Back to search results

Givinostat for Long-Term Safety in Duchenne Muscular Dystrophy

This study is looking at the long-term safety and how well Givinostat works in boys and men with Duchenne Muscular Dystrophy (DMD). Givinostat is given as a liquid by mouth twice a day. You can join this study if you are a male, aged 7 or older, and have already participated in a previous Givinostat study for DMD. To be eligible, your vastus lateralis muscle fat fraction (VL MFF) must be either 5% or less, or more than 30%. The main goal is to track any side effects you might experience. The study plans to enroll 206 participants, but its current status is unclear.

Study design
This is an open-label study, meaning both you and the study team will know you are receiving Givinostat. It aims to enroll 206 male participants who have previously taken Givinostat.
What's involved
You will take Givinostat orally twice daily. Your dose may be adjusted based on your weight and platelet counts, which will be monitored frequently, especially during the first two months.
Compensation
Not stated in the trial record.
Follow-up
Your safety and tolerability will be measured throughout the study completion, which is an average of 1 year.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT03373968

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Recruiting
PHASE2Ages 7+InterventionalTreatment
Italfarmaco
~206 participants
Updated 2026-01-21 on ClinicalTrials.gov
What's tested:Givinostat

At a glance

Recruiting sites
3 of 39 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Measured over Through study completion, an average of 1 year

Conditions

Duchenne Muscular Dystrophy

Explore related trials

Duchenne Muscular Dystrophy

NCT03373968

Where you'd take part

This study runs at 39 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Hospital Materno-Infantil

    Barcelona, Spainstudy coordinator listed

    Recruiting

  • Ospedale Pediatrico Bambino Gesù, Malattie Neuromuscolari e Neurodegenerative

    Roma, Italystudy coordinator listed

    Recruiting

  • U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative, Building 16 - ground floor IRCCS Istituto Giannina Gaslini,

    Genova, Italystudy coordinator listed

    Recruiting

  • A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari

    Messina, Italyno site contact published

    Enrolling by invitation

  • Alder Hey Children's Hospital NHS Trust

    Liverpool, UK, United Kingdomno site contact published

    Enrolling by invitation

  • Centro Clinico NeMO Fondazione Serena ONLUS Area SUD

    Milan, Italyno site contact published

    Enrolling by invitation

  • CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest

    Nantes, Franceno site contact published

    Enrolling by invitation

  • Clinic of Neurology and Psychiatry for Children and Youth - Neurology Department Dr. Subotic 6a,

    Belgrade, Serbiano site contact published

    Enrolling by invitation

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Reference Study ID Number: DSC/14/2357/51
[email protected]
+ 39 02 6443 · ext. 2524
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Want this trial checked against your situation?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or \>30%, i.e. included in"off-target" group,
never been randomized because, the enrollment in the off target group was completed. 2. Aged ≥6 years old; 3. Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to localregulations); 4. Subjects must be willing to use adequate contraception:
Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following:
True abstinence (absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the subject.
Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception.
Condom with spermicide and the female partner must use an acceptable method of contraception, such as an oral,
transdermal, injectable or implanted steroid-basedcontraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such asfor example cervical cap with spermicide jelly.

What this trial measures

  • Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]Through study completion, an average of 1 year

    Type, incidence, and severity of treatment related/not related adverse events(AEs) and serious adverse event (SAEs)