Duchenne muscular dystrophy (DMD) affects the ensemble of the muscles of the organism: skeletal muscle, cardiac muscle, and some smooth muscle. The first symptoms appear in childhood at about 3 years of age. Only boys are affected. DMD is characterized by progressive muscle weakness of the limbs and the trunk. Going upstairs, then walking become difficult and then impossible. Respiratory and cardiac effects may be lethal.

Clinical trials on DMD ongoing at the Institute:

Pharmacological trials

• WAVE Dystance: A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administrated Intravenously to Patients with Duchenne Muscular Dystrophy
• WAVE-OLE: A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001
• Essence : A Double-blind, Placebo-Controlled, Multicenter Study With and Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
• SRP4658-302 : Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
• SRP 4856-102: An open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with DMD Amenable to Exon 51 Skipping
• SRP 4856-102-OLE: An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
• SRP4658-402: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
• Italfarmaco: Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
• Italfarmaco-E: Open label, long term safety, tolerability, and efficacy study of Givinostat in all DMD patients who have been previously treated in one of the Givinostat studies
• SPITFIRE (Anti-Myostatin): A Phase 2/3, Randomized,Parallel, Double-blind, Placebo-controlled, to assess the Efficacy, Safety and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy
• SIDEROS : A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid steroids
• SIDEROS E: 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
• PTC-PASS : Long-Term Observational Study of Translarna safety and effectiveness in routine care

Studies of progression of the disease

• NatHis-DMD: Outcome measures in Duchenne Muscular Dystrophy: a Natural History Study
• GNT-014-MDYF: Natural History of Duchenne Muscular Dystrophy – A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years
• SRP4658-407: A Natural History Study in Patients with Duchenne Muscular Dystrophy Managed in Routine Clinical Practice

Database

• UMD-DMD : Database Dystrophinopathies (UMD-DMD France) (www.umd.be/DMD)

Further details on the clinical trials

• WAVE Dystance: A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy
• WAVE-OLE: A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001

• Essence : A Double-blind, Placebo-Controlled, Multicenter Study With and Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
• SRP4658-302 : Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy
  Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
• SRP 4856-102: An open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with DMD Amenable to Exon 51 Skipping
• SRP 4856-102-OLE: An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
• SRP4658-402: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

• Italfarmaco: Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
• Italfarmaco-E: Open label, long term safety, tolerability, and efficacy study of Givinostat in all DMD patients who have been previously treated in one of the Givinostat studies

• SPITFIRE (Anti-Myostatin): A Phase 2/3, Randomized,Parallel, Double-blind, Placebo-controlled, to assess the Efficacy, Safety and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy
• SIDEROS : A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid steroids
• SIDEROS E: 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
• PTC-PASS : Long-Term Observational Study of Translarna safety and effectiveness in routine care

Studies of progression of the disease:

• NatHis-DMD: Outcome measures in Duchenne Muscular Dystrophy: a Natural History Study
• GNT-014-MDYF: Natural History of Duchenne Muscular Dystrophy – A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years
• SRP4658-407: A Natural History Study in Patients with Duchenne Muscular Dystrophy Managed in Routine Clinical Practice

Database:

• UMD-DMD: Database Dystrophinopathies (UMD-DMD France) (www.umd.be/DMD)