113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details

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

54 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	NCT04336826 (ClinicalTrials.gov)	December 29, 2021	3/4/2020	A Study to Evaluate the Safety and Pharmacokinetics of Atalurenin Participants From =6 Months to <2 ...	An Open-Label Study Evaluating the Safety and Pharmacokinetics of Atalurenin Children From =6 Months ...	Nonsene Mutation Duchenne Muscular Dystrophy	Drug: Ataluren	PTC Therapeutics	NULL	Recruiting	6 Months	2 Years	Male	10	Phase 2	United States
2	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 w ...	Nonsense Mutation Duchenne Muscular Dystrophy	Subjects will receive atalurentid 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
3	NCT03796637 (ClinicalTrials.gov)	April 11, 2019	4/1/2019	A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrop ...	Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mu ...	Duchenne Muscular Dystrophy	Other: Dystrophin levels;Drug: Ataluren	PTC Therapeutics	NULL	Completed	N/A	N/A	Male	6	Phase 2	United States
4	NCT03648827 (ClinicalTrials.gov)	December 21, 2018	24/8/2018	A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrop ...	Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and Af ...	Duchenne Muscular Dystrophy	Drug: Ataluren	PTC Therapeutics	NULL	Completed	2 Years	7 Years	Male	20	Phase 2	United States
5	EUCTR2017-001223-49-BG (EUCTR)	16/01/2018	11/10/2017	A clinical trial to evaluate the safety and efficacy of Atalurenversus placebo in people who have sp ...	A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Atalurenin Pati ...	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 I ...	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 ...
6	NCT03179631 (ClinicalTrials.gov)	July 6, 2017	1/6/2017	Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy	A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Atalurenin Pati ...	Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Mus ...	Drug: Ataluren;Drug: PLACEBO	PTC Therapeutics	NULL	Active, not recruiting	5 Years	N/A	Male	360	Phase 3	United States;Australia;Brazil;Bulgaria;Canada;China;Hong Kong;India;Japan;Korea, Republic of;Malays ...
7	EUCTR2013-005489-20-FR (EUCTR)	18/10/2016	18/06/2015	Extension study of atalurenin patients with nonsense mutation Duchenne and Becker muscular dystrophy ...	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 ...
8	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 Patien ...	Duchenne Muscular Dystrophy	Drug: Ataluren	PTC Therapeutics	NULL	Completed	2 Years	5 Years	Male	14	Phase 2	United States
9	EUCTR2013-005489-20-BG (EUCTR)	25/08/2015	26/06/2015	Extension study of atalurenin patients with nonsense mutation Duchenne and Becker muscular dystrophy ...	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 ...
10	EUCTR2013-005489-20-CZ (EUCTR)	08/01/2015	24/10/2014	Extension study of atalurenin patients with nonsense mutation Duchenne and Becker muscular dystrophy ...	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 ...

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