italy;United Kingdom | ||
| 22 | 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. | 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 | 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] | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 1;Phase 2 | Portugal;Belgium;Israel;United Kingdom;Italy | ||
| 23 | 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 | 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] | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 1;Phase 2 | Portugal;Belgium;Israel;United Kingdom;Italy | ||
| 24 | 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. | 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 | 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] | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 1;Phase 2 | Portugal;Belgium;United Kingdom;Italy | ||
| 25 | 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 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 | 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] | Product Name: Promethera HepaStem Product Code: HHALPC INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells (HHALPC) | PROMETHERA BIOSCIENCES | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 1/2 | Belgium;United Kingdom;Italy | ||
| 26 | 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 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 | 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] | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 1/2 | Belgium;Italy;United Kingdom | ||
| 27 | 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 | 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 | 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] | Product Name: HepaStem INN or Proposed INN: Heterologous Human Adult Liver-derived Progenitor Cells Other descriptive name: NA | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 1/2 | Belgium;Italy;United Kingdom | ||
| 28 | EUCTR2006-000136-27-DE (EUCTR) | 17/03/2008 | 04/09/2007 | 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) | 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) | Neonates and Infants up to 3 months including with prenatally or postnatally confirmed urea cycle disorder with below listed deficiency and children aged > 3 months up to 5 years including with confirmed urea cycle disorder and unstable metabolism with deficiency of either: - Carbamyl-phosphate Synthetase I Deficiency (CPS1D) - Ornithine Transcarbamylase Deficiency (OTCD) - Argininosuccinate Synthetase Deficiency (ASSD/Citrullinaemia)can be included. MedDRA version: 18.1;Level: PT;Classification code 10052450;Term: Ornithine transcarbamoylase deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10058298;Term: Argininosuccinate synthetase deficiency;Classification code 10058297;Term: Carbamoyl phosphate synthetase deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Human heterologous liver cells (for infusion) Product Code: HHLivC | Cytonet GmbH & Co KG | NULL | Not Recruiting | Female: yes Male: yes | Germany | ||||
| 29 | NCT00004307 (ClinicalTrials.gov) | December 1999 | 18/10/1999 | Study of Treatment and Metabolism in Patients With Urea Cycle Disorders | Therapeutic and Metabolic Studies of Urea Cycle Disorders: Part A: Nitrogen Flux and Ureagenesis; Part B (Closed): Phase I Adenovirus Vector-Mediated Gene Therapy for Ornithine Transcarbamylase Deficiency | Amino Acid Metabolism, Inborn Errors | Behavioral: Protein and calorie controlled diet;Genetic: Ornithine transcarbamylase vector | National Center for Research Resources (NCRR) | Baylor College of Medicine | Recruiting | 6 Months | 64 Years | Both | 66 | Phase 1 | United States |
| 30 | NCT00004498 (ClinicalTrials.gov) | July 1998 | 18/10/1999 | Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Genetic: Adenoviral Vector-Mediated Gene Transfer | University of Pennsylvania | NULL | Terminated | 18 Years | 69 Years | Both | 21 | Phase 1 | NULL | |
| 31 | NCT00004386 (ClinicalTrials.gov) | October 1995 | 18/10/1999 | Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Children's Research Institute | Terminated | 18 Years | 65 Years | Both | Phase 1 | NULL | ||
| 32 | EUCTR2021-001081-38-BE (EUCTR) | 26/08/2021 | Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants with Ornithine Transcarbamylase Deficiency | Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants with Ornithine Transcarbamylase Deficiency - Phase 2 nested single/multiple ascending dose study of ARCT-810 in Participants with OTC Deficiency | Ornithine transcarbamylase deficiency MedDRA version: 21.1;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism;System Organ Class: 200000003094;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ARCT-810 Product Code: ARCT-810 INN or Proposed INN: Not assigned Other descriptive name: mRNA encoding modified Ornithine transcarbamylase | Arcturus Therapeutics, Inc. | NULL | NA | Female: yes Male: yes | 24 | Phase 2 | Belgium;United Kingdom |