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

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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 trial will use a treatment called Givinostat, which is an oral suspension medication. Givinostat is a type of drug known as a histone deacetylase (HDAC) inhibitor, which is being investigated for its potential to help manage symptoms in young boys with DMD.

The purpose of the study is to understand how Givinostat is processed in the body and to evaluate its safety when used over a long period. The study is divided into two phases: the Core Phase and the Extension Phase. In the Core Phase, participants will receive Givinostat to see how their bodies absorb and process the medication. In the Extension Phase, the focus will be on monitoring the safety and any side effects of the medication over a longer duration.

Participants in the study will be young boys aged between 2 and less than 6 years old who have been diagnosed with DMD. The study will involve regular check-ups and assessments to monitor the effects of Givinostat on the participants' health and well-being. The trial aims to provide valuable information on the potential benefits and risks of using Givinostat in managing Duchenne muscular dystrophy in young children.

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

    Core phase initiation

    The study begins with the core phase, which lasts for 48 weeks. During this phase, the focus is on understanding how the body processes the medication givinostat when taken regularly.

    Participants will receive givinostat as an oral suspension. The exact dosage and frequency will be determined by the study team based on individual needs and responses.

  2. Step 2

    Core phase monitoring

    Throughout the core phase, regular monitoring will occur to assess the concentration of givinostat in the blood. This includes measuring the highest concentration reached and how long it takes for the body to eliminate the medication.

    Participants will also be monitored for any side effects or changes in health, including vital signs, laboratory tests, and heart function through electrocardiograms (ECGs).

  3. Step 3

    Core phase assessment

    Physical function will be evaluated using specific scales depending on the participant's age. For younger participants, the Bayley III Gross Motor scale will be used, while older participants will be assessed using the North Star Ambulatory Assessment.

    Additional assessments may include changes in outcomes related to daily activities and overall health status.

  4. Step 4

    Extension phase initiation

    After completing the core phase, participants may enter the extension phase, which continues for up to 144 weeks. This phase focuses on the long-term safety and tolerability of givinostat.

    Participants must have completed the core phase and attended the final visit of that phase to be eligible for the extension phase.

  5. Step 5

    Extension phase monitoring

    During the extension phase, ongoing monitoring will continue to track any side effects and overall health changes. This includes regular checks of vital signs, laboratory tests, and ECGs.

    The study will also document any adverse events, which are any unwanted effects experienced during the trial.

  6. Step 6

    Extension phase assessment

    Participants' physical function and health outcomes will be assessed periodically to understand the long-term impact of givinostat treatment.

    The study aims to ensure that the medication remains safe and effective over an extended period.

Who can join the trial?

8 criteria

  • Must be a male child aged between 2 and less than 6 years old at the time of screening. Children who are 6 years old at screening cannot join the study.
  • Must have written consent from a parent or legal guardian. If the child is old enough, they may also need to give their written agreement, depending on local rules.
  • Must have a genetic diagnosis of Duchenne muscular dystrophy (DMD), which is a specific condition affecting muscles.
  • If currently taking oral systemic corticosteroids (a type of medication), the dose should not have changed significantly for at least 3 months before starting the study drug. Changes due to body weight are allowed.
  • If not currently taking corticosteroids, the child should not start taking them during the first 48 weeks of the study.
  • For the extension phase, the child must have completed the initial 48-week study and attended the final treatment visit.
  • For the extension phase, written consent and/or agreement must be given again by the parent, legal guardian, and/or the child, as required by local rules.
  • For the extension phase, if taking corticosteroids, the treatment should remain stable without significant changes, except for adjustments due to body weight. If not taking corticosteroids during the initial phase, they can start based on the doctor's medical judgment.

Who cannot join the trial?

10 criteria

  • Only boys can participate, so girls are excluded.
  • Participants must be younger boys, so older boys are excluded.
  • Participants must have **Duchenne muscular dystrophy (DMD)**, so those without this condition are excluded.
  • Participants must be able to safely take the study medication, so those with certain health issues that make it unsafe are excluded.
  • Participants must not have any other medical conditions that could interfere with the study.
  • Participants must not be taking other medications that could interfere with the study medication.
  • Participants must not have had any recent surgeries or medical procedures that could affect the study.
  • Participants must not have any allergies to the study medication or its ingredients.
  • Participants must not have any history of drug or alcohol abuse.
  • Participants must not have any mental health conditions that could interfere with their ability to participate in the study.
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Investigated drugs

Givinostat is a medication being studied for its effects on boys with Duchenne Muscular Dystrophy (DMD) who are between 2 and 6 years old. The trial aims to understand how the body processes this medication and to evaluate its safety when used over a long period. Givinostat is being tested to see if it can be safely administered to young children with DMD and to monitor any potential side effects that may occur during the treatment.

What is already known about the treatment

Givinostat – Givinostat is administered orally and is currently being studied in clinical trials for its effects on Duchenne muscular dystrophy (DMD) in young boys. It is in the investigational stage, with ongoing research to understand its pharmacokinetics and safety profile. The main therapeutic indication for Givinostat is the treatment of 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. It is classified pharmacologically as a histone deacetylase inhibitor.

Investigated diseases

Duchenne muscular dystrophy (DMD) – Duchenne muscular dystrophy 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 increased difficulty in movement. Over time, individuals with DMD may lose the ability to walk and require assistance with daily activities. The progression of muscle weakness can also affect the heart and respiratory muscles, impacting overall health and mobility.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IITrial ID2024-511823-32-00Protocol codeDSC/14/2357/52Estimated enrolment18 patientsSponsorItalfarmaco S.p.A.

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