Muscular Dystrophies
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 15 Companies   22 Products   22 Products   12 Mechanisms of Action   2 Trials   254 News 


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  • ||||||||||  Trial completion date, Trial primary completion date:  Genetics of Cardiovascular and Neuromuscular Disease (clinicaltrials.gov) -  Jul 25, 2024   
    P=N/A,  N=2000, Recruiting, 
    Trial completion date: Jan 2025 --> Jan 2026 | Trial primary completion date: Jan 2025 --> Jan 2026
  • ||||||||||  Trial completion, Enrollment change, Trial primary completion date:  STEADFAST: Stacking Exercises Aid the Decline in FVC and Sick Time (clinicaltrials.gov) -  Dec 24, 2018   
    P4,  N=70, Completed, 
    Trial completion date: Jan 2025 --> Jan 2026 | Trial primary completion date: Jan 2025 --> Jan 2026 Active, not recruiting --> Completed | N=110 --> 70 | Trial primary completion date: Aug 2018 --> Nov 2018
  • ||||||||||  NN1213 / Novo Nordisk
    Enrollment change, Trial withdrawal:  Cell Therapy in Limb Girdle Muscular Dystrophy (clinicaltrials.gov) -  Oct 25, 2018   
    P1,  N=0, Withdrawn, 
    Active, not recruiting --> Completed | N=110 --> 70 | Trial primary completion date: Aug 2018 --> Nov 2018 N=65 --> 0 | Completed --> Withdrawn
  • ||||||||||  NN1213 / Novo Nordisk
    Enrollment change, Trial withdrawal:  Stem Cell Therapy in Duchenne Muscular Dystrophy (clinicaltrials.gov) -  Oct 25, 2018   
    P1,  N=0, Withdrawn, 
    N=65 --> 0 | Completed --> Withdrawn N=245 --> 0 | Completed --> Withdrawn
  • ||||||||||  NN1213 / Novo Nordisk
    Enrollment change, Trial withdrawal:  Stem Cell Therapy in Muscular Dystrophy (clinicaltrials.gov) -  Oct 25, 2018   
    P1,  N=0, Withdrawn, 
    N=245 --> 0 | Completed --> Withdrawn N=1000 --> 0 | Recruiting --> Withdrawn
  • ||||||||||  NN1213 / Novo Nordisk
    Enrollment change, Trial withdrawal:  Stem Cell Therapy in Limb Girdle Muscular Dystrophy (clinicaltrials.gov) -  Oct 25, 2018   
    P1,  N=0, Withdrawn, 
    N=1000 --> 0 | Recruiting --> Withdrawn N=65 --> 0 | Completed --> Withdrawn
  • ||||||||||  lisinopril / Generic mfg.
    Trial completion, Enrollment change:  Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies (clinicaltrials.gov) -  Jun 15, 2018   
    P2/3,  N=63, Completed, 
    Trial completion date: Mar 2012 --> Mar 2016 | Trial primary completion date: Mar 2012 --> Mar 2016 Enrolling by invitation --> Completed | N=120 --> 63
  • ||||||||||  Trial completion date, Trial primary completion date:  Stem Cell Therapy in Muscular Dystrophy (clinicaltrials.gov) -  Mar 30, 2018   
    P1,  N=1000, Recruiting, 
    Phase classification: P=N/A --> P Trial completion date: May 2018 --> May 2019 | Trial primary completion date: Dec 2017 --> Dec 2018
  • ||||||||||  Trial completion:  DM1: Arrhythmias in Myotonic Muscular Dystrophy (clinicaltrials.gov) -  Feb 14, 2018   
    P=N/A,  N=448, Completed, 
    Recruiting --> Completed | Trial completion date: Feb 2028 --> Nov 2017 | Trial primary completion date: Feb 2020 --> Nov 2017 Active, not recruiting --> Completed
  • ||||||||||  Emflaza (deflazacort) / Marathon, PTC Therap
    Trial completion, Trial primary completion date:  An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort (clinicaltrials.gov) -  Sep 26, 2017   
    P1,  N=24, Completed, 
    Active, not recruiting --> Completed | Trial primary completion date: Aug 2018 --> Aug 2017 Active, not recruiting --> Completed | Trial primary completion date: Apr 2017 --> Aug 2017
  • ||||||||||  Trial completion, Phase classification, Trial initiation date:  Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy (clinicaltrials.gov) -  Mar 10, 2017   
    P=N/A,  N=32, Completed, 
    Recruiting --> Enrolling by invitation | Trial primary completion date: Dec 2016 --> Jun 2017 Recruiting --> Completed | Phase classification: P4 --> PN/A | Initiation date: Feb 2011 --> Jul 2012
  • ||||||||||  Biomarker, Enrollment closed, Enrollment change, Trial primary completion date:  MOS-DM1/POP: Multicenter Observational Study of Myotonic Dystrophy Type 1 (clinicaltrials.gov) -  Mar 1, 2017   
    P=N/A,  N=120, Active, not recruiting, 
    Recruiting --> Completed | Phase classification: P4 --> PN/A | Initiation date: Feb 2011 --> Jul 2012 Recruiting --> Active, not recruiting | N=100 --> 120 | Trial primary completion date: Dec 2015 --> Jul 2017
  • ||||||||||  Enrollment closed, Enrollment change:  Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy (clinicaltrials.gov) -  Feb 23, 2017   
    P=N/A,  N=4, Active, not recruiting, 
    Recruiting --> Active, not recruiting | N=100 --> 120 | Trial primary completion date: Dec 2015 --> Jul 2017 Recruiting --> Active, not recruiting | N=20 --> 4
  • ||||||||||  Enrollment closed:  STEADFAST: Stacking Exercises Aid the Decline in FVC and Sick Time (clinicaltrials.gov) -  Jan 18, 2017   
    P4,  N=110, Active, not recruiting, 
    Recruiting --> Completed | N=105 --> 50 | Trial primary completion date: Oct 2012 --> Nov 2013 Recruiting --> Active, not recruiting
  • ||||||||||  Trial termination, HEOR:  A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. (clinicaltrials.gov) -  Oct 28, 2016   
    P=N/A,  N=269, Terminated, 
    Active, not recruiting --> Terminated | Trial primary completion date: May 2009 --> Sep 2016; Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD. Active, not recruiting --> Terminated
  • ||||||||||  BMN 045 / BioMarin
    Enrollment change, Trial termination, Trial primary completion date:  Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy (clinicaltrials.gov) -  Oct 28, 2016   
    P2b,  N=15, Terminated, 
    N=45 --> 9 | Active, not recruiting --> Terminated N=45 --> 15 | Active, not recruiting --> Terminated | Trial primary completion date: Jul 2016 --> Jul 2017
  • ||||||||||  BMN 044 / BioMarin
    Trial termination, Trial primary completion date:  Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) -  Oct 28, 2016   
    P2,  N=15, Terminated, 
    N=67 --> 53 | Recruiting --> Terminated Enrolling by invitation --> Terminated | Trial primary completion date: Sep 2016 --> Sep 2017
  • ||||||||||  Trial completion, Enrollment change, Trial primary completion date:  Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study (clinicaltrials.gov) -  Oct 4, 2016   
    P=N/A,  N=20, Completed, 
    Trial primary completion date: Feb 2016 --> Feb 2020 Recruiting --> Completed | N=30 --> 20 | Trial primary completion date: Jan 2014 --> Apr 2014
  • ||||||||||  Trial primary completion date:  Stem Cell Therapy in Muscular Dystrophy (clinicaltrials.gov) -  Sep 26, 2016   
    P1,  N=1000, Recruiting, 
    Recruiting --> Completed | N=30 --> 20 | Trial primary completion date: Jan 2014 --> Apr 2014 Trial primary completion date: Jan 2016 --> Dec 2017
  • ||||||||||  Trial completion, Enrollment change, Trial initiation date, Trial primary completion date:  Cell Therapy in Limb Girdle Muscular Dystrophy (clinicaltrials.gov) -  Sep 26, 2016   
    P1,  N=65, Completed, 
    Trial primary completion date: Jan 2016 --> Dec 2017 Recruiting --> Completed | N=200 --> 65 | Initiation date: Dec 2013 --> Dec 2010 | Trial primary completion date: Dec 2015 --> Jan 2015
  • ||||||||||  Trial completion, Enrollment change:  Stem Cell Therapy in Duchenne Muscular Dystrophy (clinicaltrials.gov) -  Sep 26, 2016   
    P1,  N=245, Completed, 
    Recruiting --> Completed | N=200 --> 65 | Initiation date: Dec 2013 --> Dec 2010 | Trial primary completion date: Dec 2015 --> Jan 2015 Recruiting --> Completed | N=500 --> 245