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

32 trials found

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No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04808505 (ClinicalTrials.gov)	April 2023	10/3/2021	A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglusta ...	An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immuno ...	Glycogen Storage Disease Type II Infantile Onset	Biological: Cipaglucosidase alfa;Drug: Miglustat	Amicus Therapeutics	NULL	Recruiting	N/A	17 Years	All	36	Phase 3	United States
2	EUCTR2019-000954-67-AT (EUCTR)	19/06/2020	13/03/2020	A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ...	A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ...	Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ...	Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ...	Amicus Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	110	Phase 3	Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hung ...
3	EUCTR2019-000954-67-SI (EUCTR)	03/06/2020	10/03/2020	A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ...	A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ...	Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ...	Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ...	Amicus Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	120	Phase 3	United States;Taiwan;Slovenia;Greece;Spain;Austria;United Kingdom;Italy;France;Hungary;Canada;Argent ...
4	EUCTR2019-000954-67-DE (EUCTR)	17/04/2020	23/12/2019	A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ...	A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ...	Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ...	Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ...	Amicus Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	110	Phase 3	Germany;Japan;Sweden;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina ...
5	EUCTR2019-000954-67-GB (EUCTR)	08/04/2020	13/12/2019	A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ...	A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ...	Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ...	Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ...	Amicus Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	110	Phase 3	United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Her ...
6	EUCTR2019-000954-67-GR (EUCTR)	02/04/2020	13/02/2020	A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ...	A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ...	Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ...	Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ...	Amicus Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	110	Phase 3	United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republi ...
7	EUCTR2019-000954-67-IT (EUCTR)	01/04/2020	17/06/2021	A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ...	A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ...	Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ...	Product Name: ATB200 Product Code: [ATB200] INN or Proposed INN: cipaglucosidasi alfa Product Name: AT2 ...	AMICUS THERAPEUTICS, INC.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	120	Phase 3	United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republi ...
8	EUCTR2019-000954-67-DK (EUCTR)	16/03/2020	20/01/2020	A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ...	A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ...	Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ...	Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ...	Amicus Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	110	Phase 3	United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Korea, Republi ...
9	EUCTR2019-000954-67-FR (EUCTR)	14/03/2020	20/01/2020	A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ...	A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ...	Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ...	Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ...	Amicus Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	120	Phase 3	United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Her ...
10	NCT03911505 (ClinicalTrials.gov)	February 13, 2020	9/4/2019	ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 ...	An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity ...	Pompe Disease (Late-onset)	Biological: Cipaglucosidase Alfa;Drug: Miglustat	Amicus Therapeutics	NULL	Recruiting	0 Years	17 Years	All	22	Phase 3	United States;Australia;Canada;Japan;Taiwan

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