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Duchenne Muscular DystrophyPhase 2/3NCT03373968

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

A Phase 2/3 study testing Givinostat in people with Duchenne Muscular Dystrophy, run by Italfarmaco at 39 sites in 11 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 7 years and older, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
Givinostat
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
206 participants
Where
39 sitesin 11 countries
Study dates
24 Oct 2017to Dec 2029
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 7 years and older
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.
  • Aged ≥6 years old;
  • Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to localregulations);
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  • 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 cannot join

  • 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;
  • Use of any current investigational drug other than Givinostat;
  • 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;
  • Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD;
  • 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);
  • 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);
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  • 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);
  • Have heart failure (New York Heart Association Class III or IV)
  • 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;
  • 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);
  • 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.
  • Have any hypersensitivity to the components of study medication;
  • 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.

Trial locations and contacts

39 sites in 11 countries. Contact the study team or a site near you to ask about taking part.

Reference Study ID Number: DSC/14/2357/51 contact

+ 39 02 6443 ext. 2524

patientadvocacy@italfarmaco.com

39 sites in 11 countries

United States10 sites, 2 recruiting

  • MD Rare Disease Research, LLC

    Atlanta, Georgia, 30318

    Enrolling by invitation

  • Child Health Research Institute

    Gainesville, Florida, 32610

    Completed

  • Connecticut Children's Medical Center, Neurology Division

    Hartford, Connecticut, 06106

    Completed

  • University of Iowa Children's Hospital

    Iowa City, Iowa, 52242

    Enrolling by invitation

  • The Children's Hospital of Philadelphia Colket Translational Research Building

    Philadelphia, Pennsylvania, 19104

    Completed

  • Shriners Hospitals for Children

    Portland, Oregon, 97239

    Completed

  • Virginia Commonwealth University Childrens Hospital of Richmond at

    Richmond, Virginia, 23298

    Completed

  • University of California - Davis Medical Center - Devis Physical Medicine & Rehabilitation

    Sacramento, California, 95817

    Completed

  • Rady Children's Hospital center - UCSD Department of Neuroscience

    San Diego, California, 92123

    Completed

  • Washington University School of Medicine in St Louis Department of Neurology 660 S.Euclid Avenue, Campus Box 8111

    St Louis, Missouri, 63110

    Completed

Other trials in United States

Italy7 sites, 6 recruiting

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

    Genova, 16147

    Claudio Bruno, MD, +39 010 56362 2620, claudio2246@gmail.com

    Claudio Bruno, MD

    Recruiting

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

    Messina, 98125

    Enrolling by invitation

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica

    Milan, 20122

    Active, not recruiting

  • IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133

    Enrolling by invitation

  • Centro Clinico NeMO Fondazione Serena ONLUS Area SUD

    Milan, 20162

    Enrolling by invitation

  • Ospedale Pediatrico Bambino Gesù, Malattie Neuromuscolari e Neurodegenerative

    Roma, 00146

    Enrico Bertini, MD, enricosilvio.bertini@opbg.net

    Enrico Bertini, MD

    Recruiting

  • Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile

    Roma, 00168

    Enrolling by invitation

Other trials in Italy

Spain4 sites, 4 recruiting

  • Hospital Materno-Infantil

    Barcelona, 08035

    Francina Munell, MD, francina.munell@vhir.org

    Francina Munell, MD

    Recruiting

  • Neuromuscular Pathology Unit - Hospital Sant Joan de Déu

    Esplugues de Llobregat, Barcellona, 08950

    Enrolling by invitation

  • Hospital Universitario Virgen del Rocio

    Seville, 41013

    Enrolling by invitation

  • Hospital Universitari i Politècnic La Fe - Servicio Neurologia

    Valencia

    Enrolling by invitation

Other trials in Spain

United Kingdom4 sites, 4 recruiting

  • The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust

    Gobowen, SY10 7AG

    Enrolling by invitation

  • Alder Hey Children's Hospital NHS Trust

    Liverpool, UK, L12 2AP

    Enrolling by invitation

  • UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD

    London, , WC1N 1EH

    Enrolling by invitation

  • The John Walton Muscular Dystrophy Research Centre

    Newcastle upon Tyne, NE1 3BZ

    Enrolling by invitation

Other trials in United Kingdom

Canada3 sites, 3 recruiting

  • Kinsmen Research Centre - Alberta Children's Hospital

    Calgary, Alberta, T3B 6A8

    Enrolling by invitation

  • Holland Bloorview Kids Rehabilitation Hospital

    Toronto, Ontario, M4G1R8

    Enrolling by invitation

  • The University of British Columbia, Children's and Womens Health Centre of BC Branch

    Vancouver, British Columbia, V6H 3V4

    Enrolling by invitation

Other trials in Canada

Germany3 sites, 2 recruiting

  • Universitätsklinikum Essen - Kinder-und Jugendmedizin Neuropadiatrie

    Essen, D-45147

    Enrolling by invitation

  • Klinik- und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum HamburgEppendorf, Martinistr. 52

    Hamburg, 20246

    Enrolling by invitation

  • Klinikum der Universitat Munchen, Campus Innenstadt, Lindwurmstr. 4

    München, 80337

    Active, not recruiting

Other trials in Germany

Belgium2 sites, 2 recruiting

  • University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology

    Leuven, 03000

    Enrolling by invitation

  • Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN)

    Liège, 04000

    Enrolling by invitation

Other trials in Belgium

France2 sites, 2 recruiting

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

    Nantes, 44093

    Enrolling by invitation

  • Hôpital Armand Trousseau I-Motion - Plateforme d'essais cliniques pédiatriques Bâtiment Lemariey - Porte 20 * 2ème étage 26 Avenue du Dr Arnold Nette

    Paris, 760

    Enrolling by invitation

Other trials in France

Netherlands2 sites, 2 recruiting

  • Leiden University Medical Center LUMC, Albinusdreef 2

    Leiden, 2300

    Enrolling by invitation

  • Radboud University Medical Centre

    Nijmegen, 6500

    Enrolling by invitation

Other trials in Netherlands

Israel1 site, 1 recruiting

  • Institute of Neurology - Schneider Children's Medical Center of Israel Kaplan, 14

    Petah Tikva, 4920235

    Enrolling by invitation

Other trials in Israel

Serbia1 site, 1 recruiting

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

    Belgrade, 11000

    Enrolling by invitation

Other trials in Serbia

About this study

Official title: 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

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.

Read the detailed description

GIVINOSTAT oral suspension (10 mg/mL) has to be administered orally as 2 oral doses daily while the subject is in a fed state. The starting dose of GIVINOSTAT in the present long term study will be the same that the subject was receiving at the end of the previous DMD GIVINOSTAT study.

As weight affects GIVINOSTAT exposures, the dosage will be modified based on subject weight according the rules detailed in the study protocol section 11.2.2.1.

In addition, in case a subject will have a consistent (e.g., at least 2 consecutive evaluations) platelets count ≤150 x 10^9/L and not meet the stopping criteria for platelets, the Investigator will have to reduce the dose of 1/3 or 20% less of the current dose as described in the study protocol section 10.5.1.3. During the first month of treatment, platelets count assessment will be performed weekly, while during the second month it will be performed every 2 weeks, in order to strictly monitor this parameter for safety reasons, with the exclusion of subjects coming from study DSC/11/2357/43 for which the first visit will be 4 months after the Visit 1/baseline visit.

Study drug should be permanently interrupted if any of the following occurs:

  • severe drug-related diarrhoea (i.e., increase of ≥7 stools per day);
  • any drug-related SAE;
  • QTcF >500 msec;
  • platelets count ≤50 x 10^9/L;
  • white blood cells ≤2.0 x 10^9/L;
  • hemoglobin ≤8.0 g/dL; To avoid laboratory errors and anomalous values, test must be confirmed with a repeated test performed on the next working day. The treatment should be stopped until the retest result becomes available. If the repeated test is still under the stopping limit value, study drug must be permanently discontinued. If the repeated test is acceptable, the subject can resume treatment.

The Investigator will follow up the patient until resolution or acceptable stabilization of the event occurs and document all the relevant information, as applicable. After the resolution/stabilization of the event, the subject will be withdrawn from the study and the EOS Visit (see Section 12.1.10) will be performed.

Any decision relevant to the dose adjustment and/or modification of schedule of assessments can be discussed with the Medical Monitor, but the final decision remains with the Investigator only or its authorized designee.

Being tested
Givinostat
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GivinostatDrug

    suspension of givinostat (10 mg/mL)

Study groups in NCT03373968
Study groupRoleWhat they receive
givinostatExperimentalGivinostat oral suspension (10 mg/mL) twice daily in a fed state
Receives: Drug: Givinostat

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

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

    Measured: Through study completion, an average of 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Dec 2017
  2. Study start24 Oct 2017
  3. Primary completionDec 2029 (estimated)
  4. Study completionDec 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
206 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03373968
Sponsor's protocol ID
DSC/14/2357/51
Status last verified
Jan 2026
First submitted
4 Dec 2017
Last updated
21 Jan 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • McDonald CM, Guglieri M, Vucinic D, Acsadi G, Brandsema JF, Bruno C, Finanger EL, Harper A, Lobato ML, Masson R, Muelas N, Munell F, Nevo Y, Pereon Y, Phan H, Sansone VA, Scoto M, Willis T, Finkel RS, Vandenborne K, Cazzaniga S, Montrasio S, Alessi F, Bettica P, Mercuri E; Givinostat Study 51 Investigators; Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS) Investigators; ImagingDMD Investigators. Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons. Ann Clin Transl Neurol. 2025 Nov;12(11):2335-2348. doi: 10.1002/acn3.70165. Epub 2025 Aug 19.

    PubMed 40830818

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Givinostat in people with Duchenne Muscular Dystrophy, run by Italfarmaco at 39 sites in 11 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 21 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 39 sites in United States, Italy, Spain, United Kingdom, Canada and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Reference Study ID Number: DSC/14/2357/51 (+ 39 02 6443 ext. 2524, patientadvocacy@italfarmaco.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: Through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03373968, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jan 2026.

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