Table of Contents
Trial overview
The clinical trial NCT04004065 studied Vesleteplirsen in people with Duchenne muscular dystrophy who were amenable to exon 51-skipping treatment.[1] The study was called MOMENTUM and had two parts: Part A focused on dose finding, and Part B focused on dose efficacy, which means whether the chosen dose showed a helpful effect.[1]
Who was studied
The target population was participants with Duchenne muscular dystrophy.[1] The trial specifically looked at people whose disease was amenable to exon 51-skipping treatment, so it was not a general study for all patients with this condition.[1]
Study design and phase
This was an interventional study, which means the researchers gave a planned treatment instead of only observing participants.[1] It was a Phase 2 trial, a stage that usually checks both safety and early signs that a treatment may work.[1] In Part A, multiple ascending doses were given to help find the maximum tolerated dose, meaning the highest dose that participants could handle without too many serious problems.[1] The treatment was given by intravenous use every 4 weeks.[1]
What was measured
The main outcome in Part A was the incidence of adverse events from baseline up to 75 weeks.[1] Adverse events are medical problems that happen during a study, whether or not they are caused by the treatment.[1] The main outcome in Part B was the change from baseline in dystrophin protein level at Week 28.[1] Dystrophin is a protein found in muscle, and the study measured it in skeletal muscle tissue, which is the muscle attached to bones and used for movement.[1]
Trial status and size
The trial was completed and enrolled 61 participants.[1] This makes it a relatively small study, which is common in early-phase research for rare diseases.[1]
