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Vamorolone

Clinical trials are investigating Vamorolone in boys with Duchenne Muscular Dystrophy (DMD). These studies aim to learn about long-term safety and how well treatment works over time. The trial data here focus on children who have already taken part in earlier Vamorolone studies.

Table of Contents

Trial overview

The available clinical trial is an open-label study of Vamorolone in boys with Duchenne Muscular Dystrophy (DMD).[1] It is designed to collect information on long-term treatment, with a focus on both safety and effectiveness.[1]

The trial is interventional, which means the researchers are giving a treatment and then observing what happens during the study.[1] The study is authorised and plans to enroll 65 participants.[1]

Who can participate

This study is for boys with Duchenne Muscular Dystrophy who have already completed prior studies with Vamorolone.[1] This makes the trial a follow-up study for a specific group of patients who have taken part in earlier research.[1]

Because the trial is focused on long-term follow-up, the participants are not a general DMD group, but a selected group with prior Vamorolone study experience.[1]

Study design and phase

The study is in Phase 3.[1] Phase 3 studies usually involve more participants than early trials and help researchers learn more about how well a treatment works and how safe it is in a broader group.[1]

Because this is an open-label trial, there is no blinding in the study data provided.[1] In simple terms, the treatment is known during the study rather than hidden from participants and researchers.[1]

What is being measured

The main outcome is the number of vertebral fractures per 1000 person-years, based on X-ray central reading.[1] Vertebral fractures are breaks in the bones of the spine, and central reading means experts review the X-rays using the same method for all participants.[1]

This endpoint shows that the study is especially interested in bone safety during long-term treatment.[1] The brief study summary also says the goal is to evaluate safety of long-term treatment with Vamorolone in boys with DMD regarding vertebral fractures.[1]

The GUARDIAN Study

The trial title names it as the GUARDIAN Study.[1] It is an open-label study to collect safety and effectiveness information on long-term treatment with Vamorolone in boys with DMD who have completed prior studies with Vamorolone.[1]

In patient terms, this means the study is following children over time to see how they do after earlier Vamorolone research.[1] The main focus is not a new comparison with another drug, but ongoing monitoring of outcomes during continued treatment.[1]

Trial ID Phase Condition studied Status Enrollment
2024-512828-12-00 Phase 3 Duchenne Muscular Dystrophy Authorised 65

FAQ

  • What is being studied in Vamorolone clinical trials? The trial is studying long-term treatment with Vamorolone in boys with Duchenne Muscular Dystrophy. It looks mainly at safety and how effective the treatment is over time.
  • Who can take part in the study? The study is for boys with Duchenne Muscular Dystrophy who have already completed earlier Vamorolone studies. This means it is meant for a specific group of patients already known to the research team.
  • What phase is this Vamorolone trial? This study is a Phase 3 trial. Phase 3 trials usually look at a treatment in a larger group of people to better understand its benefits and safety.
  • What result is the trial measuring? The main result is the number of vertebral fractures per 1000 person-years, based on central X-ray reading. Vertebral fractures are breaks in the bones of the spine.
  • What is the goal of the GUARDIAN Study? The GUARDIAN Study aims to collect safety and effectiveness information during long-term treatment with Vamorolone. It focuses especially on vertebral fractures in boys with Duchenne Muscular Dystrophy.

Ongoing Clinical Trials on Vamorolone

  • Study on Long-Term Safety and Effectiveness of Vamorolone in Boys with Duchenne Muscular Dystrophy Who Completed Previous Vamorolone Studies

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Czechia Greece Ireland The Netherlands Spain

Glossary

  • Duchenne Muscular Dystrophy (DMD): A genetic disease that causes muscles to become weaker over time, usually starting in childhood.
  • Open-label study: A study in which the participants and researchers know which treatment is being given.
  • Long-term treatment: Treatment given over a long period of time so researchers can learn about lasting effects.
  • Safety: How well a treatment can be used without causing unacceptable harm.
  • Effectiveness: How well a treatment works in real study conditions.
  • Phase 3: A late stage of clinical research that usually includes more participants and checks safety and benefit more carefully.
  • Interventional study: A study where researchers give a treatment and observe what happens.
  • Enrollment: The number of participants planned for the study.
  • Vertebral fractures: Breaks in the bones of the spine.
  • X-ray central reading: A special review of X-rays by experts using the same method for all participants.
  • Person-years: A way to measure events in a study that takes both the number of people and the time they are followed into account.

References

  1. https://clinicaltrials.gov/study/2024-512828-12-00