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

• GNT-016-MDYF: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory DMD
• 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
• SRP4045-302 : Long-term, Open-label Extension Study for Patients With DMD Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
• MIS51ON : 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 DMD With Deletion Mutations Amenable to Exon 51 Skipping
• Italfarmaco 51: 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
• PTC-PASS: Long-Term Observational Study of Translarna safety and effectiveness in routine care
• FGCL-3019-093: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory DMD
• FGCL-3019-094 : A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in ambulatory subjects with DMD

Study of progression of the disease

• GNT-014-MDYF: Natural History of Duchenne Muscular Dystrophy – A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years

Database

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

Further details on the clinical trials

• GNT-014-MDYF: Natural History of Duchenne Muscular Dystrophy – A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years
• MIS51ON: 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 DMD With Deletion Mutations Amenable to Exon 51 Skipping

• 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
• SRP4045-302 : Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen

• Italfarmaco 51: 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
• PTC-PASS: Long-Term Observational Study of Translarna safety and effectiveness in routine care

• FGCL-3019-093: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory DMD
• FGCL-3019-094: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in ambulatory subjects with DMD

Study of progression of the disease:

• GNT-014-MDYF: Natural History of Duchenne Muscular Dystrophy – A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years

Database:

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