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Hope for duchenne: Long-Term drug safety trial now recruiting

NCT ID NCT03373968

Disease control Sponsor: Italfarmaco Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at the long-term safety and effects of the drug givinostat in people with Duchenne muscular dystrophy (DMD). It is open to those who have already taken part in a previous givinostat study. The goal is to track side effects and see how well the drug works over time. About 206 participants will be enrolled.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 206 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2017

Expected to finish

Dec 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

7 years and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Duchenne muscular dystrophy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    29 sites in 11 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

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

    ENROLLING_BY_INVITATION

    Messina, 98125, Italy

  • Alder Hey Children's Hospital NHS Trust

    ENROLLING_BY_INVITATION

    Liverpool, UK, L12 2AP, United Kingdom

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

    ENROLLING_BY_INVITATION

    Nantes, 44093, France

  • Centro Clinico NeMO Fondazione Serena ONLUS Area SUD

    ENROLLING_BY_INVITATION

    Milan, 20162, Italy

  • Child Health Research Institute

    COMPLETED

    Gainesville, Florida, 32610, United States

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

    ENROLLING_BY_INVITATION

    Belgrade, 11000, Serbia

  • Connecticut Children's Medical Center, Neurology Division

    COMPLETED

    Hartford, Connecticut, 06106, United States

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

    ACTIVE_NOT_RECRUITING

    Milan, 20122, Italy

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

    ENROLLING_BY_INVITATION

    Roma, 00168, Italy

  • Holland Bloorview Kids Rehabilitation Hospital

    ENROLLING_BY_INVITATION

    Toronto, Ontario, M4G1R8, Canada

  • Hospital Materno-Infantil

    RECRUITING

    Barcelona, 08035, Spain

  • Hospital Universitari i Politècnic La Fe - Servicio Neurologia

    ENROLLING_BY_INVITATION

    Valencia, Spain

  • Hospital Universitario Virgen del Rocio

    ENROLLING_BY_INVITATION

    Seville, 41013, Spain

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

    ENROLLING_BY_INVITATION

    Liège, 04000, Belgium

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

    ENROLLING_BY_INVITATION

    Paris, 760, France

  • IRCCS Istituto Neurologico Carlo Besta

    ENROLLING_BY_INVITATION

    Milan, 20133, Italy

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

    ENROLLING_BY_INVITATION

    Petah Tikva, 4920235, Israel

  • Kinsmen Research Centre - Alberta Children's Hospital

    ENROLLING_BY_INVITATION

    Calgary, Alberta, T3B 6A8, Canada

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

    ENROLLING_BY_INVITATION

    Hamburg, 20246, Germany

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

    ACTIVE_NOT_RECRUITING

    München, 80337, Germany

  • Leiden University Medical Center LUMC, Albinusdreef 2

    ENROLLING_BY_INVITATION

    Leiden, 2300, Netherlands

  • MD Rare Disease Research, LLC

    ENROLLING_BY_INVITATION

    Atlanta, Georgia, 30318, United States

  • Neuromuscular Pathology Unit - Hospital Sant Joan de Déu

    ENROLLING_BY_INVITATION

    Esplugues de Llobregat, Barcellona, 08950, Spain

  • Ospedale Pediatrico Bambino Gesù, Malattie Neuromuscolari e Neurodegenerative

    RECRUITING

    Roma, 00146, Italy

  • Radboud University Medical Centre

    ENROLLING_BY_INVITATION

    Nijmegen, 6500, Netherlands

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

    COMPLETED

    San Diego, California, 92123, United States

  • Shriners Hospitals for Children

    COMPLETED

    Portland, Oregon, 97239, United States

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

    COMPLETED

    Philadelphia, Pennsylvania, 19104, United States

  • The John Walton Muscular Dystrophy Research Centre

    ENROLLING_BY_INVITATION

    Newcastle upon Tyne, NE1 3BZ, United Kingdom

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

    ENROLLING_BY_INVITATION

    Gobowen, SY10 7AG, United Kingdom

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

    ENROLLING_BY_INVITATION

    Vancouver, British Columbia, V6H 3V4, Canada

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

    RECRUITING

    Genova, 16147, Italy

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

    ENROLLING_BY_INVITATION

    London, , WC1N 1EH, United Kingdom

  • University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology

    ENROLLING_BY_INVITATION

    Leuven, 03000, Belgium

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

    COMPLETED

    Sacramento, California, 95817, United States

  • University of Iowa Children's Hospital

    ENROLLING_BY_INVITATION

    Iowa City, Iowa, 52242, United States

  • Universitätsklinikum Essen - Kinder-und Jugendmedizin Neuropadiatrie

    ENROLLING_BY_INVITATION

    Essen, D-45147, Germany

  • Virginia Commonwealth University Childrens Hospital of Richmond at

    COMPLETED

    Richmond, Virginia, 23298, United States

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

    COMPLETED

    St Louis, Missouri, 63110, United States

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Other studies related to the condition(s) this trial covers.