Duchenne Muscular Dystrophy (DMD) is an X-linked progressive muscular disorder caused by mutations in the dystrophin gene, affecting up to 1 in 5000 boys. Mutations within this gene result in a lack of expression of the functional form of its major protein product, dystrophin, causing damage to the muscle fibers, resulting in progressive muscle weakness and atrophy. The latter is responsible for the clinical manifestations of decreased physical functionality, respiratory and cardiac failure, and eventually death.
A Study to Evaluate the Efficacy and Safety of Casimersen and Golodirsen in patients with Duchenne Muscular Dystrophy (Sarepta Therapeutics)
Principal Investigator: Prof. Liesbeth De Waele
Essence-Sarepta 301: A double-blind, placebo-controlled, multicenter study with an open-label extension to evaluate the efficacy and safety of SRP-4045 and SRP-4053 in patients with Duchenne Muscular Dystrophy.
The Essence-Sarepta 301 trial is studying the efficacy and safety of Golodirsen (IV exon skipping 53, SRP-4053) and Casimersen (IV exon skipping 45, SRP-4045) (compared to placebo) in patients with Duchenne muscular dystrophy. These exon skipping techniques aim to transform the phenotype of the patients to a milder form, Becker muscular dystrophy.
Golodirsen and Casimersen are administered intravenously every week.
A study to Evaluate the Efficacy and Safety of Givinostat in patients with Duchenne Muscular Dystrophy (Italfarmaco)
Principal Investigator: Prof. Liesbeth De Waele
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.
The Italfarmaco 51 trial is an open-label study with Givinostat for patients who participated in one of the Givinostat studies, e.g. the Italfarmaco 48 study. The goal of this study is to slow down the disease progression by inhibiting the production of fibrous tissue. In this trial the long-term safety and efficacy of Givinostat will be evaluated.
This therapy consists of a daily oral administration of Givinostat.
A study to evaluate the efficacy and safety of Pamrevlumab versus placebo in combination with systemic corticosteroids administered every two weeks in subjects with Duchenne muscular dystrophy (FibroGen)
Principal Investigator: Prof. Liesbeth De Waele
Lelantos Two: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy
The Lelantos Two study is evaluating the efficacy and safety of Pamrevlumab (compared to placebo) in ambulant patients with Duchenne Muscular Dystrophy (DMD). Pamrevlumab is an antibody that inactivates the Connective Tissue Growth Factor (CTGF). Inhibiting CTGF would slow the fibrosis or scarring of muscle tissue, slowing the disease progression.
Pamlevrumab is administered intravenously every two weeks.
A study to evaluate the safety and efficacy of PF-0639926 for the treatment of Duchenne Muscular Dystrophy (Pfizer)
Principal Investigator: Prof. Liesbeth De Waele
C3391003: A phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of PF-0639926 for the treatment of Duchenne Muscular Dystrophy
This study examines the efficacy and safety of PF-0639926 (compared to placebo) in outpatients with Duchenne Muscular Dystrophy (DMD). PF-0639926 is a genetically modified organism (GMO) consisting of a viral vector derived from the adeno-associated virus (AAV) with a miniaturized version of the human dystrophin gene. Administering this vector once intravenously would provide stable production of the dystrophin protein in skeletal and heart muscle, stabilizing the condition of DMD patients.
A study of SRP-5051 for dose determination, then dose expansion, in patients with Duchenne Muscular Dystrophy amendable to exon 51-skipping treatment (Sarepta Therapeutics)
Principal investigator: Prof. Dr. Liesbeth De Waele
5051-201 MOMENTUM: A phase 2, two-part, multiple-ascending dose study of SRP-5051 for dose determination, then dose expansion, in patients with Duchenne Muscular Dystrophy amendable to exon 51-skipping treatment.
The Momentum study is examining the efficacy and safety of vesleteplirsen (SRP-5051) in patients with Duchenne muscular dystrophy (compared to placebo). Vesleteplirsen is an exon skipping technique aiming to transform the phenotype of patients to a milder form, Becker muscular dystrophy.
Vesleteplirsen is administered intravenously on a monthly basis.
A Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (Dyne Therapeutics)
Principal investigator: Prof. Dr. Liesbeth De Waele
DELIVER study: A Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The DELIVER study is examining the efficacy and safety of Dyne-251 in patients with Duchenne muscular dystrophy (compared to placebo). Dyne-251 is an exon skipping technique aiming to transform the phenotype of patients to a milder form, Becker muscular dystrophy.
Dyne-251 is administered intravenously on a monthly basis.