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Ongoing Clinical Trials for Duchenne Muscular Dystrophy

There are currently 22 clinical trials studying new treatments for Duchenne muscular dystrophy across Europe. These trials are testing different approaches including gene therapies, medications that help muscle cells produce dystrophin protein, and treatments to improve muscle function and reduce complications. The studies involve both children who can still walk and those who use wheelchairs, testing various innovative treatments aimed at slowing disease progression and improving quality of life.

Clinical trial locations

Long-Term Safety and Efficacy Study of Givinostat for Patients with Duchenne Muscular Dystrophy Previously Treated with Givinostat

This study focuses on patients who have already taken part in previous research with the medication givinostat. The trial aims to understand how safe and effective this treatment is over a longer period of time. Givinostat is given as a liquid medication taken by mouth.

Who can participate: Boys at least 6 years old with confirmed genetic diagnosis who have previously been in a givinostat study can join. Participants must agree to use reliable birth control methods and provide consent along with their parents or guardians.

What the study involves: All participants receive givinostat oral suspension. Regular check-ups monitor any side effects and track changes in physical abilities such as walking distance, muscle strength, breathing function, and quality of life. For boys who can walk, assessments include tests like the 6-minute walk test. For those using wheelchairs, evaluations focus on upper body strength and daily activities. Major health milestones like loss of ability to walk or need for breathing support are also tracked.

Study medication: Givinostat works by affecting specific enzymes in the body that influence how genes work in muscle cells, potentially reducing inflammation and muscle damage. It belongs to a class of drugs called histone deacetylase inhibitors.

Long-term Safety Study of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy Who Previously Received SRP-9001

This trial follows patients who previously received a gene therapy called delandistrogene moxeparvovec in an earlier study. The research aims to monitor the long-term safety of this treatment and track how patients’ physical abilities change over time.

Who can participate: Male patients who received this gene therapy in a previous clinical study can join. They or their legal caregivers must be able to understand and follow the study schedule and requirements.

What the study involves: The study monitors participants over several years, watching for any side effects or health changes. Regular assessments track physical abilities including walking and running tests, upper body function, breathing capacity, and heart health using special imaging scans. Muscle tissue analysis measures how much dystrophin protein the treatment helped produce. The follow-up period extends up to five years after receiving the gene therapy.

Study medication: Delandistrogene moxeparvovec is a gene therapy that delivers a working copy of the dystrophin gene to muscle cells. It aims to help the body produce dystrophin protein, which is missing or deficient in people with this condition. The treatment is given once as an intravenous infusion.

Study of BMN 351 given by intravenous infusion to evaluate safety and tolerability in patients with Duchenne Muscular Dystrophy

This research tests a new medication called BMN 351 in patients with a specific genetic form of the condition that could benefit from exon 51 skipping. The study aims to find out if the medication is safe and well-tolerated at different dose levels.

Who can participate: Boys aged 4 to 10 years with confirmed genetic diagnosis suitable for exon 51 skipping treatment can join. Participants must be able to walk independently, complete a 10-meter walk in 8 seconds or less, not need daytime breathing support, and have been taking corticosteroid medications for at least 12 weeks. Normal urine test results are also required.

What the study involves: BMN 351 is given through an intravenous line at gradually increasing doses to determine the safest and most effective amount. Regular check-ups include physical exams, safety tests, heart monitoring, and measurements of how the body processes the medication. Blood, urine, and muscle tissue samples help researchers understand how the drug works in the body. Participants continue taking their current corticosteroid medications throughout the study.

Study medication: BMN 351 is designed to target a specific part of genetic material to help improve muscle function in this genetic condition. It is given as a diluted solution directly into the bloodstream.

Study of ENTR-601-44 in Children with Duchenne Muscular Dystrophy Who Are Eligible for Exon 44 Skipping Treatment

This study evaluates a new treatment called ENTR-601-44 for children with a specific genetic type of the condition that could benefit from exon 44 skipping. The research examines both safety and how well the treatment works.

Who can participate: Male children and adolescents aged 4 to 20 years with genetic confirmation suitable for exon 44 skipping can join. They must be able to walk and have good upper body function as measured by specific tests. Adequate muscle tissue for biopsy procedures is also required.

What the study involves: The study has two parts. In Part A, participants receive either ENTR-601-44 or placebo through intravenous infusion. Neither participants nor doctors know who receives which treatment. Regular assessments include physical exams, blood tests, heart monitoring, and muscle biopsies to measure protein levels. Tests also evaluate walking ability, climbing stairs, and upper body strength. In Part B, all participants receive the actual medication.

Study medication: ENTR-601-44 aims to help the body produce a form of dystrophin protein through the exon skipping process. The goal is to improve muscle function and potentially slow disease progression.

Study of ENTR-601-45 Safety and Effectiveness in Patients with Duchenne Muscular Dystrophy Who Can Skip Exon 45

This trial tests ENTR-601-45 in patients whose genetic type could benefit from exon 45 skipping. The research evaluates safety and effectiveness of this new treatment approach.

Who can participate: Male participants aged 4 to 20 years with confirmed genetic changes suitable for exon 45 skipping can join. They must demonstrate ability to walk and perform upper body tasks as measured by specific tests. Adequate muscle tissue for biopsy is also required.

What the study involves: Participants receive either ENTR-601-45 or placebo through intravenous infusion in a blinded manner, meaning neither they nor their doctors know which treatment they receive. Regular monitoring includes blood samples to measure medication levels, urine collection, muscle biopsies to assess protein production, and tests checking for antibodies. Physical assessments measure walking ability, running speed, stair climbing, and upper body function. At week 25, changes from the starting point are evaluated.

Study medication: ENTR-601-45 works through the exon skipping mechanism to help cells produce a functional form of dystrophin protein. This approach aims to improve muscle function in patients with specific genetic mutations.

Study of Microdystrophin (GNT0004) Gene Therapy for Duchenne Muscular Dystrophy in Boys Who Can Walk

This study tests a gene therapy called GNT0004 in boys who can still walk. The research is divided into three parts: finding the right dose, comparing effects against placebo, and long-term safety monitoring.

Who can participate: Boys aged 6 to 10 years who can walk and have genetic confirmation of the condition can join. They must be part of a specific previous study, have body weight at or below the 75th percentile for height, and have mutations outside exons 1-17 of the gene. They cannot have certain mutations affecting specific parts of the gene.

What the study involves: The study starts with finding the safest and most effective dose of GNT0004. Then participants receive either the gene therapy or placebo for one year while researchers compare the effects. Finally, long-term follow-up continues to monitor safety and effectiveness. The treatment is given once through an intravenous infusion. Additional medications may be given including eculizumab, sirolimus, and prednisolone. Regular assessments track muscle function, overall health, and any side effects.

Study medication: GNT0004 is a gene therapy that delivers a modified version of the dystrophin gene to muscle cells. It aims to help the body produce microdystrophin, a smaller but functional version of the protein that is missing in this condition.

Study of SGT-003 Gene Therapy for Safety and Tolerability in Walking Males with Duchenne Muscular Dystrophy

This research examines a gene therapy called SGT-003 in boys who can walk without assistance. The study focuses on safety and how well patients tolerate the treatment.

Who can participate: Boys aged 4 to less than 7 years or 7 to less than 12 years who can walk without help can join. They must have genetic confirmation of the condition, test negative for certain antibodies, and have been taking stable doses of prednisone or deflazacort for at least 12 weeks. Specific requirements for walking and rising tests must be met, and body weight must be 50 kg or less.

What the study involves: Participants receive a single dose of SGT-003 gene therapy through an intravenous infusion. Regular monitoring tracks any side effects and assesses changes in muscle function through various tests. For boys who can walk, assessments include the North Star Ambulatory Assessment, 6-minute walk test, and muscle strength measurements. All participants have breathing function and heart health evaluated. Muscle tissue samples help measure microdystrophin protein levels.

Study medication: SGT-003 uses a specially designed virus to deliver the human microdystrophin gene to muscle cells. The goal is to help the body produce this protein, which is missing or not working properly in the condition.

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

This trial follows boys who completed earlier studies with vamorolone medication. The research monitors long-term safety and effectiveness, with special attention to bone health.

Who can participate: Boys who finished previous vamorolone studies and are currently taking the medication can join. They must be willing to follow the study schedule and procedures along with their parents or guardians. Participants must agree to use reliable birth control methods during the study.

What the study involves: All participants receive AGAMREE vamorolone oral suspension. The primary focus is monitoring for bone fractures, particularly in the spine. Regular assessments include X-rays, measurements of height, weight, and growth milestones. Physical function tests measure how quickly participants can stand and how far they can walk in six minutes. The North Star Ambulatory Assessment evaluates overall motor function. Blood tests monitor sugar levels and morning cortisol. Any side effects are carefully tracked.

Study medication: Vamorolone works by reducing inflammation and stabilizing cell membranes, potentially helping reduce muscle damage. It is a type of steroidal anti-inflammatory drug designed to have fewer side effects than traditional steroids while still providing benefits.

Study on Metoprolol for Preventing Heart Disease in Boys with Duchenne Muscular Dystrophy Aged 8-17

This study examines whether adding metoprolol to standard heart care can help prevent or slow heart problems in boys with the condition. Heart muscle weakness is a serious concern that can develop over time.

Who can participate: Boys aged 8 to 16 years with confirmed genetic diagnosis can join. They must be able to take oral medication and have been taking ACE inhibitor heart medications at required doses for at least 30 days. Parents or guardians must provide written consent, and boys over 16 years must also agree to participate.

What the study involves: Participants are randomly assigned to receive either metoprolol or placebo in addition to their regular heart treatments. Neither participants nor doctors know who receives which treatment. The medication is taken orally in tablet form. Regular heart function tests using echocardiography measure how well the heart pumps blood. The study monitors how long it takes for heart problems to develop and tracks changes in heart function over time. Participants continue their current corticosteroid or vamorolone treatments throughout the study.

Study medication: Metoprolol is a beta-blocker that works by slowing the heart rate and reducing the force of heart contractions. This decreases the heart’s workload, potentially helping protect the heart muscle from weakening.

Study on the Effects of Satralizumab in Children and Adolescents with Duchenne Muscular Dystrophy

This research tests whether satralizumab can improve bone health in children and adolescents with the condition. The study focuses on bone mineral density, which measures bone strength.

Who can participate: Boys aged 8 to 16 years with the condition can join. They must be taking daily corticosteroid medications for at least 12 months at stable doses. The study includes two groups: those who have never had fractures and can walk, and those with a history of fractures (some may use wheelchairs). Specific requirements for walking ability and fracture history determine which group participants join.

What the study involves: Participants receive either satralizumab or placebo through injections under the skin. Regular DEXA scans measure changes in bone mineral density at weeks 24 and 52. Blood tests, physical exams, heart monitoring, and tests of breathing function occur throughout the study. For those who can walk, physical ability tests measure strength and movement. Those using wheelchairs have upper body function assessed. The study tracks any new fractures and monitors for side effects.

Study medication: Satralizumab is a monoclonal antibody that blocks a specific protein involved in inflammation. By targeting this pathway, it may help improve bone health and reduce symptoms associated with the condition.

Study on the Safety and Effects of Givinostat in Young Children with Duchenne Muscular Dystrophy (Ages 2 to Less Than 6 Years)

This trial studies givinostat in very young children with the condition. The research examines how the body processes the medication and monitors safety over an extended period.

Who can participate: Boys aged 2 to less than 6 years with genetic confirmation of the condition can join. Parents or guardians must provide written consent. If currently taking corticosteroids, the dose must have been stable for at least 3 months. Those not taking corticosteroids should not start them during the first 48 weeks of the study.

What the study involves: The study has two phases. The Core Phase lasts 48 weeks and focuses on understanding how the body processes givinostat given as oral suspension. Regular monitoring measures medication levels in the blood and tracks any side effects through vital signs, laboratory tests, and heart monitoring. Physical function is evaluated using age-appropriate tests. Children completing the Core Phase can continue into the Extension Phase for up to 144 additional weeks of long-term safety monitoring. In the Extension Phase, corticosteroids can be started if medically appropriate.

Study medication: Givinostat is a histone deacetylase inhibitor that affects how genes work in muscle cells. By inhibiting these enzymes, it may reduce inflammation and protein dysfunction, potentially helping protect muscles.

Summary

The 22 ongoing trials for Duchenne muscular dystrophy represent a diverse range of therapeutic approaches being tested across Europe. A notable concentration of studies is taking place in Italy, which hosts 12 trials, followed by Spain with 11 trials and Belgium with 10 trials. France, Germany, and the Netherlands also serve as significant research hubs with multiple studies each.

Gene therapy approaches feature prominently, with several trials testing medications like delandistrogene moxeparvovec, fordadistrogene movaparvovec, GNT0004, and SGT-003. These therapies aim to deliver working copies of genes to help produce dystrophin or similar proteins. Another major category involves exon-skipping treatments such as ENTR-601-44, ENTR-601-45, eteplirsen, casimersen, golodirsen, DYNE-251, and viltolarsen, which target specific genetic mutations to help cells produce functional protein.

Several studies focus on oral medications that work through different mechanisms. Givinostat appears in multiple trials examining both younger children and long-term safety. Vamorolone is being studied for its potential to provide steroid benefits with fewer side effects, particularly regarding bone health. Other approaches include ATL1102 for non-walking patients, satralizumab for bone health, and metoprolol for heart protection.

The trials encompass a wide age range from infants as young as 6 months to young adults, and include both children who can walk and those who use wheelchairs. This comprehensive approach reflects the evolving understanding of the condition and the need for treatments appropriate to different stages and manifestations of the disease.

Ongoing Clinical Trials on Duchenne muscular dystrophy

  • Study on Eteplirsen for Patients with Duchenne Muscular Dystrophy with Deletion Mutations Amenable to Exon 51 Skipping

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Denmark France Germany Greece Hungary +7

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

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Greece Italy The Netherlands Norway Spain

  • Study of DYNE-251 safety and effectiveness in patients with Duchenne muscular dystrophy who are candidates for exon 51 skipping treatment

    Not recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    Belgium Ireland Italy Spain

  • Study on the Effects of Fordadistrogene Movaparvovec for Patients with Duchenne Muscular Dystrophy

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium France Germany Italy Spain

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