Duchenne muscular dystrophy

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Information
Disease name
Duchenne muscular dystrophy
Disease ID
DOID:11723
Description
"A muscular dystrophy that has_material_basis_in X-linked mutations in the DMD gene found on the X chromosome. It is characterized by rapidly progressing muscle weakness and muscle atrophy initially involving the lower extremities and eventually affecting the whole body. It affects males whereas females can be carriers. The symptoms start before the age of six and may appear at infancy." [url:http\://en.wikipedia.org/wiki/Duchenne_muscular_dystrophy, url:http\://omim.org/entry/300377, url:http\://www.genome.gov/19518854]
Disease area statistics
Chromosome band
Gene symbol Chromosome Start Stop The number of variant
DMD X 31,119,222 33,211,549 34
Annotation
Genes Mutation Description Source Links
NCT ID Status Phase Summary Start date Completion date
NCT05938023 Active, not recruiting Phase 2 A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy May 18, 2023 March 5, 2025
NCT06241950 Active, not recruiting Phase 1 A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74) January 29, 2024 September 30, 2026
NCT04433234 Active, not recruiting Phase 2 Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy June 30, 2020 January 31, 2027
NCT04428476 Active, not recruiting Phase 2 Open-label Extension of the HOPE-2 Trial July 20, 2020 March 2025
NCT04004065 Active, not recruiting Phase 2 Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment June 26, 2019 January 31, 2029
NCT01633866 Active, not recruiting Pediatric Radio Frequency Coils Generic July 2012 June 2025
NCT05661071 Active, not recruiting Neuropsychological Profiles of Children With Duchenne Muscular Dystrophy and Its Effects on Motor Functions May 11, 2022 March 1, 2023
NCT05514249 Active, not recruiting Phase 1 Treatment of a Single Patient With CRD-TMH-001 August 31, 2022 September 2023
NCT03936894 Active, not recruiting Phase 1/Phase 2 Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy May 1, 2019 April 2024
NCT04687020 Active, not recruiting Phase 4 Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) June 10, 2021 October 2032
NCT04906460 Active, not recruiting Phase 1/Phase 2 Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) September 28, 2021 May 2025
NCT03368742 Active, not recruiting Phase 1/Phase 2 Microdystrophin Gene Transfer Study in Adolescents and Children With DMD December 6, 2017 December 2027
NCT03362502 Active, not recruiting Phase 1 A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy January 23, 2018 March 30, 2026
NCT03333590 Active, not recruiting Phase 1/Phase 2 Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy November 6, 2017 October 2023
NCT04281485 Active, not recruiting Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy November 5, 2020 April 19, 2029
NCT05185622 Active, not recruiting Phase 2 A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) March 21, 2022 July 2024
NCT04768062 Active, not recruiting Phase 3 Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) April 13, 2021 November 2025
NCT06274983 Active, not recruiting N/A DMD- Interactive Virtual Reality Study April 20, 2023 February 29, 2024
NCT02500381 Active, not recruiting Phase 3 Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) September 28, 2016 October 3, 2025
NCT04240314 Active, not recruiting Phase 1/Phase 2 AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. January 15, 2020 November 19, 2025
NCT05096221 Active, not recruiting Phase 3 A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) October 27, 2021 November 30, 2024
NCT02592941 Approved for marketing Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT03863119 Available Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
NCT01388764 Completed Phase 1 Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids January 2012 May 2012
NCT01396239 Completed Phase 2 Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients July 2011 June 2012
NCT01422200 Completed Phase 4 Flu Vaccine Study in Neuromuscular Patients 2011 August 2011 May 2012
NCT01491555 Completed Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy April 2012 September 2015
NCT01521546 Completed N/A Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy February 2012 June 2016
NCT01523964 Completed N/A DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls February 2012 August 2012
NCT01540604 Completed Phase 2 CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers
NCT01557400 Completed Phase 3 Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada May 20, 2012 January 19, 2018
NCT01580501 Completed Phase 1 PDE Inhibitors in DMD Study (Acute Dosing Study) March 2012
NCT01603407 Completed Phase 3 Finding the Optimum Regimen for Duchenne Muscular Dystrophy January 2013 November 2019
NCT01645098 Completed N/A Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy August 2011 June 2013
NCT01648634 Completed Phase 3 Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy February 13, 2012 July 20, 2021
NCT01874275 Completed N/A Duchenne Muscular Dystrophy Clinical Trial June 2013 September 2014
NCT01931644 Completed At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions July 2013 April 2024
NCT01954940 Completed N/A Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy March 2013 March 2017
NCT01963897 Completed Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of Life. August 2013 June 2017
NCT01981915 Completed N/A Optimum Insufflation Capacity in NMD January 2011 March 2013
NCT01999075 Completed Phase 4 Stacking Exercises Aid the Decline in FVC and Sick Time March 2013 November 22, 2018
NCT02034305 Completed Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy January 2014 January 1, 2021
NCT02056808 Completed Phase 1 A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD) November 2013 July 2014
NCT00004646 Completed Phase 3 Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy April 1995
NCT00005574 Completed Phase 1 Gentamicin Treatment of Muscular Dystrophy February 2000 January 2001
NCT00110669 Completed Phase 3 High-dose Prednisone in Duchenne Muscular Dystrophy January 2004 February 2008
NCT00159250 Completed Phase 1/Phase 2 Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy October 26, 2007 March 31, 2009
NCT00264888 Completed Phase 2 Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy December 2005 May 2007
NCT00312247 Completed Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy April 2006 March 2015
NCT00428935 Completed Phase 1 Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy March 2006 July 2010
NCT00451074 Completed Phase 1 Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons March 2007 July 2009
NCT00592553 Completed Phase 2 Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) February 29, 2008 December 31, 2009
NCT00758225 Completed Phase 2 Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) September 2008 January 2011
NCT00844597 Completed Phase 1/Phase 2 Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients January 2009 December 2010
NCT00873782 Completed Phase 1 Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy March 2009 February 2014
NCT01037309 Completed Phase 1/Phase 2 Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) December 2009 October 2013
NCT01066455 Completed Cardiac Outcome Measures in Children With Muscular Dystrophy January 2010 May 2012
NCT01081080 Completed Cardiac Magnetic Resonance in Children With Muscular Dystrophy April 2010 October 2011
NCT01098708 Completed This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy March 2010 November 2013
NCT01125709 Completed Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS) January 2010 August 2010
NCT01126697 Completed Phase 2/Phase 3 Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies February 2010 December 2017
NCT01182324 Completed The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor July 30, 2010 June 24, 2013
NCT01183767 Completed Phase 2/Phase 3 Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy December 30, 2010 September 6, 2018
NCT01207908 Completed Phase 1/Phase 2 Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy November 2010 June 2013
NCT01335295 Completed Phase 1 Safety Study of Flavocoxid in Duchenne Muscular Dystrophy March 2011 December 2013
NCT01359670 Completed Early Phase 1 Tadalafil and Sildenafil for Duchenne Muscular Dystrophy May 2011 May 2013
NCT02081625 Completed Phase 1 Exploratory Study of NS-065/NCNP-01 in DMD June 2013 August 2015
NCT02167217 Completed Phase 2 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy April 17, 2014 March 22, 2017
NCT02168114 Completed N/A Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle July 2014 January 8, 2018
NCT02246478 Completed Phase 1 A Study of TAS-205 for Duchenne Muscular Dystrophy September 2014 September 2015
NCT02251600 Completed Phase 1 A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy December 2014 October 2015
NCT02295748 Completed Phase 1 An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort December 2014 August 24, 2017
NCT02310906 Completed Phase 1/Phase 2 Phase I/II Study of SRP-4053 in DMD Patients January 13, 2015 March 25, 2019
NCT02340923 Completed A Device for Rapid, Painless, Bedside Muscle Evaluation of Children January 2015 August 2016
NCT02354352 Completed Phase 3 Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy March 20, 2015 May 2018
NCT02354781 Completed Phase 1/Phase 2 Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy January 2015 November 2017
NCT02376816 Completed Phase 1 Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy March 2015 September 2017
NCT02418338 Completed N/A 2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children January 2014 June 2017
NCT02421523 Completed N/A Strength Training in Duchenne Muscular Dystrophy May 30, 2015 October 8, 2018
NCT02485938 Completed Phase 1/Phase 2 HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) January 2016 September 2017
NCT02516085 Completed Phase 1 Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin January 2012 October 2012
NCT02530905 Completed Phase 1 Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients October 8, 2015 October 3, 2018
NCT02571205 Completed Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy November 2015 March 2019
NCT02667483 Completed Phase 1/Phase 2 Study of DS-5141b in Patients With Duchenne Muscular Dystrophy October 2015 October 20, 2020
NCT02740972 Completed Phase 2 Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) December 2016 April 2018
NCT02752048 Completed Phase 2 A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy May 2016 October 17, 2017
NCT02760264 Completed Phase 2 A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) June 2016 May 1, 2018
NCT02760277 Completed Phase 2 An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) July 28, 2016 April 26, 2018
NCT02780492 Completed Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study April 11, 2012 April 28, 2022
NCT02819557 Completed Phase 2 Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy June 9, 2016 February 9, 2018
NCT02835079 Completed Phase 1 Treatment Effect of Tamoxifen on Patients With DMD November 2016 November 2020
NCT02851797 Completed Phase 3 Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy June 6, 2017 February 22, 2022
NCT02891434 Completed N/A Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy February 2016 December 2016
NCT02964377 Completed Phase 1/Phase 2 Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents November 2016 July 2018
NCT02994030 Completed Biomarker for Duchenne Muscular Dystrophy August 20, 2018 March 11, 2022
NCT03002298 Completed N/A Virtual Reality in Individuals With Duchenne Muscular Dystrophy January 2016 October 2016
NCT03038399 Completed Phase 2 Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) February 2, 2017 April 30, 2020
NCT03039686 Completed Phase 2/Phase 3 Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy July 6, 2017 April 28, 2020
NCT03167255 Completed Phase 2 Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) July 6, 2017 November 15, 2021
NCT03218995 Completed Phase 2 Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping August 16, 2017 March 10, 2021
NCT03319030 Completed Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD) September 1, 2017 September 30, 2018
NCT03354039 Completed Phase 3 Tamoxifen in Duchenne Muscular Dystrophy June 12, 2018 October 18, 2022
NCT03375164 Completed Phase 1/Phase 2 A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) January 4, 2018 April 25, 2023
NCT03439670 Completed Phase 2 A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) June 29, 2018 August 19, 2021
NCT03490214 Completed N/A Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography June 1, 2018 September 1, 2018
NCT03508947 Completed Phase 1 Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy January 24, 2018 March 6, 2019
NCT03521271 Completed Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy April 5, 2017 January 26, 2018
NCT03529240 Completed N/A Kinesiology Taping in Duchenne Muscular Dystrophy: Effects on Performance, Gait Characteristics, and Energy Consumption June 10, 2017 October 5, 2017
NCT03534349 Completed Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance January 2017 December 2017
NCT03541070 Completed N/A The Effect of Kinesiology Taping on Balance in Duchenne Muscular Dystrophy June 10, 2017 November 10, 2017
NCT03552874 Completed Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy January 1, 2017 March 31, 2018
NCT03589573 Completed Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy March 24, 2017 January 1, 2018
NCT03589612 Completed Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy March 8, 2018 January 24, 2019
NCT03648827 Completed Phase 2 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) December 21, 2018 October 23, 2020
NCT03680365 Completed Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families September 20, 2018 March 15, 2019
NCT03760029 Completed N/A A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy July 24, 2019 March 21, 2023
NCT03786913 Completed Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases March 8, 2016 February 2, 2019
NCT03796637 Completed Phase 2 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren April 11, 2019 June 3, 2019
NCT03947112 Completed Physical Activity Level of Norwegian Boys With Duchenne Muscular Dystrophy August 20, 2020 June 30, 2021
NCT03951675 Completed The Burden of Access in Duchenne Muscular Dystrophy in the US June 18, 2019 May 28, 2020
NCT04060199 Completed Phase 3 Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) April 14, 2020 October 19, 2023
NCT04120168 Completed Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease April 1, 2019 October 21, 2022
NCT04129294 Completed Phase 1/Phase 2 Exploratory Study of NS-089/NCNP-02 in DMD December 2, 2019 May 31, 2022
NCT04173234 Completed N/A Aerobic Exercise in Duchenne Muscular Dystrophy April 11, 2019 December 15, 2020
NCT04179409 Completed Phase 2 A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. February 18, 2020 September 1, 2023
NCT04193085 Completed Wearable Technology to Assess Gait Function in SMA and DMD November 25, 2019 August 31, 2023
NCT04335942 Completed N/A Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor September 30, 2021 June 1, 2023
NCT04384354 Completed Digestive Events in Duchenne Muscular Dystrophy Patients January 2007 April 2019
NCT04607824 Completed Heart Rate Variability in Duchenne Muscular Dystrophy During Computer Task March 2014 June 2020
NCT04740554 Completed Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy March 1, 2013 February 1, 2015
NCT04956289 Completed Phase 2 Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) July 1, 2021 July 13, 2023
NCT05244395 Completed N/A Development of a New Instrument to Evaluate Gait Characteristics of Individuals With Duchenne Muscular Dystrophy February 10, 2020 February 10, 2022
NCT05274555 Completed Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dystrophy March 27, 2019 June 29, 2021
NCT05281120 Completed N/A Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular Dystrophy November 2006 November 2007
NCT05305976 Completed N/A Telerehabilitation in Duchenne Muscular Dystrophy January 11, 2021 January 1, 2022
NCT05330195 Completed N/A Bicycle Ergometer Training in Duchenne Muscular Dystrophy April 18, 2022 May 25, 2023
NCT05338099 Completed Phase 1 Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD) January 18, 2022 December 28, 2022
NCT05436210 Completed Postural and Anthropometric Properties of Foot and Ankle of Patients With DMD June 23, 2022 November 23, 2022
NCT05464446 Completed Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy October 1, 2021 June 20, 2022
NCT05549999 Completed Cultural Adaptation, Validity, and Reliability of the Turkish Version of North Star Ambulatory Assessment November 1, 2022 May 25, 2023
NCT05559710 Completed Motor Imagery in Duchenne Muscular Dystrophy June 20, 2022 September 25, 2023
NCT05564962 Completed Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD) January 19, 2021 September 20, 2021
NCT05575648 Completed N/A Dual Task in Duchenne Muscular Dystrophy September 6, 2022 May 1, 2023
NCT05657938 Completed Evaluation of Home Based Assessments on Participants With DMD October 13, 2022 April 4, 2023
NCT06103006 Completed N/A Remote Physiotherapy to Protect Physical Health in Duchenne Muscular Dystrophy January 1, 2021 February 10, 2022
NCT06174025 Completed Validity and Reliability of the 6 Minute Pegboard Ring Test June 30, 2023 January 1, 2024
NCT06304064 Completed Phase 2 Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) June 21, 2018 March 6, 2019
NCT06379906 Completed Upper Extremity Muscle Strength, Balance and Functional Skills in DMD November 10, 2023 March 10, 2024
NCT06412328 Completed N/A Psychoeducation Program for Parents of Children With DMD June 21, 2023 August 29, 2023
NCT03836300 Enrolling by invitation N/A Parent and Infant Inter(X)Action Intervention (PIXI) November 30, 2018 June 30, 2025
NCT05967351 Enrolling by invitation Phase 3 A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study September 27, 2023 November 30, 2030
NCT06295718 Enrolling by invitation Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Validity and Reliability Study July 1, 2023 May 10, 2024
NCT01247207 Enrolling by invitation Phase 3 Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) November 30, 2010 April 30, 2024
NCT03373968 Enrolling by invitation Phase 2/Phase 3 Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study October 24, 2017 December 2025
NCT06244082 Enrolling by invitation Phase 2 Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping January 22, 2024 December 31, 2026
NCT03655223 Enrolling by invitation Early Check: Expanded Screening in Newborns October 15, 2018 December 31, 2025
NCT02636686 No longer available Extension Study of Drisapersen in DMD Subjects
NCT03433807 No longer available Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
NCT06450639 Not yet recruiting Phase 2 An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy September 30, 2024 June 30, 2027
NCT05558813 Not yet recruiting N/A Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP) November 2024 April 2029
NCT06109103 Not yet recruiting N/A Telerehabilitation-based Motor Imagery Training in Children With Duchenne Muscular Dystrophy December 15, 2023 December 1, 2025
NCT05849688 Not yet recruiting N/A Bicycle Training on Ventilatory Functions in Duchenne Muscular Dystrophy May 15, 2023 August 15, 2023
NCT06053814 Not yet recruiting Phase 1/Phase 2 NS-050/NCNP-03 in Boys With DMD (Meteor50) July 2024 May 2027
NCT05715957 Not yet recruiting Follow-up Study on Female Carriers With DMD Gene Variants March 1, 2023 August 1, 2025
NCT06290713 Not yet recruiting Phase 2 Vasodilator and Exercise Study for DMD (VASO-REx) March 2024 November 2026
NCT06363526 Not yet recruiting N/A Effectiveness of 5-week Digital Respiratory Practice in a Group of Children With Duchenne Muscular Dystrophy and Becker Muscular Dystrophy. May 5, 2024 November 30, 2024
NCT06328725 Not yet recruiting Phase 1/Phase 2 Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy March 2024 November 2025
NCT05540860 Recruiting Phase 2 A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) October 24, 2022 February 2026
NCT06093100 Recruiting Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim August 2024 December 2030
NCT06100887 Recruiting Phase 2 Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (FOX) March 22, 2024 June 2025
NCT01484678 Recruiting Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy September 1, 2020 August 31, 2025
NCT05689164 Recruiting Phase 3 A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. March 13, 2023 May 15, 2039
NCT05683379 Recruiting AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Boys With DMD December 20, 2022 December 2024
NCT06445985 Recruiting N/A Hydrotherapy in Duchenne Muscular Dystrophy (DMD) November 2, 2022 December 12, 2025
NCT05753462 Recruiting Phase 1/Phase 2 Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy April 26, 2023 February 2025
NCT05933057 Recruiting Phase 3 Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy February 19, 2024 February 2028
NCT04529707 Recruiting N/A Sleep Intervention in Young Boys With Duchenne Muscular Dystrophy February 17, 2021 August 31, 2026
NCT04583917 Recruiting Brain Involvement in Dystrophinopathies Part 1 March 30, 2021 December 2023
NCT04587908 Recruiting Phase 3 A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy(REACH-DMD) November 1, 2020 May 2027
NCT06114056 Recruiting Phase 1 A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD) January 22, 2024 December 31, 2028
NCT02069756 Recruiting The Duchenne Registry October 2007 October 2047
NCT05982119 Recruiting N/A Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study July 10, 2020 March 2026
NCT05670730 Recruiting Phase 1/Phase 2 Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping November 9, 2022 March 2025
NCT04668716 Recruiting Brain Involvement in Dystrophinopathies Part 2 October 11, 2021 May 2024
NCT06366815 Recruiting phenotypeS in Non Ambulant Duchenne Muscular Dystrophy August 30, 2022 December 30, 2025
NCT05641805 Recruiting Physical Activity Level and Cognitive Functions in Children With Duchenne Muscular Dystrophy November 29, 2022 November 1, 2023
NCT06124196 Recruiting Wearable Technology to Evaluate Hyperglycemia and HRV in DMD March 20, 2024 February 2031
NCT05712447 Recruiting Duchenne Muscular Dystrophy Video Assessment Registry September 1, 2022 September 1, 2027
NCT05601986 Recruiting N/A Motor Imagery on Children With DMD on Gait and Balance Functions December 16, 2022 December 1, 2024
NCT03882827 Recruiting Natural History of Duchenne Muscular Dystrophy December 19, 2019 June 30, 2023
NCT03611244 Recruiting N/A Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device August 7, 2018 December 2024
NCT06128564 Recruiting Phase 2 A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) November 29, 2023 November 30, 2032
NCT04972604 Recruiting CureDuchenne Link®: A Resource for Research July 9, 2021 July 9, 2031
NCT05016908 Recruiting Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy November 30, 2019 May 2024
NCT05019625 Recruiting Biomarker Development for Muscular Dystrophies February 20, 2015 June 2027
NCT05967793 Recruiting N/A Effect of Kinesiology Taping on Head and Trunk Control in Children With Duchenne Muscular Dystrophy July 2023 November 2023
NCT03779646 Recruiting Phase 2/Phase 3 Bisoprolol in DMD Early Cardiomyopathy January 16, 2019 July 1, 2024
NCT05881408 Recruiting Phase 3 A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) May 31, 2023 January 31, 2027
NCT05195775 Recruiting Phase 2/Phase 3 Tadalafil as Adjuvant Therapy for DMD December 14, 2021 January 20, 2025
NCT05693142 Recruiting Phase 1/Phase 2 AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) January 4, 2023 December 2025
NCT04012671 Recruiting A Registered Cohort Study on Duchenne Muscular Dystrophy July 1, 2019 December 31, 2049
NCT06138639 Recruiting Phase 1/Phase 2 A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) April 5, 2024 January 15, 2030
NCT05249361 Recruiting Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy May 1, 2023 October 1, 2025
NCT06402942 Recruiting N/A Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy June 1, 2024 May 8, 2025
NCT05280730 Recruiting Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy February 2, 2022 December 2024
NCT03057002 Recruiting UTSW HP [13-C] Pyruvate Injection in HCM May 1, 2018 November 8, 2024
NCT06270719 Recruiting An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy February 7, 2024 December 31, 2038
NCT05996003 Recruiting Phase 2 NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) February 22, 2024 November 28, 2025
NCT05409079 Recruiting N/A Schulze Muscular Dystrophy Ability Clinical Study May 26, 2022 June 30, 2023
NCT05412394 Recruiting Phase 4 Once Weekly Infant Corticosteroid Trial for DMD April 30, 2021 December 30, 2026
NCT06054971 Recruiting NSAA NON-Interventional Study Protocol October 2, 2023 May 2024
NCT06079736 Recruiting Phase 2 A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment January 8, 2024 May 2027
NCT06280209 Recruiting Phase 1/Phase 2 A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy January 3, 2024 December 2025
NCT02972580 Recruiting Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD June 2016 December 2025
NCT03603288 Terminated Phase 3 Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) July 4, 2018 November 25, 2020
NCT03907072 Terminated Phase 2/Phase 3 Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy September 4, 2019 January 9, 2020
NCT03917719 Terminated Phase 3 An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy March 14, 2019 October 26, 2020
NCT01753804 Terminated A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. September 1, 2012 October 1, 2016
NCT03985878 Terminated Phase 2 A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) June 26, 2019 August 31, 2022
NCT04371666 Terminated Phase 3 Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD) August 10, 2020 August 17, 2023
NCT01239758 Terminated Phase 2 Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy October 2010 May 2011
NCT04632940 Terminated Phase 3 Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD December 11, 2020 December 12, 2023
NCT01168908 Terminated Phase 2 Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy September 2010 January 2014
NCT04708314 Terminated Phase 4 An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy October 31, 2020 May 13, 2021
NCT01099761 Terminated Phase 2 Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy April 2010 June 2011
NCT02310763 Terminated Phase 2 A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy November 24, 2014 November 23, 2018
NCT02315339 Terminated European Home Mechanical Ventilation Registry August 2014 January 2019
NCT02329769 Terminated Phase 2 Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) December 2014 August 31, 2016
NCT01009294 Terminated Phase 2 Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) January 13, 2010 March 23, 2010
NCT00847379 Terminated Phase 2 Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) January 31, 2009 May 24, 2010
NCT01978366 Terminated Phase 2 Open Label Extension Study of HT-100 in Patients With DMD October 2013 April 30, 2016
NCT02525302 Terminated Phase 2 HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02 May 2015 December 30, 2016
NCT00839033 Terminated Phase 3 Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders June 2009 December 2011
NCT01957059 Terminated Phase 1/Phase 2 A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) June 2013 August 3, 2016
NCT02606136 Terminated Phase 2 Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) January 4, 2016 August 7, 2023
NCT00308113 Terminated Phase 3 CoQ10 and Prednisone in Non-Ambulatory DMD April 2007 November 2010
NCT02858362 Terminated Phase 2 Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD) June 2016 September 11, 2018
NCT02907619 Terminated Phase 2 An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy October 13, 2016 November 22, 2018
NCT02958202 Terminated Phase 2 Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) April 2016 September 2016
NCT01847573 Terminated Phase 1/Phase 2 Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy May 2013 March 30, 2016
NCT01826474 Terminated Phase 1/Phase 2 Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy January 2013 August 31, 2016
NCT00759876 Terminated Phase 2 Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) August 13, 2008 May 17, 2010
NCT03531788 Terminated N/A Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD August 30, 2018 December 31, 2020
NCT03532542 Terminated Phase 3 An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy August 2, 2018 July 26, 2023
NCT03558958 Terminated Phase 2 Safety and Efficacy of P-188 NF in DMD Patients August 8, 2018 September 1, 2021
NCT01610440 Unknown status Phase 1/Phase 2 Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy October 2011 October 2013
NCT00468832 Unknown status Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD) December 2005 December 2019
NCT03067831 Unknown status Phase 1/Phase 2 Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy September 2015 December 2021
NCT05110885 Unknown status Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy Practices November 2021 January 2022
NCT02484560 Unknown status Phase 1 Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2 June 2015 December 2015
NCT02436720 Unknown status Upper Limb Assessment in Duchenne Muscular Dystrophy January 2013 May 2015
NCT01834040 Unknown status Phase 1/Phase 2 Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy September 2014 October 2016
NCT01772043 Unknown status Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping September 2012 August 2016
NCT03372655 Unknown status Prognostic Factors Affecting Duchenne Muscular Dystrophy January 1, 2018 March 1, 2019
NCT05029232 Unknown status N/A Comprehensive Study of Duchenne Muscular Dystrophy at Sohag University Hospital October 1, 2021 August 1, 2023
NCT04782440 Unknown status N/A The Effect of Telerehabilitation of Patients With Duchenne Muscular Dystrophy April 2021 September 2021
NCT02285673 Unknown status Phase 1/Phase 2 Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy November 2013 November 2015
NCT03443115 Unknown status Duchenne Muscular Dystrophy Heart Study June 27, 2017 March 15, 2020
NCT02196467 Unknown status Phase 1/Phase 2 Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients May 2014 January 2024
NCT03507530 Unknown status Effects of Fear of Falling on Physical Performance and Quality of Life in Children With Duchenne Muscular Dystrophy April 2018 January 2019
NCT03513367 Unknown status The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM. September 19, 2018 September 19, 2019
NCT02109692 Unknown status N/A Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies May 19, 2014 November 2019
NCT04287582 Unknown status The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystrophy April 3, 2019 April 1, 2020
NCT00606775 Unknown status Phase 4 The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy December 2007 December 2012
NCT03689660 Unknown status N/A Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback February 1, 2019 October 1, 2023
NCT01385917 Unknown status Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping October 2011 December 2018
NCT00819845 Unknown status Phase 4 Ramipril Versus Carvedilol in Duchenne and Becker Patients December 2008 December 2016
NCT03335384 Withdrawn N/A Relationship Between PFTs and Pdi in DMD May 1, 2018 May 2018
NCT03642145 Withdrawn Phase 3 A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) October 31, 2018 July 31, 2021
NCT02241434 Withdrawn Phase 1 Stem Cell Therapy in Duchenne Muscular Dystrophy January 2009 June 2016
NCT02704325 Withdrawn Phase 1/Phase 2 Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 April 2016 February 2020
Disase is a (Disease Ontology)
DOID:9884
Cross Reference ID (Disease Ontology)
GARD:6291
Cross Reference ID (Disease Ontology)
MESH:D020388
Cross Reference ID (Disease Ontology)
MIM:310200
Cross Reference ID (Disease Ontology)
NCI:C75482
Cross Reference ID (Disease Ontology)
SNOMEDCT_US_2023_03_01:155095006
Cross Reference ID (Disease Ontology)
UMLS_CUI:C0013264
Exact Synonym (Disease Ontology)
Muscular dystrophy, Duchenne
OrphaNumber from OrphaNet (Orphanet)
98896
MedGen concept unique identifier (MedGen Concept name)
C0013264
MedGen unique identifier (MedGen Concept name)
3925