256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details


臨床試験数 : 180 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105

  
4 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1EUCTR2019-001283-30-DK
(EUCTR)
03/11/202105/10/2021A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease)Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease Pompe Disease (also known as glycogen storage disease type II)
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Code: SPK-3006
INN or Proposed INN: SPK-3006
Spark TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 1;Phase 2France;United States;Spain;Denmark;Netherlands;Germany;United Kingdom;Italy
2EUCTR2019-001283-30-NL
(EUCTR)
06/01/202108/11/2020A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease)Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease Pompe Disease (also known as glycogen storage disease type II)
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Code: SPK-3006
INN or Proposed INN: SPK-3006
Spark TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 1;Phase 2United States;France;Germany;Netherlands;United Kingdom;Italy
3EUCTR2019-001283-30-DE
(EUCTR)
20/08/202020/01/2020A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease)Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease Pompe Disease (also known as glycogen storage disease type II)
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Code: SPK-3006
INN or Proposed INN: SPK-3006
Spark TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 1;Phase 2France;United States;Spain;Denmark;Netherlands;Germany;United Kingdom;Italy
4EUCTR2019-001283-30-FR
(EUCTR)
16/06/202018/09/2019A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease)Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease Pompe Disease (also known as glycogen storage disease type II)
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Code: SPK-3006
INN or Proposed INN: SPK-3006
Spark TherapeuticsNULLNot RecruitingFemale: yes
Male: yes
20Phase 1;Phase 2United States;France;Denmark;Netherlands;Germany;United Kingdom;Italy