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

  
2 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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PhaseCountries
1EUCTR2021-001081-38-ES
(EUCTR)
12/01/202231/08/2021A 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 DeficiencyPhase 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 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]
Product Name: ARCT-810
Product Code: ARCT-810
INN or Proposed INN: Not assigned
Other descriptive name: mRNA encoding modified Ornithine transcarbamylase
Arcturus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 2Belgium;Spain;United Kingdom
2EUCTR2021-001081-38-BE
(EUCTR)
26/08/2021Phase 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 DeficiencyPhase 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 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]
Product Name: ARCT-810
Product Code: ARCT-810
INN or Proposed INN: Not assigned
Other descriptive name: mRNA encoding modified Ornithine transcarbamylase
Arcturus Therapeutics, Inc.NULLNAFemale: yes
Male: yes
24Phase 2Belgium;United Kingdom