Sodium Ethanoate (1-13C) ( DrugBank: Ethanoate )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
251尿素サイクル異常症1

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_
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agemin
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PhaseCountries
1EUCTR2020-003384-25-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