251. Urea cycle disorder
42 clinical trials, 45 drugs (DrugBank: 10 drugs), 4 drug target genes, 29 drug target pathways
Searched query = "Urea cycle disorder", "N-acetylglutamate synthase deficiency", "NAGS deficiency", "Carbamoyl phosphate synthetase I deficiency", "CPS1 deficiency", "Ornithine transcarbamylase deficiency", "OTC deficiency", "Classic citrullinemia", "Citrullinemia type I", "Argininosuccinic aciduria", "Argininemia", "Hiperornitinemia-hiperamonemia-homocitrulinuria syndrome", "HHH syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | 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 | ||
| 2 | NCT04442347 (ClinicalTrials.gov) | October 2020 | 19/6/2020 | Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency | A Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Clinically Stable Patients With Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency | Biological: ARCT-810;Other: Placebo | Arcturus Therapeutics, Inc. | NULL | Recruiting | 18 Years | N/A | All | 12 | Phase 1 | United States |
| 3 | NCT04416126 (ClinicalTrials.gov) | June 1, 2020 | 28/5/2020 | Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects | A Phase 1 Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Healthy Adult Subjects | Ornithine Transcarbamylase Deficiency | Biological: ARCT-810;Other: Placebo | Arcturus Therapeutics, Inc. | NULL | Recruiting | 18 Years | 65 Years | All | 30 | Phase 1 | New Zealand |
| 4 | EUCTR2018-004842-40-FR (EUCTR) | 14/01/2020 | 18/11/2019 | International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs. | A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care | Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Kaleido Biosciences | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 29 | Phase 2 | United States;France;Saudi Arabia;Mexico;Canada;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland | |||
| 5 | 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 |
| 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 |
| 6 | EUCTR2018-004842-40-DE (EUCTR) | 25/11/2019 | 04/06/2019 | International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs. | A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care | Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Kaleido Biosciences | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 29 | Phase 2 | France;United States;Saudi Arabia;Mexico;Canada;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland | |||
| 7 | EUCTR2018-004842-40-ES (EUCTR) | 06/11/2019 | 11/06/2019 | International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs. | A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care | Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195 | Kaleido Biosciences | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 29 | Phase 2 | France;United States;Saudi Arabia;Mexico;Canada;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland | ||
| 8 | NCT03933410 (ClinicalTrials.gov) | September 17, 2019 | 29/4/2019 | UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder | A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects With A Urea Cycle Disorder With Inadequate Control on Standard of Care | Urea Cycle Disorder | Drug: KB195 | Kaleido Biosciences | NULL | Recruiting | 12 Years | 70 Years | All | 24 | Phase 2 | United States;Belgium;Germany;Spain;Switzerland;Turkey;United Kingdom;Mexico |
| 9 | EUCTR2018-004842-40-GB (EUCTR) | 30/08/2019 | 22/10/2020 | International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs. | A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects witha Urea Cycle Disorder with Inadequate Control on Standard of Care - Kaleido K020-218 | Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195 | Kaleido Biosciences | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 29 | Phase 1;Phase 2 | United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Germany;Switzerland;United Kingdom | ||
| 10 | EUCTR2018-004842-40-BE (EUCTR) | 21/08/2019 | 28/05/2019 | International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs. | A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects witha Urea Cycle Disorder with Inadequate Control on Standard of Care | Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195 | Kaleido Biosciences | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 29 | Phase 2 | United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Germany;United Kingdom;Switzerland | ||
| 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 |
| 11 | 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 | Recruiting | Not applicable | < 75age old | Both | 2 | Phase 4 | Japan |
| 12 | 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 | ||
| 13 | NCT03884959 (ClinicalTrials.gov) | July 12, 2018 | 3/3/2019 | 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 | Urea Cycle Disorder | Biological: HepaStem Infusion | HLB Cell Co., Ltd. | NULL | Withdrawn | N/A | 12 Years | All | 0 | Phase 2 | Korea, Republic of |
| 14 | 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 | ||
| 15 | NCT03335488 (ClinicalTrials.gov) | February 20, 2018 | 18/10/2017 | Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders | A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders | Urea Cycle Disorder | Drug: RAVICTI;Drug: NaPBA | Horizon Therapeutics, LLC | NULL | Recruiting | N/A | 99 Years | All | 18 | Phase 4 | United States;Belgium;Italy;Spain;Switzerland |
| 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 |
| 16 | NCT03064048 (ClinicalTrials.gov) | September 15, 2017 | 10/2/2017 | Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD | Effect of Nitric Oxide (NO) Supplementation on Neurocognitive Measures in Argininosuccinate Lyase Deficiency (ASLD) | Argininosuccinate Lyase Deficiency;Urea Cycle Disorder;Urea Cycle Disorders, Inborn;Argininosuccinic Aciduria | Dietary Supplement: Neo-ASA;Dietary Supplement: Placebo | Baylor College of Medicine | Rare Diseases Clinical Research Network;Neogenis Laboratories | Recruiting | 6 Years | 50 Years | All | 16 | N/A | United States |
| 17 | NCT03179878 (ClinicalTrials.gov) | June 16, 2017 | 6/6/2017 | Safety and Tolerability of SYNB1020-CP-001 | A Phase 1, First-in-human, Oral Single and Multiple Dose-Escalation, Randomized, Double-blinded, Placebo-controlled Study of SYNB1020 in Healthy Adult Volunteers to Evaluate Safety, Tolerability, Dosing, and Pharmacodynamics | Healthy Volunteer;Urea Cycle Disorder | Drug: Placebo;Drug: SYNB1020 | Synlogic | NULL | Completed | 18 Years | 64 Years | All | 52 | Phase 1 | United States |
| 18 | NCT03181828 (ClinicalTrials.gov) | March 24, 2017 | 1/6/2017 | Manipulating the Gut Microbiome Study | Manipulating the Gut Microbiome Study | Urea Cycle Disorder | Drug: Acetohydroxamic Acid Oral Tablet [Lithostat] | Nicholas Ah Mew | NULL | Terminated | 18 Years | 60 Years | All | 4 | Phase 1;Phase 2 | United States |
| 19 | 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] | 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 | |||
| 20 | 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 | ||
| 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 |
| 21 | NCT02991144 (ClinicalTrials.gov) | January 2017 | 9/12/2016 | Safety and Dose-Finding Study of DTX301 (scAAV8OTC) 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 (OTC) Deficiency | Genetic: scAAV8OTC;Drug: Oral prednisone | Ultragenyx Pharmaceutical Inc | NULL | Recruiting | 18 Years | N/A | All | 18 | Phase 1;Phase 2 | United States;Canada;Spain;United Kingdom |
| 22 | 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 |
| 23 | 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 | Spain;Belgium;Poland | ||
| 24 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Belgium;Spain | |||
| 25 | NCT02246218 (ClinicalTrials.gov) | December 31, 2014 | 17/9/2014 | A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders | An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI®) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs) | Urea Cycle Disorder | Drug: RAVICTI | Horizon Therapeutics, LLC | NULL | Completed | N/A | 2 Years | All | 27 | Phase 4 | United States;Canada |
| 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 |
| 26 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Spain;Belgium | |||
| 27 | NCT02252770 (ClinicalTrials.gov) | September 2014 | 25/9/2014 | Nitric Oxide Supplementation in Argininosuccinic Aciduria | Nitric Oxide Supplementation as a Therapeutic Intervention in Argininosuccinic Aciduria | Argininosuccinic Aciduria;Argininosuccinate Lyase Deficiency;Urea Cycle Disorders | Dietary Supplement: Nitric oxide supplement;Dietary Supplement: Placebo | Baylor College of Medicine | NULL | Completed | 8 Years | 64 Years | All | 12 | N/A | United States |
| 28 | 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] | Promethera Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 19 | Phase 1;Phase 2 | Portugal;Belgium;Italy;United Kingdom | |||
| 29 | 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 | ||
| 30 | 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 | ||
| 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 |
| 31 | 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 | ||
| 32 | 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;Phase 2 | Belgium;United Kingdom;Italy | ||
| 33 | 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;Phase 2 | Belgium;Italy;United Kingdom | ||
| 34 | 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;Phase 2 | Belgium;Italy;United Kingdom | ||
| 35 | EUCTR2008-003865-23-GB (EUCTR) | 08/09/2008 | 25/07/2008 | A Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea Cycle Disorders | A Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea Cycle Disorders | Urea Cycle Disorder (urea cycle enzyme or transporter deficiency) MedDRA version: 9.1;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism | Product Name: glyceryl tri-(4-phenylbutyrate)(GT4P) Product Code: HPN-100 INN or Proposed INN: NA Other descriptive name: glyceryl tri-(4-phenylbutyrate) | Hyperion Therapeutics Limited | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United Kingdom | ||
| 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 |
| 36 | NCT00718627 (ClinicalTrials.gov) | July 2008 | 16/7/2008 | Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders | 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) | Urea Cycle Disorders;Carbamoylphosphate Synthetase I Deficiency;Ornithine Transcarbamylase Deficiency;Citrullinemia | Biological: Human Heterologous Liver Cells | Cytonet GmbH & Co. KG | NULL | Completed | N/A | 5 Years | Both | 12 | Phase 2 | Germany |
| 37 | 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 | ||||
| 38 | 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 | 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) | Completed | 5 Years | N/A | All | 12 | Phase 2 | United States |
| 39 | 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 |
| 40 | 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 | ||
| 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 |
| 41 | EUCTR2014-000650-11-FR (EUCTR) | 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 | NA | Female: yes Male: yes | 20 | Phase 2 | France;Poland;Spain;Belgium | |||
| 42 | EUCTR2015-000988-14-Outside-EU/EEA (EUCTR) | 12/10/2016 | Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders | Title: Open, Prospective, Historic-Controlled, Multicenter Study toEvaluate the Safety and Efficacy of Infusion of Liver Cell Suspension(HHLivC) in Children with Urea Cycle Disorders | Neonatal-onset type of the following urea cycle disorders(UCD):ornithine transcarbamylase deficiency (OTCD),carbamoylphosphate synthetase I deficiency (CPS1D), orargininosuccinate synthetase deficiency (ASSD orcitrullinemia);Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Heparesc Product Code: HHLivC INN or Proposed INN: not available Other descriptive name: ALLOGENIC HUMAN HETEROLOGOUS LIVER CELLS | PROMETHERA Biosciences S.A./N.V. | NULL | NA | Female: yes Male: yes | 21 | Phase 2 | United States;Canada |