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 54 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 | NCT05412160 (ClinicalTrials.gov) | January 18, 2023 | 6/6/2022 | Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD: a Multicenter, Randomized, Controlled, Proof of Concept Study Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD: a Multic ... | Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD: a Multicenter, Randomized, Controlled, Proof of Concept Study Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD: a Multic ... | COPD;Dyspnea;Argininemia | Drug: Arginine;Drug: Placebo | University of Milan | NULL | Recruiting | 40 Years | N/A | All | 152 | N/A | Italy |
| 2 | 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 clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in pati ... | 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 A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (A ... | 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] Late-onset Ornithine transcarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classificatio ... | 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) Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Aceta ... | 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 United States;Portugal;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;G ... | ||
| 3 | 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 A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Seroty ... | OTC Deficiency | Genetic: DTX301;Other: Placebo;Drug: Oral Corticosteroids;Drug: Placebo for oral corticosteroids;Drug: Sodium Acetate Genetic: DTX301;Other: Placebo;Drug: Oral Corticosteroids;Drug: Placebo for oral corticosteroids;Dru ... | 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 United States;Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spai ... |
| 4 | 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 clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in pati ... | 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 A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (A ... | 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] Late-onset Ornithine transcarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classificatio ... | 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) Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Aceta ... | 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 United States;Portugal;Spain;United Kingdom;Italy;France;Canada;Argentina;Brazil;Croatia;Australia;G ... | ||
| 5 | 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 Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiencyto Evaluate Sa ... | 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 Stu ... | 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 |
| 6 | 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 clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in pati ... | 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 A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (A ... | 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] Late-onset Ornithine transcarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classificatio ... | 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) Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Aceta ... | 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 Portugal;United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;G ... | ||
| 7 | 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. Phase I/II Open Label, Multicentre Clinical Trial to Assess Safety and Efficacy of AAVLK03hOTC for P ... | Ornithine Transcarbamylase Deficiency | Genetic: AAVLK03hOTC | University College, London | NULL | Not yet recruiting | N/A | 16 Years | All | 12 | Phase 1/Phase 2 | NULL |
| 8 | 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 | ||
| 9 | 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 urea formation capacity in babies up to 12 months old using 15N ammonium ch ... | 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 urea cycle disorders: ASS, CPS ... | 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 | ||
| 10 | 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 urea cycle disorders: ASS, CPS ... | 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 ... |