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

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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 Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness and degeneration. The treatment being investigated is called GIVINOSTAT, which is administered as an oral suspension. The purpose of the study is to evaluate the long-term safety, tolerability, and effectiveness of GIVINOSTAT in patients with Duchenne Muscular Dystrophy who have previously participated in other studies involving this medication.

During the study, all participants will receive the same investigational drug, GIVINOSTAT. The study will monitor the type, frequency, and severity of any side effects that may occur. For patients who can walk, changes in physical function and muscle strength will be assessed. For those who cannot walk, changes in physical function, daily living activities, and upper limb strength will be evaluated. Additionally, the study will track changes in respiratory function and quality of life for all participants.

The study will continue over several years, with assessments taking place at specific intervals, such as after 48 weeks and then annually. The goal is to gather comprehensive data on how GIVINOSTAT affects patients with Duchenne Muscular Dystrophy over an extended period. This information will help determine the long-term benefits and risks of using GIVINOSTAT as a treatment option for this condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation requires previous involvement in a GIVINOSTAT study for Duchenne Muscular Dystrophy (DMD) or screening in study DSC/14/2357/48.

    Eligibility includes being at least 6 years old and able to provide written consent or assent.

  2. Step 2

    Medication administration

    The investigational drug, GIVINOSTAT, is administered as an oral suspension.

    The dosage and frequency are determined by the study protocol and are consistent throughout the study duration.

  3. Step 3

    Monitoring and assessments

    Regular assessments are conducted to monitor the type, incidence, and severity of any adverse effects.

    For ambulant patients, physical function is evaluated using tests like the 6-minute walk test (6MWT) and muscle strength assessments at week 48 and then yearly.

  4. Step 4

    Non-ambulant patient assessments

    Non-ambulant patients undergo evaluations of physical function using the Egen Klassifikation (EK) score and muscle strength assessments.

    Changes in daily living activities are measured using the Barthel Index at week 48 and then yearly.

  5. Step 5

    General assessments

    All patients have their respiratory function and quality of life assessed at week 48 and then yearly.

    The study tracks major disease milestones such as age at loss of ambulation and need for respiratory support.

  6. Step 6

    Study duration

    The study is expected to continue until December 3, 2027.

    Assessments and monitoring occur regularly throughout the study period to ensure safety and efficacy.

Who can join the trial?

6 criteria

  • The study is for patients with Duchenne Muscular Dystrophy (DMD).
  • Participants must have been part of a previous study with the drug GIVINOSTAT for DMD and completed the study visit, or they must have been screened in a specific study and met all the necessary criteria without being excluded.
  • Participants must be at least 6 years old.
  • Participants need to be able to provide written agreement to join the study, which can be signed by them or their parent/legal guardian, depending on local rules.
  • Participants must agree to use reliable birth control methods during the study and for three months after the last dose of the study drug. Acceptable methods include:
    • True abstinence, meaning no sexual intercourse, if it fits the participant's lifestyle.
    • Using a condom with spermicide, and the female partner must also use a reliable birth control method, such as birth control pills, patches, injections, implants, or a barrier method like a diaphragm with spermicide.

Who cannot join the trial?

10 criteria

  • Only males can participate in the study. Females are not eligible.
  • Participants must not have any other serious health conditions that could interfere with the study.
  • Participants must not have any allergies or reactions to the study medication or similar drugs.
  • Participants must not be taking any other medications that could interfere with the study medication.
  • Participants must not have participated in another clinical trial recently.
  • Participants must not have any history of drug or alcohol abuse.
  • Participants must not have any mental health conditions that could affect their ability to participate in the study.
  • Participants must not have any infections or illnesses that could affect the study results.
  • Participants must not have any heart problems that could be worsened by the study medication.
  • Participants must not have any liver or kidney problems that could affect how the body processes the study medication.
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Investigated drugs

Givinostat is a medication being studied for its long-term safety, tolerability, and effectiveness in patients with Duchenne Muscular Dystrophy (DMD). This study includes patients who have previously been treated with Givinostat in other studies, as well as new patients who meet specific criteria. The goal is to understand how well patients tolerate the medication over a longer period and to monitor any potential side effects while assessing its benefits in managing DMD symptoms.

What is already known about the treatment

Givinostat – Givinostat is administered orally in the form of tablets. It is currently being studied in clinical trials for its long-term safety, tolerability, and effectiveness in treating Duchenne Muscular Dystrophy (DMD). The medication is primarily indicated for patients with DMD, a genetic disorder characterized by progressive muscle degeneration. At the molecular level, Givinostat works by inhibiting enzymes known as histone deacetylases, which play a role in gene expression and protein function, potentially reducing inflammation and muscle damage. It is classified as a histone deacetylase inhibitor in pharmacology.

Investigated diseases

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. Symptoms usually begin in early childhood, with affected children experiencing difficulty in walking, running, and jumping. As the disease progresses, muscle weakness spreads to the arms, neck, and other areas, leading to loss of ambulation. Over time, individuals with DMD may also experience respiratory and cardiac complications due to muscle deterioration. The progression of the disease varies, but it generally leads to significant physical disability.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-504520-26-00Protocol codeDSC/14/2357/51Estimated enrolment222 patientsSponsorItalfarmaco S.p.A.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).