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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 2 / Phase 3INTERVENTIONAL

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

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

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study (NCT03373968) is a Phase 2 / Phase 3 interventional studying Duchenne Muscular Dystrophy, sponsored by Italfarmaco. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Duchenne Muscular Dystrophy and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 206 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Duchenne Muscular Dystrophy subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

Who May Qualify: 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. Who Should NOT Join This Trial: 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; ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

Givinostat

suspension of givinostat (10 mg/mL)

Locations (20)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

University of California - Davis Medical Center - Devis Physical Medicine & Rehabilitation
Sacramento, California, United States
Rady Children's Hospital center - UCSD Department of Neuroscience
San Diego, California, United States
Connecticut Children's Medical Center, Neurology Division
Hartford, Connecticut, United States
Child Health Research Institute
Gainesville, Florida, United States
MD Rare Disease Research, LLC
Atlanta, Georgia, United States
University of Iowa Children's Hospital
Iowa City, Iowa, United States
Washington University School of Medicine in St Louis Department of Neurology 660 S.Euclid Avenue, Campus Box 8111
St Louis, Missouri, United States
Shriners Hospitals for Children
Portland, Oregon, United States
The Children's Hospital of Philadelphia Colket Translational Research Building
Philadelphia, Pennsylvania, United States
Virginia Commonwealth University Childrens Hospital of Richmond at
Richmond, Virginia, United States
University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology
Leuven, Belgium
Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN)
Liège, Belgium
Kinsmen Research Centre - Alberta Children's Hospital
Calgary, Alberta, Canada
The University of British Columbia, Children's and Womens Health Centre of BC Branch
Vancouver, British Columbia, Canada
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, Canada
CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest
Nantes, France
Hôpital Armand Trousseau I-Motion - Plateforme d'essais cliniques pédiatriques Bâtiment Lemariey - Porte 20 * 2ème étage 26 Avenue du Dr Arnold Nette
Paris, France
Universitätsklinikum Essen - Kinder-und Jugendmedizin Neuropadiatrie
Essen, Germany
Klinik- und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum HamburgEppendorf, Martinistr. 52
Hamburg, Germany
Klinikum der Universitat Munchen, Campus Innenstadt, Lindwurmstr. 4
München, Germany

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT03373968), the sponsor (Italfarmaco), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT03373968 clinical trial studying?

This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT03373968?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT03373968?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT03373968. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT03373968. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.