111. 先天性ミオパチー Congenital myopathy Clinical trials / Disease details

臨床試験数 : 10 / 薬物数 : 17 - (DrugBank : 5) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 9

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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	NCT05099107 (ClinicalTrials.gov)	October 25, 2021	15/10/2021	Changes of Motor Function Tests in Congenital MyopathySubjects Treated With Oral Salbutamol as Compa ...	COMPIS- Congenital MyopathyIntervention Study. An Open-label, Cross Over, Randomised, Controlled Stu ...	Congenital Myopathy;Neuromuscular Diseases;Musculoskeletal Diseases;Nemaline Myopathy;Centronuclear ...	Drug: Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet;Drug: Salbutamol Only Product in Oral Dose F ...	Vastra Gotaland Region	NULL	Enrolling by invitation	6 Years	30 Years	All	20	N/A	Sweden
2	NCT04915846 (ClinicalTrials.gov)	December 18, 2020	6/5/2021	Tamoxifen Therapy for Myotubular Myopathy	TAM4MTM: A Phase 1/2 Randomized, Placebo-Controlled, Double-Blinded, Single Crossover Study to Deter ...	X Linked Myotubular Myopathy	Drug: ApoTamox 10mg;Drug: Placebo	James Dowling	Canadian Institutes of Health Research (CIHR);Cures Within Reach;The Joshua Frase Foundation USA;Wil ...	Recruiting	2 Years	N/A	Male	16	Phase 1/Phase 2	United States;Canada;United Kingdom
3	EUCTR2019-001147-51-SE (EUCTR)	31/03/2020	27/09/2019	This is a treatment study for a muscle disorder known as congenital myopathy	Congenital myopathy intervention study - COMPIS	Congenital myopathy MedDRA version: 20.0;Level: PT;Classification code 10062547;Term: Congenital myo ...	Trade Name: Ventoline tablet 2mg INN or Proposed INN: SALBUTAMOL SULFATE Trade Name: Ventoline oral solut ...	Västra götalandsregionen	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 4	Sweden
4	EUCTR2017-000876-27-DE (EUCTR)	22/05/2018	22/09/2017	First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients	ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control ...	X-linked Myotubular Myopathy(XLMTM) MedDRA version: 20.0;Level: HLGT;Classification code 10029317;Te ...	Product Name: rAAV8-Des-hMTM1 Product Code: AT132	Audentes Therapeutics Inc.	NULL	Not Recruiting			Female: no Male: yes	26	Phase 1;Phase 2	France;United States;Canada;Germany;United Kingdom
5	EUCTR2017-000876-27-FR (EUCTR)	23/04/2018	25/05/2018	First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients	ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Cl ...	X-linked Myotubular Myopathy (XLMTM);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: rAAV8-Des-hMTM1 Product Code: AT132	Audentes Therapeutics Inc.	NULL	Not Recruiting			Female: no Male: yes	12	Phase 1;Phase 2	United States;France;Germany;United Kingdom
6	EUCTR2017-000876-27-GB (EUCTR)	06/12/2017	31/07/2017	First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients	ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Cl ...	X-linked Myotubular Myopathy (XLMTM);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: rAAV8-Des-hMTM1 Product Code: AT132	Audentes Therapeutics Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	20	Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ...	France;United States;Germany;United Kingdom
7	NCT03199469 (ClinicalTrials.gov)	August 2, 2017	21/6/2017	Gene Transfer Clinical Study in X-Linked Myotubular Myopathy	ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control ...	X-Linked Myotubular Myopathy	Genetic: AT132	Astellas Gene Therapies	NULL	Active, not recruiting	N/A	5 Years	Male	26	Phase 1/Phase 2	United States;Canada;France;Germany
8	NCT02362425 (ClinicalTrials.gov)	February 12, 2015	12/2/2015	Antioxidant Therapy in RYR1-Related Congenital Myopathy	Antioxidant Therapy in RYR1-Related Congenital Myopathy	Neuromuscular Disease	Drug: N-acetylcysteine;Drug: Placebo	National Institute of Nursing Research (NINR)	NULL	Completed	7 Years	N/A	All	63	Phase 1/Phase 2	United States
9	NCT02035501 (ClinicalTrials.gov)	January 2014	2/7/2013	Treatment of TNNT1-Myopathy With L-Tyrosine.	Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.	Nemaline Myopathy	Drug: L-Tyrosine;Drug: Placebo	Hadassah Medical Organization	NULL	Enrolling by invitation	N/A	20 Years	Both	10	Phase 2	Israel
10	EUCTR2020-004608-32-DE (EUCTR)		27/05/2021	N/A	A Phase 1/2, multicenter, open-label, dose-confirmation trial to evaluate the safety and preliminary ...	Centronuclear myopathy(CNM), in subjects 2 to 17 years of age (all ages inclusive)caused by mutation ...	Product Name: DYN101 Product Code: DYN101 INN or Proposed INN: DYN101	Dynacure	NULL	NA			Female: yes Male: yes	9	Phase 1;Phase 2	France;Spain;Netherlands;Germany

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