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

Verified siteRegistered drugNo 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 the long-term effects of a treatment called vamorolone in boys with Duchenne Muscular Dystrophy (DMD). DMD is a genetic disorder characterized by progressive muscle weakness and degeneration. The treatment being studied, vamorolone, is an oral suspension that belongs to a group of medications known as glucocorticoids, which are often used to reduce inflammation and suppress the immune system.

The purpose of this study is to gather information on the safety and effectiveness of long-term treatment with vamorolone, particularly in relation to bone health, such as the risk of vertebral fractures. Participants in this study are boys who have previously been involved in other studies with vamorolone and are continuing their treatment. The study will monitor various health aspects over time, including the occurrence of fractures, changes in body weight and height, and other health markers.

Throughout the study, participants will undergo regular assessments to track their progress and any potential side effects. These assessments will help researchers understand how vamorolone affects the body over an extended period. The study aims to provide valuable insights into the long-term use of vamorolone for managing symptoms of Duchenne Muscular Dystrophy.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment

    Participation begins after providing written informed consent. This consent must be given by the participant or their parent/legal guardian.

    Eligibility requires completion of previous studies with vamorolone and current use of the medication on the day of enrollment.

  2. Step 2

    Treatment phase

    The study involves long-term treatment with vamorolone, specifically using AGAMREE 40 mg/ml oral suspension.

    The medication is administered orally. The exact dosage and frequency will be determined by the study protocol and the healthcare provider.

  3. Step 3

    Safety and effectiveness assessments

    The primary focus is on monitoring the safety of long-term treatment, particularly regarding vertebral fractures.

    Regular assessments will include X-rays to evaluate the number of vertebral fractures per 1000 person-years.

  4. Step 4

    Secondary assessments

    Additional evaluations will track the time to first vertebral and non-vertebral fractures, as well as the number of non-vertebral fractures.

    Other assessments include the number of cataracts, growth milestones, and changes in body weight, height, and body mass index (BMI).

  5. Step 5

    Functional assessments

    Functional abilities will be measured using tests such as the Time to Stand Test (TTSTAND) and the 6-Minute Walk Test (6MWT).

    The NorthStar Ambulatory Assessment (NSAA) will also be used to evaluate motor function.

  6. Step 6

    Monitoring adverse events

    The frequency of adverse events (AEs) and serious adverse events (SAEs) will be recorded.

    Laboratory tests will monitor clinically relevant abnormalities, including glycosylated hemoglobin (HbA1c) and morning cortisol levels.

  7. Step 7

    Study duration

    The study is expected to continue until October 31, 2028.

    Participants will be involved in regular assessments and follow-ups throughout the study duration.

Who can join the trial?

5 criteria

  • The patient or the patient's parent(s) or legal guardian must have given written permission to participate in the study. This is called informed consent.
  • The patient must have completed a previous study called VBP15-LTE or VBP15-004 and moved through specific programs like CUP, NPP, or EAP.
  • The patient must be taking a medication called vamorolone on the day they join the study.
  • The patient and their parent or legal guardian must be willing and able to follow the study's schedule, tests, and requirements.
  • The study is only for boys, not girls.

Who cannot join the trial?

7 criteria

  • Patients who are not boys. This study is only for boys.
  • Patients who do not have Duchenne Muscular Dystrophy. This is a condition that affects muscles, making them weaker over time.
  • Patients who are outside the specified age range. The study is for a specific age group.
  • Patients who have certain health conditions that might interfere with the study. These conditions are not specified here but are important for safety.
  • Patients who are taking medications that could affect the study results. Some medications might interfere with the treatment being tested.
  • Patients who have had recent surgeries or medical procedures that could impact the study.
  • Patients who are unable to follow the study procedures or attend required visits. Participation requires commitment to the study schedule.
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Investigated drugs

Vamorolone is a medication being studied for its safety and effectiveness in treating boys with Duchenne Muscular Dystrophy (DMD). This study focuses on understanding how safe it is to use vamorolone over a long period, particularly looking at its effects on bone health, such as the risk of vertebral fractures. Vamorolone is being tested in boys who have already participated in previous studies with this medication.

What is already known about the treatment

Vamorolone – Vamorolone is administered orally and is currently being studied for its long-term safety and effectiveness in treating Duchenne Muscular Dystrophy (DMD). It is in the clinical trial phase, with ongoing research to assess its impact on vertebral fractures in boys with DMD. The medication is primarily indicated for DMD, a genetic disorder characterized by progressive muscle degeneration. Vamorolone works by modulating inflammation and stabilizing cell membranes, which may help in reducing muscle damage. It is classified as a steroidal anti-inflammatory drug, offering potential benefits with fewer side effects compared to traditional steroids.

Investigated diseases

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy is a genetic disorder characterized by progressive muscle weakness and degeneration. It primarily affects boys and usually becomes noticeable in early childhood. The disease is caused by mutations in the dystrophin gene, which leads to the absence of dystrophin, a protein essential for muscle function. As the condition progresses, individuals may experience difficulty walking, frequent falls, and challenges with motor skills. Over time, muscle weakness can spread to the arms, neck, and other areas, eventually affecting the heart and respiratory muscles. The progression of muscle weakness varies among individuals, but it generally leads to a loss of ambulation and increased dependency on assistive devices.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2024-512828-12-00Protocol codeSNT-IV-VAM-011Estimated enrolment43 patientsSponsorSanthera Pharmaceuticals (Schweiz) AG

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