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

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What is this study about?

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.

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 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 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 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 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 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 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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hospital Universitario Y Politecnico La Fe Valencia Spain
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Hospital Universitario Puerta De Hierro De Majadahonda Majadahonda Spain
Nosokomeio Paidon I Agia Sofia Athens Greece
Fakultni Nemocnice Brno Brno Czechia
Stichting Radboud University Medical Center Nijmegen The Netherlands
Universitair Ziekenhuis Gent Gent Belgium
Children’s Health Ireland Dublin Ireland
Lpikz Ujrhnhdzsgjr Mrdxcxs Cypjntv (bijyk Leiden The Netherlands
Fhlonzxp nwvfcfmqg Msodr a Hzanaey Prague Czechia

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
31.10.2024
Czechia Czechia
Not recruiting
31.10.2024
Greece Greece
Not recruiting
31.10.2024
Ireland Ireland
Not recruiting
31.10.2024
Spain Spain
Not recruiting
31.10.2024
The Netherlands The Netherlands
Not recruiting
31.10.2024

Trial locations

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.

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 ID:
2024-512828-12-00
Protocol code:
SNT-IV-VAM-011
Trial Phase:
Therapeutic confirmatory (Phase III)

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