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Viltolarsen

Clinical trials of Viltolarsen are studying boys with Duchenne Muscular Dystrophy (DMD). The main goal is to check safety and tolerability, along with other health measures such as heart, kidney, and lab test results. These trials help researchers understand how Viltolarsen performs in this patient group.

Table of contents

Trial overview

The main trial listed for Viltolarsen was NCT04768062, a Phase 3 interventional study in boys with Duchenne Muscular Dystrophy (DMD).[1] The study was completed and was designed to evaluate the safety and tolerability of Viltolarsen given by intravenous infusion once a week at 80 mg/kg.[1]

The study title says it was a phase 3 study of Viltolarsen in boys with DMD.[1] The brief summary says the goal was to evaluate safety and tolerability in boys who had completed the NS-065/NCNP-01-301 study.[1]

Who participated

This trial focused on boys with Duchenne Muscular Dystrophy, which is a genetic muscle disease that causes progressive weakness.[1] Participation was limited to boys who had already completed the earlier NS-065/NCNP-01-301 study.[1]

This means the study did not recruit a broad patient group from the general public.[1] Instead, it followed a specific group from a prior trial to continue learning about the treatment in the same condition.[1]

What was measured in the study

The primary outcomes included vital signs, physical examination, and clinical laboratory tests such as hematology, clinical chemistry, urinalysis, and urine cytology.[1] These checks help researchers look for changes in general health and body function during the study.[1]

The study also measured antibodies to dystrophin and Viltolarsen, a 12-lead electrocardiogram (ECG), renal ultrasound, and treatment-emergent adverse events and serious adverse events.[1] In simple terms, this means researchers looked at immune response, heart rhythm, kidney structure, and any new health problems that appeared during treatment.[1]

The trial’s main purpose was safety and tolerability rather than a direct comparison with another treatment.[1] That makes it a study focused on how the treatment is handled by the body and what monitoring findings appear over time.[1]

Trial status and size

NCT04768062 is listed as completed.[1] The enrollment number was 74 participants.[1]

Because the study is completed, the listed trial record reflects a finished research project rather than an ongoing recruitment effort.[1] The available data show a relatively small, targeted group of boys with DMD who had already taken part in an earlier study.[1]

What this means for patients

For patients and families, this trial shows that Viltolarsen has been studied in a focused group of boys with Duchenne Muscular Dystrophy.[1] The research centered on safety checks, routine medical monitoring, and signs of how the treatment was tolerated.[1]

The study also shows how clinical trials often follow patients over time after an earlier study ends.[1] In this case, the trial continued observation in boys who had already completed a previous Viltolarsen study.[1]

Trial ID Phase Condition studied Status Enrollment
NCT04768062 Phase 3 Duchenne Muscular Dystrophy (DMD) Completed 74

FAQ

  • What is being studied in the Viltolarsen trials? The trial is studying Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD). It focuses on safety and tolerability, and it also tracks several health checks such as lab tests, heart testing, and kidney checks.
  • Who can take part in the trial? The study includes boys with Duchenne Muscular Dystrophy who had already completed the NS-065/NCNP-01-301 study. This means participation is limited to a specific group from a prior trial.
  • What phase is the Viltolarsen trial? This is a Phase 3 trial. Phase 3 studies usually look at how well a treatment works and continue to monitor safety in a larger group of people.
  • What outcomes are being measured? The trial measures vital signs, physical exam findings, blood and urine tests, antibodies to dystrophin and Viltolarsen, ECG results, kidney ultrasound findings, and any treatment-emergent adverse events or serious adverse events.
  • Is the Viltolarsen trial completed? Yes, the trial listed here is completed. The study enrolled 74 participants.

Ongoing Clinical Trials on Viltolarsen

  • Study on the Safety and Efficacy of Viltolarsen for Boys with Duchenne Muscular Dystrophy

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Greece Italy The Netherlands Norway Spain

Glossary

  • Duchenne Muscular Dystrophy (DMD): A genetic muscle disease that causes muscles to become weak over time. The trial is focused on boys with this condition.
  • Phase 3: A later stage of clinical research that studies treatment effects and safety in a larger group of participants.
  • Interventional study: A trial where researchers give a treatment to see what happens, instead of only observing people.
  • Safety: How well a treatment can be used without causing serious harm.
  • Tolerability: How well participants can handle the treatment.
  • Vital signs: Basic body measurements such as pulse, blood pressure, and temperature.
  • Physical examination: A hands-on checkup by a study team to look for changes in health.
  • Clinical laboratory tests: Tests of blood or urine used to check body function and health.
  • Hematology: Blood testing that looks at cells in the blood.
  • Clinical chemistry: Blood tests that measure chemicals and substances in the body.
  • Urinalysis: A urine test that checks for signs of disease or changes in body function.
  • Electrocardiogram (ECG): A test that records the heart’s electrical activity.

References