Sodium Acetate ( DrugBank: Sodium acetate, Acetate )


2 diseases
告示番号疾患名(ページ内リンク)臨床試験数
81先天性副腎皮質酵素欠損症1
251尿素サイクル異常症4

81. 先天性副腎皮質酵素欠損症


臨床試験数 : 87 薬物数 : 87 - (DrugBank : 23) / 標的遺伝子数 : 12 - 標的パスウェイ数 : 68
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT00529841
(ClinicalTrials.gov)
January 200712/9/2007Research Study for Children With Salt Wasting Congenital Adrenal HyperplasiaA Novel Therapeutic Modality for Congenital Adrenal HyperplasiaAdrenal Hyperplasia, CongenitalDrug: Hydrocortisone sodium acetateBaylor College of MedicineNULLCompleted3 Years18 YearsBoth7N/AUnited States

251. 尿素サイクル異常症


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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
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