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

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness and degeneration. The study involves the use of a treatment called Viltolarsen, which is administered as a solution for infusion. Viltolarsen is also known by its code name, NS-065/NCNP-01, and is designed to help manage symptoms in boys with DMD.

The purpose of this study is to evaluate the safety and tolerability of Viltolarsen when given intravenously, meaning directly into a vein, at weekly doses. Participants in this study are boys who have previously completed another study involving Viltolarsen. The study will monitor various health indicators, such as vital signs and physical examinations, to ensure the treatment is safe and well-tolerated.

Throughout the study, participants will undergo several assessments to track their progress. These assessments include tests like the Time to Stand Test, which measures how quickly a participant can stand up, and the Six-minute Walk Test, which evaluates how far a participant can walk in six minutes. These tests help researchers understand the effects of Viltolarsen on muscle strength and function in boys with Duchenne Muscular Dystrophy.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after completing the previous study, NS-065/NCNP-01-301.

    Written consent from a parent or guardian is required, along with Health Insurance Portability and Accountability Act authorization, if applicable.

    Assent from the patient is needed, either written or verbal, based on local requirements.

  2. Step 2

    Treatment administration

    The medication used is viltolarsen, administered as a solution for infusion.

    The dosage is 80 mg per kg of body weight, given intravenously (IV) once a week.

  3. Step 3

    Safety and health assessments

    Regular monitoring of vital signs and physical examinations are conducted.

    Laboratory tests include blood tests for hematology and clinical chemistry, urinalysis, and urine cytology.

    Antibodies to dystrophin and viltolarsen are checked.

    A 12-lead electrocardiogram (ECG) and renal ultrasound are performed.

    Any treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) are recorded.

  4. Step 4

    Physical performance tests

    The Time to Stand Test (TTSTAND) measures how quickly the patient can stand up.

    The Time to Run/Walk 10 Meters Test (TTRW) assesses the time taken to cover 10 meters.

    The Six-minute Walk Test (6MWT) evaluates the distance walked in six minutes.

    The North Star Ambulatory Assessment (NSAA) is used to assess motor abilities.

    The Time to Climb 4 Stairs Test (TTCLIMB) measures the time taken to climb four stairs.

    Muscle strength is measured using a hand-held device, focusing on elbow and knee movements on the dominant side.

Who can join the trial?

3 criteria

  • The patient must be a boy who has completed the previous study called NS-065/NCNP-01-301.
  • The patient's parent(s) or legal guardian(s) must have given written permission for the patient to join the study. This is called **informed consent**. In some places, the patient might also need to agree, either by writing or speaking, depending on local rules.
  • The patient and their parent(s) or guardian(s) must be willing and able to attend scheduled visits, follow the plan for taking the study medicine, and participate in study procedures.

Who cannot join the trial?

4 criteria

  • Participants must be boys, as the study does not include girls.
  • Participants should have completed a previous study called NS-065/NCNP-01-301.
  • Participants must be within a specific age range, which is not specified here.
  • Participants should not belong to any vulnerable population that is not specified here.
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Investigated drugs

Viltolarsen is a medication being studied for its safety and effectiveness in treating boys with Duchenne Muscular Dystrophy (DMD). It is given through an intravenous (IV) infusion once a week. The goal of this study is to see how well boys tolerate this treatment and to monitor any side effects.

What is already known about the treatment

Viltolarsen – This medication is administered intravenously, typically given as a weekly infusion. It is currently being studied in clinical trials, particularly for its safety and effectiveness in treating Duchenne Muscular Dystrophy (DMD) in boys. Viltolarsen is designed to help produce a functional version of the dystrophin protein, which is lacking in individuals with DMD, by skipping exon 53 during the genetic transcription process. It is classified as an antisense oligonucleotide, a type of drug that targets specific genetic sequences to modify protein production.

Investigated diseases

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. Symptoms usually begin in early childhood, with affected individuals experiencing difficulty in walking, running, and jumping. As the disease progresses, muscle weakness spreads to the arms, neck, and other areas, leading to increased difficulty in performing daily activities. Over time, individuals may lose the ability to walk and require the use of a wheelchair. The progression of muscle weakness can also affect the heart and respiratory muscles.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2023-507146-91-00Protocol codeNS-065/NCNP-01-302Estimated enrolment74 patientsSponsorNs Pharma Inc.

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