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

  
10 trials found
No.TrialIDDate_
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PhaseCountries
1EUCTR2020-003384-25-FR
(EUCTR)
16/12/202226/11/2021A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiencyA 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 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]
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)
Ultragenyx Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
50Phase 3United States;Portugal;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;Germany;Netherlands;Japan
2NCT05345171
(ClinicalTrials.gov)
October 18, 202218/4/2022Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) DeficiencyA 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 DeficiencyOTC DeficiencyGenetic: DTX301;Other: Placebo;Drug: Oral Corticosteroids;Drug: Placebo for oral corticosteroids;Drug: Sodium AcetateUltragenyx Pharmaceutical IncNULLRecruiting12 YearsN/AAll50Phase 3United States;Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom
3EUCTR2020-003384-25-DE
(EUCTR)
02/08/202217/12/2021A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiencyA 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 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]
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)
Ultragenyx Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 3United States;Portugal;Spain;United Kingdom;Italy;France;Canada;Argentina;Brazil;Croatia;Australia;Germany;Netherlands;Japan
4EUCTR2020-003384-25-ES
(EUCTR)
04/04/202217/12/2021A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiencyA 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 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]
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)
Ultragenyx Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 3Portugal;United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;Germany;Netherlands;Japan
5EUCTR2018-000156-18-FR
(EUCTR)
02/11/202031/01/2020A 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 termA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18Phase 1;Phase 2United States;France;Canada;Spain;United Kingdom
6EUCTR2018-000156-18-GB
(EUCTR)
15/08/201802/05/2018A 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 termA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18Phase 1;Phase 2United States;Canada;Spain;United Kingdom
7EUCTR2018-000156-18-ES
(EUCTR)
18/06/201814/05/2018A 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 termA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
9Phase 1;Phase 2United States;Canada;Spain;United Kingdom
8NCT02991144
(ClinicalTrials.gov)
July 31, 20179/12/2016Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) DeficiencyA 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 DeficiencyOrnithine Transcarbamylase (OTC) DeficiencyGenetic: scAAV8OTC;Drug: Reactive Corticosteroid Taper Regimen;Drug: Prophylactic Corticosteroid Taper RegimenUltragenyx Pharmaceutical IncNULLCompleted18 YearsN/AAll16Phase 1/Phase 2United States;Canada;Spain;United Kingdom
9EUCTR2016-001057-40-GB
(EUCTR)
14/03/201715/12/2016An early-phase clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiencyA 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]
Product Name: DTX301
INN or Proposed INN: Not yet assigned
Other descriptive name: DTX301
Ultragenyx Pharmaceutical, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
18 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Spain;United Kingdom
10EUCTR2016-001057-40-ES
(EUCTR)
08/03/201727/12/2016An 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 1;Phase 2France;United States;Canada;Spain;United Kingdom