In short
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.
Key points
- Viltolarsen clinical trial data here focus on boys with Duchenne Muscular Dystrophy (DMD). The listed study is a Phase 3 trial and is completed. Researchers mainly looked at safety and tolerability, while also checking vital signs, physical exams, blood and urine tests, ECG results, kidney ultrasound, and antibodies. The trial included boys who had already finished a previous study, NS-065/NCNP-01-301. In total, 74 participants were enrolled.
Trial overview
The main trial listed for Viltolarsen was NCT04768062, a Phase 3 interventional study in boys with Duchenne Muscular Dystrophy (DMD). 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.
The study title says it was a phase 3 study of Viltolarsen in boys with DMD. The brief summary says the goal was to evaluate safety and tolerability in boys who had completed the NS-065/NCNP-01-301 study.
Who participated
This trial focused on boys with Duchenne Muscular Dystrophy, which is a genetic muscle disease that causes progressive weakness. Participation was limited to boys who had already completed the earlier NS-065/NCNP-01-301 study.
This means the study did not recruit a broad patient group from the general public. Instead, it followed a specific group from a prior trial to continue learning about the treatment in the same condition.
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. These checks help researchers look for changes in general health and body function during the study.
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. In simple terms, this means researchers looked at immune response, heart rhythm, kidney structure, and any new health problems that appeared during treatment.
The trial’s main purpose was safety and tolerability rather than a direct comparison with another treatment. That makes it a study focused on how the treatment is handled by the body and what monitoring findings appear over time.
Trial status and size
NCT04768062 is listed as completed. The enrollment number was 74 participants.
Because the study is completed, the listed trial record reflects a finished research project rather than an ongoing recruitment effort. The available data show a relatively small, targeted group of boys with DMD who had already taken part in an earlier study.
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. The research centered on safety checks, routine medical monitoring, and signs of how the treatment was tolerated.
The study also shows how clinical trials often follow patients over time after an earlier study ends. In this case, the trial continued observation in boys who had already completed a previous Viltolarsen study.