251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details
臨床試験数 : 54 / 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
Showing 1 to 10 of 34 diseases
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 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 A Multi-dose Study of an Investigational Treatment to Evaluate Safety, Tolerability, and Ability to ... | 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 Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Stu ... | 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] Ornithine transcarbamylase deficiency MedDRA version: 21.1;Level: LLT;Classification code 10013373;T ... | Product Name: ARCT-810 Product Code: ARCT-810 INN or Proposed INN: Not assigned Other descriptive name: mRNA encoding modified Ornithine transcarbamylase Product Name: ARCT-810 Product Code: ARCT-810 INN or Proposed INN: Not assigned Other descriptive name: ... | Arcturus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | Belgium;Spain;United Kingdom | ||
| 2 | EUCTR2021-000824-36-AT (EUCTR) | 22/12/2021 | 29/07/2021 | Clinical trial assessing urea formation capacity in babies up to 12 months old using 15N ammonium chloride tracer Clinical trial assessing ureaformation capacity in babies up to 12 months old using 15N ammonium chl ... | An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in newborns and infants up to the age of 12 months with neonatal and infantile onset of urea cycle disorders (UCD) using a 15N ammonium chloride tracer compared to newborns and infants without UCD. - 15N ammonium chloride ureagenesis validation clinical trial An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in ... | Subject has a genetically confirmed diagnosis of any of the following urea cycle disorders: ASS, CPS1, ASL, OTC Subjects without UCD can have other stable illness that not interfere with the clinical trial according to the investigator judgement 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] Subject has a genetically confirmed diagnosis of any of the following ureacycle disorders: ASS, CPS1 ... | Product Name: 15N ammonium chloride (15NH4Cl) INN or Proposed INN: Ammonium (15N) chloride Other descriptive name: Ammonium (15N) chloride Product Name: 15N ammonium chloride (15NH4Cl) INN or Proposed INN: Ammonium (15N) chloride Other descr ... | Unicyte AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 1 | France;Saudi Arabia;Spain;Belgium;Turkey;Austria;Israel;Germany;United Kingdom;Switzerland;Italy | ||
| 3 | EUCTR2021-000824-36-ES (EUCTR) | 09/12/2021 | 11/06/2021 | An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in newborns and infants up to the age of 12 months with neonatal and infantile onset of urea cycle disorders (UCD) using a 15N ammonium chloride tracer compared to newborns and infants without UCD. - 15N ammonium chloride ureagenesis validation clinical trial An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in ... | An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in newborns and infants up to the age of 12 months with neonatal and infantile onset of urea cycle disorders (UCD) using a 15N ammonium chloride tracer compared to newborns and infants without UCD. - 15N ammonium chloride ureagenesis validation clinical trial An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in ... | Subject has a genetically confirmed diagnosis of any of the following urea cycle disorders: ASS, CPS1, ASL, OTC Subjects without UCD can have other stable illness that not interfere with the clinical trial according to the investigator judgement 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] Subject has a genetically confirmed diagnosis of any of the following ureacycle disorders: ASS, CPS1 ... | Product Name: 15N ammonium chloride (15NH4Cl) INN or Proposed INN: Ammonium (15N) chloride Other descriptive name: Ammonium (15N) chloride Product Name: 15N ammonium chloride (15NH4Cl) INN or Proposed INN: Ammonium (15N) chloride Other descr ... | Unicyte AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 1 | Portugal;Czechia;Saudi Arabia;Spain;Turkey;Austria;Israel;United Kingdom;Italy;Switzerland;France;Belgium;Poland;Germany;Netherlands Portugal;Czechia;Saudi Arabia;Spain;Turkey;Austria;Israel;United Kingdom;Italy;Switzerland;France;Be ... | ||
| 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. International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects wi ... | 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 A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a UreaCycl ... | 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] Subjects with a UreaCycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1; ... | Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195 | Kaleido Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 29 | Phase 2 | United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Netherlands;Germany;United Kingdom;Switzerland United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Netherlands;Germany;United King ... | ||
| 5 | 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. International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects wi ... | 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 A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects witha UreaCycle ... | 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] Subjects with a UreaCycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1; ... | Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195 | Kaleido Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 29 | Phase 2 | United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Netherlands;Germany;United Kingdom;Switzerland United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Netherlands;Germany;United King ... | ||
| 6 | 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. International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects wi ... | 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 A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a UreaCycl ... | 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] Subjects with a UreaCycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1; ... | Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195 | Kaleido Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 29 | Phase 2 | United States;France;Saudi Arabia;Mexico;Canada;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland United States;France;Saudi Arabia;Mexico;Canada;Belgium;Spain;Turkey;Netherlands;Germany;United King ... | ||
| 7 | 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 A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects With A UreaCycl ... | Urea Cycle Disorder | Drug: KB195 | Kaleido Biosciences | NULL | Terminated | 18 Years | 65 Years | All | 13 | Phase 2 | United States;Belgium;Germany;Spain;Switzerland;Turkey;United Kingdom;Mexico |
| 8 | 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. International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects wi ... | 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 A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects witha UreaCycle ... | 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] Subjects with a UreaCycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1; ... | 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 United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Germany;Switzerland;United King ... | ||
| 9 | 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. International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects wi ... | 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 A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects witha UreaCycle ... | 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] Subjects with a UreaCycle Disorder with Inadequate Control on Standard of Care MedDRA version: 20.1; ... | Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195 | Kaleido Biosciences | NULL | Not Recruiting | Female: yes Male: yes | 29 | Phase 2 | United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Germany;United Kingdom;Switzerland United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Germany;United Kingdom;Switzerl ... | ||
| 10 | 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 A Prospective, Open Label, Safety and Efficacy Study of Infusions of HepaStem in UreaCycle Disorders ... | Urea Cycle Disorder | Biological: HepaStem Infusion | HLB Cell Co., Ltd. | NULL | Withdrawn | N/A | 12 Years | All | 0 | Phase 2 | Korea, Republic of |