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Efficacy and Safety of Givinostat for Non-Ambulant Patients with Duchenne Muscular Dystrophy

Fast replyInvestigationalNo 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 studying Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness that primarily affects males. The study will test a medication called givinostat compared to placebo in non-ambulant patients (those who are wheelchair-bound and unable to walk independently). The purpose of this research is to evaluate whether givinostat can reduce muscle decline in patients with DMD who have lost their ability to walk.

The trial is designed for children and adolescent males with genetically confirmed DMD who use wheelchairs. Participants will receive either givinostat or placebo for 18 months. During this time, their upper limb function will be assessed using a standardized test called the Performance of the Upper Limb test. Additionally, breathing capacity will be monitored through measurements of Peak Expiratory Flow (the maximum speed of breath when exhaling) and Forced Vital Capacity (the total amount of air that can be forcibly exhaled).

Throughout the study, researchers will track how the medication affects muscle function in the arms and hands, as well as respiratory function. They will also monitor for any side effects that might occur during treatment, including changes in vital signs, laboratory test results, and heart function.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment and Randomization

    After joining the study, you will be randomly assigned to receive either givinostat (the study medication) or a placebo (a substance that looks like the medication but has no active ingredients).

    Neither you nor your doctor will know which treatment you are receiving. This is called a 'double-blind' study design.

  2. Step 2

    Treatment Period

    You will take givinostat hydrochloride monohydrate (also called ITF2357) 10 mg/mL oral suspension or a matching placebo for a total of 18 months.

    The medication or placebo will be in liquid form (oral suspension) that you will take by mouth according to the dosing schedule provided by your study doctor.

    Throughout this period, you will continue taking your stable dose of corticosteroids that you have been on for at least 6 months before starting the study.

  3. Step 3

    Study Assessments

    During the 18-month treatment period, you will have regular visits to assess how the treatment is affecting your muscle function.

    The main test will be the Performance of the Upper Limb (PUL) test, which measures arm and hand function.

    You will also have breathing tests including Peak Expiratory Flow (measuring how fast you can breathe out) and Forced Vital Capacity (measuring how much air you can exhale).

    Your vital signs, laboratory tests, heart tests (ECG and ECHO), height, and weight will be monitored regularly.

  4. Step 4

    Safety Monitoring

    Throughout the study, you will be monitored for any side effects or adverse events that might occur.

    The study team will track if you experience diarrhea, respiratory infections, or require assisted ventilation.

    If you are taking medications for DMD-associated heart problems (cardiomyopathy), these will be continued during the study.

  5. Step 5

    End of Study and Follow-up

    After completing the 18-month treatment period, you will have a final assessment to evaluate the overall effects of the treatment.

    There will be a follow-up visit after you stop taking the study medication to check on your health.

    The study is expected to run until December 2027, though your individual participation will last approximately 18 months plus the follow-up period.

Who can join the trial?

11 criteria

  • Must be male, aged between 9 and 18 years old at screening
  • Able to provide informed consent/assent in writing (signed by the patient and/or parent/legal guardian)
  • Must have a genetic diagnosis of Duchenne muscular dystrophy (DMD) - a genetic disorder causing progressive muscle weakness
  • Must be non-ambulant (unable to walk), which means:
    • Being wheelchair bound, and
    • Unable to perform the 10-meter walk/run test, or
    • Unable to complete the 10-meter walk/run test in 30 seconds or less without support or devices
    • Must score between 3 and 6 on the Performance of the Upper Limb test (PUL version 2.0) entry items - a test that measures upper limb function
    • If taking medication for DMD-related heart problems, must be on a stable dose for at least 1 month before starting the study
    • Must be on stable corticosteroids (anti-inflammatory medications), which means:
      • Taking corticosteroids for at least 6 months before starting the study
      • No significant change in dose or schedule for at least 6 months before starting the study (except for adjustments due to weight changes)
      • Willing to use adequate contraception from the randomization visit through 3 months after the last dose of the study drug

Who cannot join the trial?

13 criteria

  • Patients who are unable to sit without support for at least one hour.
  • Patients who need mechanical ventilation for more than 16 hours per day.
  • Patients who cannot perform the necessary breathing tests (forced vital capacity tests) properly.
  • Patients with severe heart problems (heart failure where the heart can't pump blood effectively).
  • Patients with kidney problems where kidney function is less than 60% of normal.
  • Patients with liver disease or abnormal liver function tests.
  • Patients with a condition that makes them prone to bleeding or who have low blood cell counts.
  • Patients with an active infection.
  • Patients with a history of cancer in the past 5 years.
  • Patients who are taking medications that interact with the study drug.
  • Patients who participated in another clinical trial within the past 3 months.
  • Patients who have a condition that would make it unsafe to participate in the study.
  • Patients who cannot follow the study procedures.
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Investigated drugs

Givinostat is a medication being studied for the treatment of Duchenne Muscular Dystrophy (DMD) in patients who are no longer able to walk. It is designed to help reduce muscle decline in these patients. The medication works by targeting specific proteins that may help improve muscle function and slow down the progression of the disease.

What is already known about the treatment

Givinostat – This is an oral medication taken by mouth that is currently under investigation as a potential treatment for Duchenne muscular dystrophy (DMD), particularly in non-ambulant patients who can no longer walk. Givinostat works at the molecular level as a histone deacetylase (HDAC) inhibitor, which helps regulate gene expression and may reduce inflammation and fibrosis in dystrophic muscles while promoting muscle regeneration. In clinical trials, researchers are evaluating whether givinostat can effectively slow down muscle decline in DMD patients, potentially improving muscle function and quality of life for those affected by this progressive genetic disorder. The medication belongs to the pharmacological class of epigenetic modulators, which influence how genes are expressed without changing the DNA sequence itself.

Investigated diseases

Duchenne muscular dystrophy (DMD) - Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects skeletal muscles, initially those closest to the core of the body, before impacting muscles that control breathing and heart function. DMD typically becomes apparent in early childhood, with affected children experiencing difficulties in motor development, frequent falls, and challenges in running and climbing stairs. As the disease progresses, muscles continue to weaken, leading to reduced mobility and eventual loss of walking ability. The disease also causes respiratory and cardiac muscle impairment over time. DMD is caused by mutations in the dystrophin gene, resulting in absent or abnormal dystrophin protein, which is vital for maintaining muscle cell structure and function.
Trial detailsLast updated 4 Oct 2026
Age0-17PhasePhase IIITrial ID2023-503521-19-00Protocol codeDSC/14/2357/50Estimated enrolment168 patientsSponsorItalfarmaco S.p.A.

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