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 | 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 | 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 | 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] | Product Name: 15N ammonium chloride (15NH4Cl) INN or Proposed INN: Ammonium (15N) chloride Other descriptive name: Ammonium (15N) chloride | 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 | ||
| 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 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 | 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] | Product Name: 15N ammonium chloride (15NH4Cl) INN or Proposed INN: Ammonium (15N) chloride Other descriptive name: Ammonium (15N) chloride | 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 | ||
| 3 | EUCTR2021-000824-36-BE (EUCTR) | 01/10/2021 | Clinical trial assessing urea formation capacity in babies up to 12 months old using 15N ammonium chloride tracer | 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 | 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] | Product Name: 15N ammonium chloride (15NH4Cl) INN or Proposed INN: Ammonium (15N) chloride Other descriptive name: Ammonium (15N) chloride | Unicyte AG | NULL | NA | Female: yes Male: yes | 30 | Phase 1 | France;Saudi Arabia;Spain;Belgium;Turkey;Austria;Israel;Germany;United Kingdom;Switzerland;Italy | |||
| 4 | EUCTR2021-000824-36-IT (EUCTR) | 16/08/2021 | 15N ammonium chloride ureagenesis validation clinical trial | 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. - reLiver-1 | Subject has a genetically confirmed diagnosis of any of the followingurea cycle disorders: ASS, CPS1, ASL, OTCSubjects without UCD can have other stable illness that not interferewith 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] | Product Name: 15N ammonium chloride (15NH4Cl) Product Code: [na] INN or Proposed INN: Ammonium (15N) chloride Other descriptive name: Ammonium (15N) chloride | Unicyte AG | NULL | NA | Female: yes Male: yes | 30 | Phase 1 | Portugal;Czechia;Saudi Arabia;Spain;Turkey;Austria;Israel;United Kingdom;Italy;Switzerland;France;Belgium;Poland;Germany;Netherlands |