251. Urea cycle disorder Clinical trials / Disease details
Clinical trials : 48 / Drugs : 52 - (DrugBank : 13) / Drug target genes : 4 - Drug target pathways : 29
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-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 ... | ||
| 3 | NCT05076318 (ClinicalTrials.gov) | March 1, 2021 | 26/8/2021 | Dysregulated Urea-synthesis at Terminal Uremia | Dysregulated Urea-synthesis at Terminal Uremia | Urea Cycle Disorder;Uremia | Other: Aminoacid, alanine-infusion | University of Aarhus | NULL | Recruiting | 18 Years | N/A | All | 16 | N/A | Denmark |
| 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 |