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

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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	NCT05532813 (ClinicalTrials.gov)	June 2023	6/9/2022	Evaluation of the Efficacy and Safety of Metformin in the Myotonic DystrophyType 1 (Steinert's Disea ...	Evaluation of the Efficacy and Safety of Metformin in the Myotonic DystrophyType 1 (Steinert's Disea ...	Steinert's Disease;Myotonic Dystrophy 1;Metformin	Drug: Treatment taken	Assistance Publique - Hôpitaux de Paris	NULL	Not yet recruiting	18 Years	70 Years	All	142	Phase 3	France
2	NCT05689164 (ClinicalTrials.gov)	April 14, 2023	9/1/2023	A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne ...	Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular DystrophyWho Ha ...	Duchenne Muscular Dystrophy	Biological: fordadistrogene movaparvovec	Pfizer	NULL	Not yet recruiting	0 Years	N/A	Male	250	Phase 3	NULL
3	NCT05230459 (ClinicalTrials.gov)	March 12, 2023	27/1/2022	A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1 ...	A Phase 1/2, Double-blind, Randomized, Placebo-controlled, Dose-escalation Study to Evaluate the Saf ...	Limb Girdle Muscular Dystrophy;Limb-Girdle Muscular DystrophyType 2;LGMD2I;Muscular Dystrophy;LGMD2; ...	Genetic: LION-101 dose level 1;Genetic: LION-101 dose level 2;Other: Placebo	Asklepios Biopharmaceutical, Inc.	NULL	Recruiting	18 Years	65 Years	All	10	Phase 1/Phase 2	United States
4	NCT05753462 (ClinicalTrials.gov)	March 2023	6/2/2023	Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrop ...	Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, PK and PD of SQY51 in Paediatric a ...	Duchenne Muscular Dystrophy	Drug: Phase 1, SQY51;Drug: Phase 2a, SQY51 (cohort 1);Drug: Phase 2a, SQY51 (cohort 2);Drug: Phase 2 ...	Sqy Therapeutics	Biotrial	Recruiting	6 Years	N/A	Male	12	Phase 1/Phase 2	France
5	NCT05548556 (ClinicalTrials.gov)	February 7, 2023	16/9/2022	A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy	A Phase II, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Pharmaco ...	Facioscapulohumeral Muscular Dystrophy (FSHD)	Drug: Placebo;Drug: RO7204239	Hoffmann-La Roche	NULL	Recruiting	18 Years	65 Years	All	48	Phase 2	Denmark
6	NCT05747924 (ClinicalTrials.gov)	February 2023	17/2/2023	Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)	A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability ...	Facio-Scapulo-Humeral Dystrophy;Atrophy, Facioscapulohumeral;Atrophies, Facioscapulohumeral;Faciosca ...	Drug: AOC 1020;Drug: Placebo	Avidity Biosciences, Inc.	NULL	Not yet recruiting	18 Years	65 Years	All	72	Phase 1/Phase 2	NULL
7	NCT05693142 (ClinicalTrials.gov)	January 4, 2023	4/1/2023	AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)	A Phase 1/2 Open-label, Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerabilit ...	Duchenne Muscular Dystrophy	Genetic: RGX-202	REGENXBIO Inc.	NULL	Recruiting	4 Years	11 Years	Male	18	Phase 1/Phase 2	United States
8	NCT05670730 (ClinicalTrials.gov)	November 9, 2022	6/12/2022	Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy(DMD) ...	A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerabilit ...	Duchenne Muscular Dystrophy;Exon 44	Drug: AOC 1044;Drug: Placebo	Avidity Biosciences, Inc.	NULL	Recruiting	7 Years	45 Years	Male	64	Phase 1/Phase 2	United States
9	NCT05558813 (ClinicalTrials.gov)	November 2022	15/4/2022	Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP)	Prospective Cardiac Magnetic Resonance Imaging Study in Duchenne Muscular Dystrophy (DMD-CMP)	Duchenne Muscular Dystrophy	Diagnostic Test: Cardiac MRI;Biological: Blood assays	Assistance Publique - Hôpitaux de Paris	NULL	Not yet recruiting	6 Years	N/A	Male	150	N/A	France
10	NCT05540860 (ClinicalTrials.gov)	October 24, 2022	6/9/2022	A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy	A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children With Duchenne Muscular ...	Duchenne Muscular Dystrophy	Drug: EDG-5506 Dose 1;Drug: EDG-5506 Dose 2;Drug: EDG-5506 Dose 3;Drug: Placebo	Edgewise Therapeutics, Inc.	NULL	Recruiting	4 Years	9 Years	Male	27	Phase 2	United States

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