PTC124 ( DrugBank: - )

4 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
19	ライソゾーム病	2
113	筋ジストロフィー	53
246	メチルマロン酸血症	5
299	嚢胞性線維症	50

19. ライソゾーム病

臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

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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	EUCTR2015-003105-41-GB (EUCTR)	13/10/2015	08/09/2015	Treatment for Nonsense Mutation Mucopolysaccharidosis Type I	CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Atal ...	Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 18.1;Level: PT;Classification code 10 ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product ...	PTC Therapeutics Inc.	NULL	Not Recruiting			Female: yes Male: yes	5	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ...	United Kingdom
2	EUCTR2014-002596-28-GB (EUCTR)	28/01/2015	12/06/2015	Treatment for Nonsense Mutation Mucopolysaccharidosis Type I	A Phase 2 Study of Ataluren (PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I ...	Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 20.1;Level: PT;Classification code 10 ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product ...	PTC Therapeutics Inc.	NULL	Not Recruiting			Female: yes Male: yes	18	Phase 2	Germany;United Kingdom

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113. 筋ジストロフィー

臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170

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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	JPRN-jRCT2041200087	07/01/2021	25/12/2020	An Open-label, Long-Term study of Ataluren in Nonsense Mutation Duchenne Muscular Dystrophy	An Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren (PTC124) in Patients ...	Nonsense Mutation Duchenne Muscular Dystrophy	Subjects will receive ataluren tid as an oral suspension at respective morning, mid-day, and evening ...	Penematsa Vinay	NULL	Recruiting	>= 5age old	Not applicable	Male	25	Phase 3	China;Japan
2	EUCTR2017-001223-49-BG (EUCTR)	16/01/2018	11/10/2017	A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have s ...	A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Pat ...	Nonsense Mutation Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 100 ...	Trade Name: Translarna 125 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 ...	PTC Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	340	Phase 3	United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argenti ...
3	EUCTR2013-005489-20-FR (EUCTR)	18/10/2016	18/06/2015	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystroph ...	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canad ...
4	NCT02819557 (ClinicalTrials.gov)	June 9, 2016	16/6/2016	Study of Ataluren in =2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy	A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patie ...	Duchenne Muscular Dystrophy	Drug: Ataluren	PTC Therapeutics	NULL	Completed	2 Years	5 Years	Male	14	Phase 2	United States
5	EUCTR2013-005489-20-BG (EUCTR)	25/08/2015	26/06/2015	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystroph ...	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canad ...
6	EUCTR2013-005489-20-CZ (EUCTR)	08/01/2015	24/10/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystroph ...	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canad ...
7	EUCTR2013-005489-20-DE (EUCTR)	29/09/2014	28/07/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystroph ...	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canad ...
8	EUCTR2013-005489-20-IT (EUCTR)	10/09/2014	30/07/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystroph ...	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canad ...
9	EUCTR2013-005489-20-ES (EUCTR)	01/09/2014	06/08/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystroph ...	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Brazi ...
10	EUCTR2013-005489-20-BE (EUCTR)	28/08/2014	11/07/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystroph ...	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canad ...

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246. メチルマロン酸血症

臨床試験数 : 23 / 薬物数 : 31 - (DrugBank : 8) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23

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Showing 1 to 5 of 5 diseases

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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	EUCTR2009-016654-41-IT (EUCTR)	11/11/2010	05/07/2010	A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidem ...	A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidem ...	Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 1006418 ...	Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ATALUREN Product ...	PTC THERAPEUTICS, INC.	NULL	Not Recruiting			Female: yes Male: yes	18	Phase 2	France;United Kingdom;Italy
2	EUCTR2009-016654-41-BE (EUCTR)	04/11/2010	22/12/2010	Study of ataluren in patients with Methylmalonic Acidemia	A Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acide ...	Nonsense Mutation Methylmalonic Acidemia (MMA) MedDRA version: 14.0;Level: LLT;Classification code 1 ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	18	Phase 2	France;Belgium;Italy;United Kingdom;Switzerland
3	NCT01141075 (ClinicalTrials.gov)	July 19, 2010	7/6/2010	Ataluren for Nonsense Mutation Methylmalonic Acidemia	A Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acide ...	Amino Acid Metabolism, Inborn Errors	Drug: Ataluren	PTC Therapeutics	Genzyme, a Sanofi Company	Terminated	2 Years	N/A	All	11	Phase 2	Belgium;France;Germany;Italy;Switzerland;United Kingdom
4	EUCTR2009-016654-41-GB (EUCTR)	01/06/2010	17/03/2010	A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidem ...	A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidem ...	Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 1006418 ...	Product Name: Ataluren 125mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	18	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ...	France;Belgium;Italy;United Kingdom
5	EUCTR2009-016654-41-FR (EUCTR)	02/03/2010	04/01/2010	A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidem ...	A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidem ...	Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 1006418 ...	Product Name: Ataluren 125mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	18	Phase 2	France;Belgium;Italy;United Kingdom

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299. 嚢胞性線維症

臨床試験数 : 1,695 / 薬物数 : 1,527 - (DrugBank : 268) / 標的遺伝子数 : 111 - 標的パスウェイ数 : 174

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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	NCT03256799 (ClinicalTrials.gov)	March 17, 2017	18/8/2017	Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations	An Open Label Study to Investigate the Role of Ivacaftor for the Treatment of Cystic Fibrosis in Com ...	Cystic Fibrosis	Drug: Ivacaftor/Ataluren	University of Alabama at Birmingham	NULL	Completed	19 Years	N/A	All	1	Phase 4	United States
2	NCT03256968 (ClinicalTrials.gov)	January 27, 2017	18/8/2017	PTC Study to Evaluate Ataluren in Combination With Ivacaftor	An Open Label N of 1 Study to Evaluate the Study and Efficacy of Long-Term Treatment With Ivacaftor ...	Cystic Fibrosis	Drug: Ataluren	University of Alabama at Birmingham	NULL	Completed	6 Years	N/A	All	1	Phase 4	United States
3	EUCTR2014-005355-83-GR (EUCTR)	05/04/2016	08/02/2016	Confirmatory extension study of ataluren in patients with Cystic Fibrosis	A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis	Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	200	Phase 3	United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ...
4	EUCTR2014-005355-83-GB (EUCTR)	22/03/2016	15/02/2016	Confirmatory extension study of ataluren in patients with Cystic Fibrosis	A Phase 3 extension Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis	Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	200	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ...
5	EUCTR2014-005355-83-BG (EUCTR)	19/02/2016	16/12/2015	Confirmatory extension study of ataluren in patients with Cystic Fibrosis	A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis	Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	200	Phase 3	United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ...
6	EUCTR2014-005355-83-NL (EUCTR)	22/01/2016	25/11/2015	Confirmatory extension study of ataluren in patients with Cystic Fibrosis	A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis	Nonsense Mutation Cystic Fibrosis MedDRA version: 19.0;Level: PT;Classification code 10011762;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	200	Phase 3	United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ...
7	EUCTR2014-005355-83-FR (EUCTR)	08/12/2015	04/12/2015	Confirmatory extension study of ataluren in patients with Cystic Fibrosis	A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis	Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	200	Phase 3	United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ...
8	EUCTR2014-005355-83-IT (EUCTR)	16/10/2015	26/08/2015	Confirmatory extension study of ataluren (PTC124) in patients with Cystic Fibrosis	A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis - ...	Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	200	Phase 3	United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ...
9	EUCTR2014-005355-83-ES (EUCTR)	08/10/2015	02/09/2015	Confirmatory extension study of ataluren in patients with Cystic Fibrosis	A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis	Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	200	Phase 3	United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ...
10	EUCTR2013-004581-34-BE (EUCTR)	01/10/2015	31/07/2014	Confirmatory study of ataluren in patients with Cystic Fibrosis	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic ...	Nonsense Mutation Cystic Fibrosis MedDRA version: 17.1;Level: PT;Classification code 10011762;Term: ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ...	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	208	Phase 3	United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ...

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