Drug Trials

Are you looking for clinical trials in Duchenne muscular dystrophy? Here you can find an overview of potential treatments currently investigated, as well as a list of recently completed and/or terminated studies. This document shows clinical trials from clinicaltrials.gov that are currently recruiting or ongoing and trials that will start in the near feature. Other sites listing clinical trials are the European Clinical Trials Register, Orphanet, the World Health Organization and national trial registries.

This overview is regularly updated. The main characteristics of the trial are stated. For a complete description of the study and inclusion/exclusion criteria, you can click on the NCT number, which directs you to the study.

Are you looking for observational trials (for example natural history studies and registries) and studies looking at the effect of a certain intervention? Click here to access other trials than clinical trials.

Drug Trials

NCT-number	Title	Compound		Phase	Patients		Company/Institution	Countries
NCT04632940	A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids, in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)	Pamrevlumab (FG-3019)		III	Ambulant	3 - 11 years	FibroGen	United States
NCT04587908	A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）	TAS-205		III	Ambulant	≥5 years	Taiho Pharmaceutical Co., Ltd.	Japan
NCT04336826	A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)	Ataluren (Translarna)	Stop codon readthrough	II		6 months - 2 years	PTC Therapeutics
NCT04322357	Weekend Steroids and Exercise as Therapy for DMD	Prednisone/prednisolone		II	Ambulant	5 - 8 years	University of Florida, United States	United States
EudraCT Number: 2019-001825-28	Mesoangioblast-mediated exon skipping for genetic correction of exon 51 mutation, based upon a single injection in individual skeletal muscles of five non ambulant patients affected by Duchenne Muscular Dystrophy: a non-randomized, open label, phase I/IIa study	Lentiviral Vector Trans-Skip gene modified Autologous Mesoangioblasts	Exon skip therapy, Exon 51	I	Non-ambulant; Amenable to exon 51 skipping	12 - 17 years	The University of Manchester, United Kingdom	United Kingdom

NCT-number	Title	Compound		Phase	Patients		Company/Institution	Countries
NCT05524883	Safety, Tolerability, Pharmacodynamics, Efficacy and Pharmacokinetic Study of DYNE-251 in Participants with DMD amenable to Exon 51 Skipping	DYNE-251		I/II	Patients: 4-16 years, ambulatory and non-ambulatory, on stable steroids for at least 12 weeks prior to start of study	4 year - 16 year	Dyne Therapeutics	Australia, Belgium, Canada, Italy, Spain, United Kingdom, United States
NCT04626674	A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)	rAAVrh74.MHCK7.micro-dystrophin (SRP-9001)	Gene therapy, Microdystrophin	I	Ambulant	4 - 7 years	Sarepta Therapeutics	United States
NCT03067831	A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults	EDG-5506		I	BMD	19 - 55 years	Edgewise Therapeutics, Inc.	United States
NCT04386304	Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy	(+)-Epicatechin		I	BMD	16 - 59 years	Epirium Bio Inc.	United States
NCT04371666	A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)	Pamrevlumab (FG-3019)		III	Non-ambulant	≥12 years	FibroGen	United States
NCT04060199	Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)	VILTEPSO (Viltolarsen/NS-065/NCNP-01)	Exon skip therapy, Exon 51	III	Ambulant; amenable to exon 53 skipping	4 - 7 years	NS Pharma, Inc.	Canada, Italy, Japan, Korea Republic Of, Netherlands, New Zealand, Russian Federation, Spain, Taiwan, Turkey, United States
NCT04004065	Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)	SRP-5051	Exon skip therapy, Exon 51	II	Ambulant and non-ambulant; amenable to exon 51 skipping	4 - 21 years	Sarepta Therapeutics	Belgium, Canada, Spain, United States
NCT03992430	A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON)	Eteplirsen (Exondys51/ AVI-4658)	Exon skip therapy, Exon 51	III	Ambulant; amenable to exon 51 skipping	7 - 13 years	Sarepta Therapeutics	Canada, Korea Republic Of, Taiwan, United States
NCT03936894	Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy	Canakinumab (ILARIS)		II	Ambulant	2 - 5 years	Children's Research Institute, District of Columbia, United States	United States
NCT03779646	Bisoprolol in DMD Early Cardiomyopathy	Bisoprolol Fumarate		III	Cardiomyopathy; using ACE-inhibitors	>7 years	Peking Union Medical College Hospital, China	China
NCT03777319	Spironolactone Versus Prednisolone in DMD	Spironolactone/prednisolone		I	Ambulant	4 - 7 years	Nationwide Children's Hospital, Columbus, United States	United States
NCT03354039	Tamoxifen in Duchenne Muscular Dystrophy: A Multicenter, Randomised, Double-blind, Placebo-controlled, Phase 3 Safety and Efficacy 48-week Trial	Tamoxifen		III	Ambulant and non-ambulant	78 months - 16 years	University Hospital, Basel, Switzerland	France, Germany, Netherlands, Spain, Switzerland, United Kingdom
NCT03340675	Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (DMD)	Ifetroban		II		>7 years	Cumberland Pharmaceuticals	United States
NCT03067831	Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy	Bone Marrow-Derived Autologous Stem Cells	Cell therapy	II		4 - 25 years	Stem Cells Arabia	Jordan
NCT02500381	Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE)	Casimersen (SRP-4045)/ Golodirsen (SRP-4053)	Exon skip therapy, Exon 45/53	III	Ambulant; Amenable to exon 45 or 53 skipping	7 - 13 years	Sarepta Therapeutics	Sweden, United Kingdom, United States, Australia, Belgium, Bulgaria, Canada, Czech Republic, France, Germany, Greece, Hungary, Israel, Italy, Poland, Spain
NCT02369731	Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)	Ataluren (Translarna)	Stop codon readthrough	NA	Currently treated for at least 5 years with ataluren, outside clinical trial setting	>2 years	PTC Therapeutics	Sweden, United Kingdom, Austria, Czech Republic, France, Germany, Greece, Hungary, Israel, Italy, Latvia, Norway, Portugal, Romania, Slovenia
NCT02196467	Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients	Myoblasts	Cell therapy	II	Able to move wrists	>16 years	CHU de Quebec-Universite Laval, Canada	Canada
NCT04281485	A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy	AAV9.microdystrophin (PF-06939926)	Gene therapy	III	Ambulant	4 - 7 years	Pfizer	Israel, Italy, Spain
NCT04184882	A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)	ASP0367 (MA-0211)		I	Non-ambulant	8 - 16 years	Astellas Pharma Inc	United States

NCT-number	Title	Compound		Phase	Patients		Company/Institution	Countries
NCT04687020	Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)	VILTEPSO (Viltolarsen/NS-065/NCNP-01)	Exon skip therapy, Exon 51	III	Completed previous viltolarsen study (NCT03167255)	-	NS Pharma, Inc.	Canada, United States
NCT03368742	Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)	AAV9.microdystrophin (SGT-001)	Gene therapy, Microdystrophin	II		4 - 17 years	Solid Biosciences	United States
NCT03769116	A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy(DMD)	rAAVrh74.MHCK7.micro-dystrophin (SRP-9001)	Gene therapy, Microdystrophin	II	Ambulant	4 - 7 years	Sarepta Therapeutics	United States
NCT03648827	A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)	Ataluren (Translarna)	Stop codon readthrough	II		2 - 7 years	PTC Therapeutics	United States
NCT03375164	Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy	rAAVrh74.MHCK7.micro-dystrophin (SRP-9001)	Gene therapy, Microdystrophin	II	Ambulant	3 months - 7 years	Sarepta Therapeutics	United States
NCT03333590	Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy	rAAVrh74.MCK.GALGT2	Gene therapy, GALGT2	II	Ambulant	>4 years	Nationwide Children's Hospital, Columbus, United States	United States
NCT03167255	Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)	VILTEPSO (Viltolarsen/NS-065/NCNP-01)	Exon skip therapy, Exon 51	II	Ambulant; participated in previous NS-065/NCNP-01 study (NCT02740972; https://clinicaltrials.gov/ct2/show/NCT02740972)	4 - 10 years	NS Pharma, Inc.	Canada, United States
NCT02835079	Treatment Effect of Tamoxifen on Patients With DMD	Tamoxifen		I	Ambulant	5 - 16 years	Hadassah Medical Organization
NCT02606136	Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) (DMD)	Pamrevlumab (FG-3019)		II	Non-ambulant	>12 years	FibroGen	United States
NCT02571205	Observational Study of Clinical Outcomes for Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy	Sustanon (testosterone)		NA	Patients with delayed puberty	12 - 17 years	Newcastle-upon-Tyne Hospitals NHS Trust, United Kingdom	United Kingdom
NCT01648634	Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy (NEBIDYS)	Nebivolol		III		10 - 15 years	Assistance Publique - Hôpitaux de Paris, France	France
NCT03439670	A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)	Vamorolone/prednisolone		II	Ambulant	4 - 7 years	ReveraGen BioPharma	Australia, Belgium, Canada, Czech Republic, Greece, Israel, Netherlands, Spain, Sweden, United Kingdom, United States
NCT03238235	Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy	Givinostat		II	Becker muscular dystrophy; ambulant	18 - 65 years	Italfarmaco	Italy, Netherlands
NCT03218995	Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping	Eteplirsen (Exondys51/ AVI-4658)	Exon skip therapy, Exon 51	II	Amenable to exon 51 skipping	6 - 48 months	Sarepta Therapeutics	Belgium, France, Germany, Italy, United Kingdom
NCT03179631	Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy	Ataluren (Translarna)	Stop codon readthrough	III	Ambulant	>5 years	PTC Therapeutics	Puerto Rico, Russian Federation, Sri Lanka, Australia, Taiwan, Brazil, Thailand, Bulgaria, Turkey, Canada, United States, China, Hong Kong, India, Japan, Jordan, Korea Republic Of, Malaysia, Mexico, Poland
NCT02851797	Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy	Givinostat		III	Ambulant	6 - 17 years	Italfarmaco	Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Spain, United Kingdom, United States, Serbia

NCT-number	Title	Compound		Phase	Patients		Company/Institution	Countries
NCT04433234	Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy	DS-5141b	Exon skip therapy, Exon 45	II	Participants of NCT02667483 (Study of DS-5141b in Patients With Duchenne Muscular Dystrophy)	>5 years	Daiichi Sankyo	Japan
NCT04428476	Open-label Extension of the HOPE-2 Trial (HOPE-2-OLE)	CAP-1002	Cell therapy	II	Participants particapting ≥ 12 months in HOPE-2 trial (NCT03406780)	≥10 years	Capricor	United States
NCT04054375	Weekly Steroids in Muscular Dystrophy (WSiMD)	Prednisone/prednisolone		II	BMD, LGMD	18 - 65 years	Northwestern University, United States
NCT04179409	A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications	Casimersen (SRP-4045)/ Eteplirsen (Exondys51/ AVI-4658)/ Golodirsen (SRP-4053)		N/A	Amenable to exon 45, 51 or 53 skipping	>6 months	Sarepta Therapeutics	United States
NCT04240314	AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications	scAAV9.U7.ACCA	Gene therapy, Exon 2 (gene therapy-mediated)	II	Pre-ambulant/ambulant; Carriering an exon 2 duplication	6 months - 13 years	Audentes Therapeutics	United States
NCT04129294	Exploratory Study of NS-089/NCNP-02 in DMD	NS-089/NCNP-02	Exon skip therapy, Exon 44	II	Ambulant	4 - 17 years	National Center of Neurology and Psychiatry, Japan/NS Pharma, Inc.	Japan
NCT03985878	A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy	Eteplirsen (Exondys51/ AVI-4658)	Exon skip therapy, Exon 51	II	Completed 96 weeks of treatment in Study 4658-102 (NCT03218995; https://clinicaltrials.gov/ct2/show/study/NCT03218995)	2 - 5 years	Sarepta Therapeutics	Belgium, France, Italy, United Kingdom
NCT03532542	An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy	Casimersen (SRP-4045)/ Golodirsen (SRP-4053)	Exon skip therapy, Exon 45/53	III	Participated in previous casimersen/golodirsen trials	7 - 23 years	Sarepta Therapeutics	Belgium, Czech Republic, France, Germany, Israel, Italy, Poland, Spain, Sweden, United Kingdom, United States
NCT01247207	Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)	Ataluren (Translarna)	Stop codon readthrough	III	Previously treated with ataluren	-	PTC Therapeutics	Canada, United States
NCT03675126	An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051	SRP-5051	Exon skip therapy, Exon 51	II	Participated in previous trials with SRP-5051	>4 years	Sarepta Therapeutics	Canada, United States
NCT03373968	Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study	Givinostat		III	Participated in previous givinostat trials	>7 years	Italfarmaco	Belgium, Canada, France, Germany, Italy, Netherlands, Spain, United Kingdom, United States
NCT03362502	A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy	AAV9.microdystrophin (PF-06939926)	Gene therapy	I	Ambulant and non-ambulant	≥4 years	Pfizer	United States

NCT-number	Title	Compound		Phase	Patients		Company/Institution	Countries
NCT03558958	Safety and Efficacy of P-188 NF in DMD Patients	P-188 NF (Carmeseal-MD™)		II	Non-ambulant	12 - 25 years	Phrixus Pharmaceuticals	United States
NCT03433807	Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)	Idebenone		NA		>8 years	Santhera Pharmaceuticals	United States

NCT-number	Title	Compound		Phase	Patients		Company/Institution	Countries
NCT02814019	A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)	Idebenone		III	Ambulant and non-ambulant	>10 years	Santhera Pharmaceuticals	United Kingdom, United States, Austria, Belgium, Bulgaria, France, Germany, Hungary, Ireland, Israel, Italy, Netherlands, Spain, Sweden, Switzerland
NCT03603288	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study	Idebenone		III	Participated in previous SIDEROS study (NCT02814019; https://clinicaltrials.gov/ct2/show/study/NCT02814019)	>11 years	Santhera Pharmaceuticals	Austria, Belgium, France, Italy, Spain, Switzerland, United Kingdom, United States

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