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Long-term Givinostat treatment for safety and tolerability in Duchenne muscular dystrophy

Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies

Phase2; Phase3 Interventional Italfarmaco · NCT03373968

This long-term treatment will try Givinostat in people with Duchenne muscular dystrophy who previously took the drug to see if it remains safe and tolerable.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment206 (estimated)
Ages7 Years and up
SexMale
SponsorItalfarmaco Industry-sponsored
Locations39 sites (Sacramento, California and 38 other locations)
Trial IDNCT03373968 on ClinicalTrials.gov

What this trial studies

Participants who previously received Givinostat in a DMD study will take oral Givinostat suspension twice daily, usually at the same dose they finished on in the prior study, with dose adjustments for weight. Blood counts (including platelets) and ECGs will be monitored frequently early on and according to protocol, with predefined dose reductions or permanent stops for specific safety thresholds. The study is open-label and focuses on long-term safety, tolerability, and continued efficacy signals in this pre-treated population. Dosing and monitoring rules are predefined to manage known risks such as thrombocytopenia, QT prolongation, and cytopenias.

Who should consider this trial

Good fit: Ideal candidates are people with Duchenne muscular dystrophy aged 6 or older who completed a prior Givinostat DMD study (or met specified screening criteria), can provide consent/assent, and agree to frequent blood and ECG monitoring and contraception requirements.

Not a fit: People who never received Givinostat before, or who have exclusionary safety issues such as significant blood count abnormalities, severe QT prolongation, or other disqualifying conditions, are unlikely to benefit from joining this long-term program.

Why it matters

Potential benefit: If successful, long-term Givinostat could offer a tolerable disease-modifying option that helps preserve muscle function in people with Duchenne muscular dystrophy.

How similar studies have performed: Earlier phase trials of Givinostat in DMD reported acceptable safety and some promising signs on muscle-related outcomes, so this long-term study follows those initial findings.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in study DSC/14/2357/48 and met:

   * all the inclusion criteria and none of the exclusion criteria,
   * 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.

Exclusion Criteria:

1. Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed;
2. Use of any current investigational drug other than Givinostat;
3. Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results;
4. Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD;
5. Have platelets count, White Blood Cell and Hemoglobin at screening \< Lower Limit of Normal (LLN)\* (for abnormal screening laboratory test results (\<LLN), the platelets count, White Blood Cell and Hemoglobin will be repeated once; if the repeat test result is still \<LLN, then exclusionary);
6. Have Triglycerides \> 300 mg/dL (3.42 mmol/L) in fasting condition at screening visit\* (for abnormal screening laboratory test results (\>300 mg/dL), the triglycerides will be repeated once; if the repeat test result is still \>300 mg/dL, then exclusionary);
7. Have inadequate renal function, as defined by serum Cystatin C \>2 x the upper limit of normal (ULN) at screening visit\*. If the value is \>2 x ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \>2 x ULN, the subject should be excluded);
8. Have heart failure (New York Heart Association Class III or IV)
9. Have a current liver disease or impairment, including but not limited to an elevated total bilirubin\* (i.e. \> 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's;
10. Have a baseline QTcF \>450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome);
11. Have a psychiatric illness/social situation rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures.
12. Have any hypersensitivity to the components of study medication;
13. Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance.

    * the Investigators to evaluate these exclusion criteria can use the laboratory results obtained within 5 months from V1, to allow the continuity of the treatment. It is worth noting, as soon as the site will receive the laboratory results done in screening/baseline (Visit 1) visit they will check the GIVINOSTAT dose and modify it as per protocol safety rules and/or dosage modifications rules.

Where this trial is running

Sacramento, California and 38 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Duchenne Muscular Dystrophy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.