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

NCT03373968 | Recruiting Phase 2 FDA Drug

• 1 other identifier: DSC/14/2357/51
• Study Type: interventional
• Target: 206
• Locations: 11 countries
• Sites: 39

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (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)

  Through study completion, an average of 1 year

Study Arms (1)

givinostat

EXPERIMENTAL

Givinostat oral suspension (10 mg/mL) twice daily in a fed state

Drug: Givinostat

Interventions

Givinostat DRUG

suspension of givinostat (10 mg/mL)

givinostat

Eligibility Criteria

• Age: 7 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:

You may not qualify if:

• 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);
• 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.
• 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 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);

Sponsors & Collaborators

• Italfarmaco: lead
• Cromsource: collaborator

Study Sites (39)

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

Sacramento, California, 95817, United States

COMPLETED

Rady Children's Hospital center - UCSD Department of Neuroscience

San Diego, California, 92123, United States

COMPLETED

Connecticut Children's Medical Center, Neurology Division

Hartford, Connecticut, 06106, United States

COMPLETED

Child Health Research Institute

Gainesville, Florida, 32610, United States

COMPLETED

MD Rare Disease Research, LLC

Atlanta, Georgia, 30318, United States

ENROLLING BY INVITATION

University of Iowa Children's Hospital

Iowa City, Iowa, 52242, United States

ENROLLING BY INVITATION

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

St Louis, Missouri, 63110, United States

COMPLETED

Shriners Hospitals for Children

Portland, Oregon, 97239, United States

COMPLETED

The Children's Hospital of Philadelphia Colket Translational Research Building

Philadelphia, Pennsylvania, 19104, United States

COMPLETED

Virginia Commonwealth University Childrens Hospital of Richmond at

Richmond, Virginia, 23298, United States

COMPLETED

University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology

Leuven, 03000, Belgium

ENROLLING BY INVITATION

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

Liège, 04000, Belgium

ENROLLING BY INVITATION

Kinsmen Research Centre - Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada

ENROLLING BY INVITATION

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

Vancouver, British Columbia, V6H 3V4, Canada

ENROLLING BY INVITATION

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, M4G1R8, Canada

ENROLLING BY INVITATION

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

Nantes, 44093, France

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, France

ENROLLING BY INVITATION

Universitätsklinikum Essen - Kinder-und Jugendmedizin Neuropadiatrie

Essen, D-45147, Germany

ENROLLING BY INVITATION

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

Hamburg, 20246, Germany

ENROLLING BY INVITATION

Klinikum der Universitat Munchen, Campus Innenstadt, Lindwurmstr. 4

München, 80337, Germany

ACTIVE NOT RECRUITING

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

Petah Tikva, 4920235, Israel

ENROLLING BY INVITATION

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

Genova, 16147, Italy

RECRUITING

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

Messina, 98125, Italy

ENROLLING BY INVITATION

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

Milan, 20122, Italy

ACTIVE NOT RECRUITING

IRCCS Istituto Neurologico Carlo Besta

Milan, 20133, Italy

ENROLLING BY INVITATION

Centro Clinico NeMO Fondazione Serena ONLUS Area SUD

Milan, 20162, Italy

ENROLLING BY INVITATION

Ospedale Pediatrico Bambino Gesù, Malattie Neuromuscolari e Neurodegenerative

Roma, 00146, Italy

RECRUITING

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

Roma, 00168, Italy

ENROLLING BY INVITATION

Leiden University Medical Center LUMC, Albinusdreef 2

Leiden, 2300, Netherlands

ENROLLING BY INVITATION

Radboud University Medical Centre

Nijmegen, 6500, Netherlands

ENROLLING BY INVITATION

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

Belgrade, 11000, Serbia

ENROLLING BY INVITATION

Neuromuscular Pathology Unit - Hospital Sant Joan de Déu

Esplugues de Llobregat, Barcellona, 08950, Spain

ENROLLING BY INVITATION

Hospital Materno-Infantil

Barcelona, 08035, Spain

RECRUITING

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

ENROLLING BY INVITATION

Hospital Universitari i Politècnic La Fe - Servicio Neurologia

Valencia, Spain

ENROLLING BY INVITATION

Alder Hey Children's Hospital NHS Trust

Liverpool, UK, L12 2AP, United Kingdom

ENROLLING BY INVITATION

The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust

Gobowen, SY10 7AG, United Kingdom

ENROLLING BY INVITATION

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

London, , WC1N 1EH, United Kingdom

ENROLLING BY INVITATION

The John Walton Muscular Dystrophy Research Centre

Newcastle upon Tyne, NE1 3BZ, United Kingdom

ENROLLING BY INVITATION

Related Publications (1)

• 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.

  PMID: 40830818 DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

givinostat

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

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

CONTACT

+ 39 02 6443; patientadvocacy@italfarmaco.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2017
• First Posted: December 14, 2017
• Study Start: October 24, 2017
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: January 21, 2026

Record last verified: 2026-01

Locations

SE (1); CA (3); IT (7); US (10); NL (2); GB (4); DE (3); IL (1); ES (4); BE (2); FR (2)