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Golodirsen

Clinical trials of Golodirsen are studying people with Duchenne Muscular Dystrophy who are amenable to exon 53 skipping. These studies look at whether Golodirsen can help with movement and muscle function, and they also assess safety and treatment effects.

Table of contents

Trial overview

The source data describe one interventional study, which means researchers gave study treatments and compared results between groups.[1] The study was designed for patients with Duchenne Muscular Dystrophy who were amenable to exon 45 or exon 53 skipping.[1]

The trial title says it was a research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients.[1] In the intervention list, Golodirsen appears as one of the study drugs under the name GOLODIRSEN (SRP-4053).[1]

Who was studied

The study included patients with Duchenne Muscular Dystrophy who were amenable to exon 45 or 53 skipping.[1] “Amenable” means the patient’s condition may fit the study approach for that specific exon-skipping strategy.[1]

The data do not give more details about age, sex, or other entry rules.[1] The enrollment number shows that 328 people took part in the trial.[1]

Trial design and treatment groups

This was a Phase 3 trial, which is a later stage of testing used to study how well a treatment works in a larger group of patients.[1] The intervention list shows three study arms: CASIMERSEN (SRP-4045), GOLODIRSEN (SRP-4053), and 0.9% sodium chloride, which served as the comparison treatment.[1]

The brief summary says the study compared the combined active group with placebo to evaluate the effect on ambulation and muscle function.[1] Placebo means a comparison treatment that does not contain the active study drug.[1]

The study also included a double-blind period, meaning that during that part of the trial, patients and study staff did not know who was receiving active treatment or placebo.[1]

What was measured

The main outcome was change from baseline at Week 96 in 4-step ascend velocity.[1] Baseline is the starting point before treatment begins, and 4-step ascend velocity is a test of how fast a patient can climb four steps.[1]

This outcome was chosen to measure ambulation, which means the ability to walk, and overall muscle function.[1] In simple terms, the study wanted to see whether treatment could help patients move better over time.[1]

Trial status and size

The trial NCT02500381 is listed as Completed.[1] The enrollment number was 328 participants, which shows it was a fairly large study for this condition.[1]

Because the source data only include one trial, the article focuses on this single Phase 3 study of Golodirsen in Duchenne Muscular Dystrophy patients who fit exon-skipping criteria.[1]

Trial ID Phase Condition studied Status Enrollment
NCT02500381 Phase 3 Duchenne Muscular Dystrophy amenable to exon 45 or 53 skipping Completed 328

FAQ

  • What condition are Golodirsen trials studying? The trial data focus on Duchenne Muscular Dystrophy, a serious muscle disease. The study includes patients who are amenable to exon 45 or exon 53 skipping.
  • Who can take part in these trials? The study enrolled patients with Duchenne Muscular Dystrophy who were amenable to exon 45 or 53 skipping. The data do not give more details about age or other entry rules.
  • What phase is the Golodirsen trial? The trial listed is a Phase 3 study. Phase 3 trials usually test how well a treatment works in a larger group of patients.
  • What is being measured in the study? The main measure is change in 4-step ascend velocity at Week 96. This means the study checks how fast patients can climb four steps over time.
  • Is the Golodirsen trial completed? Yes, the trial listed in the source data is completed. It included 328 participants.

Ongoing Clinical Trials on Golodirsen

  • Study on the Effects of Casimersen and Golodirsen for Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Bulgaria Czechia Denmark Hungary Ireland +3

Glossary

  • Duchenne Muscular Dystrophy: A serious muscle disease that causes muscles to get weaker over time.
  • Exon skipping: A trial strategy that aims to help the body make a more useful version of a protein by skipping a specific part of a gene.
  • Exon 45 skipping: A specific type of exon skipping studied in the trial for patients whose disease may fit this approach.
  • Exon 53 skipping: A specific type of exon skipping studied in the trial for patients whose disease may fit this approach.
  • Phase 3: A later stage of clinical testing that studies treatment effects in a larger group of patients.
  • Interventional study: A study where participants receive a treatment or control so researchers can compare outcomes.
  • Placebo: A treatment that does not contain the active study drug, used for comparison in a trial.
  • Double-blind period: A study period when patients and study staff do not know who is receiving the active treatment or placebo.
  • Ambulation: The ability to walk.
  • 4-step ascend velocity: A test that measures how fast a patient can climb four steps.
  • Baseline: The starting point before treatment begins, used for comparison later in the study.

References