Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
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ClinicalTrials.gov Identifier: NCT02851797 |
Recruitment Status
:
Recruiting
First Posted
: August 2, 2016
Last Update Posted
: April 12, 2018
|
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- No Results Posted
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it is a randomised, double blind, parallel group, placebo controlled study. A total of 213 male ambulant subjects will be randomised 2:1 (givinostat:placebo).
Subjects will be stratified for their concomitant use of steroids in 4 strata:
- Deflazacort daily regimen
- Deflazacort intermittent regimen
- Other steroids daily regimen
- Other steroids intermittent regimen. The study duration is planned for 19 months.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: givinostat Drug: placebo | Phase 3 |
Givinostat or placebo oral suspension (10 mg/mL) will be administered orally as 2 oral doses daily while the subject is in fed state, according to the child's weight.
Study drug should be permanently stopped if any of the following occur:
- severe drug-related diarrhoea;
- any drug-related Serious Adverse Event;
- QTcF >500 msec;
- platelets count ≤50 x 109/L.
Study drug should be temporarily stopped if any of the following occur:
- platelets count <75 x 109/L but >50 x 109/L (the treatment should be temporarily stopped and a platelets count has to be performed and re-tested until platelets will be normalized);
- moderate or severe diarrhoea.
In case the study drug was temporarily stopped, the study drug can be resumed at a level 1/3 smaller than the one at which the Adverse Event leading to temporary stop occurred, once platelets are normalized or diarrhoea is mild (if treatment was stopped for moderate or severe diarrhoea).
Two interim analyses are planned and will be conducted by the IDMC in order to ensure study integrity.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 213 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy |
Actual Study Start Date : | June 1, 2017 |
Estimated Primary Completion Date : | June 2020 |
Estimated Study Completion Date : | June 2020 |

Arm | Intervention/treatment |
---|---|
Active Comparator: givinostat
Givinostat oral suspension (10 mg/mL) twice daily in a fed state
|
Drug: givinostat
suspension of givinostat (10 mg/mL)
|
Placebo Comparator: placebo
Placebo oral suspension (10 mg/mL) twice daily in a fed state
|
Drug: placebo
suspension manufactured to mimic givinostat
|
- mean change in 4 standard stairs climb [ Time Frame: 18 months ]the primary endpoint is the mean change in 4 standard stairs climb test results before and after 18 months of treatment of givinostat versus placebo
- Other functional test as 6MWT [ Time Frame: 18 months ]the mean change in 6MWT
- time to rise from floor [ Time Frame: 18 months ]the mean change in time to rise from floor
- Magnetic Resonance Spetroscopy [ Time Frame: 18 months ]the mean change in fat fraction of vastus lateralis muscles at MRS

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 6 Years to 17 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Are an ambulant male aged ≥6 years at randomisation with DMD characteristic clinical symptoms or signs (e.g., proximal muscle weakness, Gowers' maneuver, elevated serum creatinine kinase level) already present at screening;
- Have DMD diagnosis confirmed by genetic testing;
- Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations);
- Are able to complete 2 Four Stairs Climb test (4SC) screening assessments; the results of these tests must be within ±1 second of each other;
- Have the mean of 2 screening 4SC assessments ≤8 seconds;
- Have time to rise from floor of <10 seconds at screening;
- Have manual muscle testing (MMT) of quadriceps at screening ≥ Grade 3;
- Are eligible according to the protocol-specified functional algorithm* predictive of vastus lateralis muscle fat fraction (VL MFF) that should be in the range >10% but <30% (see section 4.2.3);
-
Have used systemic corticosteroids for a minimum of 6 months immediately prior to the start of study treatment, with no significant change in dosage or dosing regimen (excluding changes related to body weight change) for a minimum of 6 months immediately prior to start of study treatment and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study.
- The protocol-specific functional algorithm will consider results relevant Four stairs climb test and knee extensor peak torque measurement.
Exclusion Criteria:
- Have exposure to another investigational drug within 3 months prior to the start of study treatment;
- Have exposure to idebenone within 3 months prior to the start of study treatment;
- Have exposure to any dystrophin restoration product (e.g., Ataluren, Exon skipping) within 6 months prior to the start of study treatment;
- Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to the start of study treatment (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed;
- Have surgery that might have an effect on muscle strength or function within 3 months before study entry or planned surgery at any time during the study;
- Ankle joint contractures due to a fixed loss of ≥10 degrees of dorsiflexion from plantagrade assuming a normal range of dorsiflexion of 20 degree;
- Change in contracture treatment such as serial casting, contracture control devices, night splints, stretching exercises (passive, active, self) within 3 months prior to enrollment, or expected need for such intervention during the study;
- 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 neurological diseases or presence of relevant somatic disorders that are not related to DMD;
- Have platelets count at screening < Lower Limit of Normal (LLN);
- Have symptomatic cardiomyopathy or heart failure (New York Heart Association Class III or IV) or left ventricular ejection fraction <50% at screening;
- Have a current or history of liver disease or impairment;
- Have inadequate renal function, as defined by serum Cystatin C >2 x the upper limit of normal (ULN);
- Have a positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening;
- Have a baseline QTcF >450 msec, 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 situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures;
-
Have any known allergic reaction to givinostat or any of its excipients.
For the subgroup of subjects who will undergo MRI and MRS (i.e., MR Cohort):
- Have contraindications to MRI or MRS (e.g., claustrophobia, metal implants, or seizure disorder).

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02851797
Contact: Reference Study ID Number EPYDIS (DSC/14/2357/48) | +39 026443 ext 2524 | patientadvocacy@italfarmaco.com |

United States, California | |
University of California - Davis Medical Center - Devis Physical Medicin & Rehabilitation | Recruiting |
Davis, California, United States, 95817 | |
Contact: Erica Goude emgoude@ucdavis.edu | |
Principal Investigator: Craig McDonald, MD | |
David Geffen School of Medicine - UCLA Neurology | Recruiting |
Los Angeles, California, United States, 90095 | |
Contact: Angela Ho ALHo@mednet.ucla.edu | |
Principal Investigator: Perry Shieh, MD | |
Rady Children's Hospital center - UCSD Department of Neuroscience | Recruiting |
San Diego, California, United States, 92123 | |
Contact: Jessica Reit jreit@ucsd.edu | |
Principal Investigator: Carla Grosmann, MD | |
United States, Colorado | |
Children's Hospital Colorado | Recruiting |
Aurora, Colorado, United States, 80045 | |
Contact: Lauri Filar, MD Lauri.Filar@childrenscolorado.org | |
Principal Investigator: Michele Yang, MD | |
United States, Connecticut | |
Connecticut Children's Medical Center - Division Neurology | Recruiting |
Hartford, Connecticut, United States, 06106 | |
Contact: Vikki Palmer VPalmer@connecticutchildrens.org | |
Principal Investigator: Gyula Acsadi, MD | |
United States, Florida | |
Child Health Research Institute - Department of Pediatrics | Recruiting |
Gainesville, Florida, United States, 32610 | |
Contact: Tina Cousins, MD couscm@peds.ufl.edu | |
Principal Investigator: Barry Byrne, MD | |
Nemours Children's Hospital | Recruiting |
Orlando, Florida, United States, 32827 | |
Contact: Virginia Rizzo, MD Virginia.Rizzo@nemours.org | |
Principal Investigator: Richard Finkel, MD | |
United States, Iowa | |
University of Iowa Children's Hospital | Recruiting |
Iowa City, Iowa, United States, 52242 | |
Contact: Corey McDaniel corey-mcdaniel@uiowa.edu | |
Principal Investigator: Katherine Mathews, MD | |
United States, Michigan | |
Wayne State University - University Pediatrics - Children's Hospital of Michigan | Not yet recruiting |
Detroit, Michigan, United States, 48201 | |
Contact: Ahlam Saleh ahsaleh@wayne.edu | |
Principal Investigator: Huiyuan Jiang, MD | |
United States, Minnesota | |
University of Minnesota - Department of Neurology | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Emily Vollbrecht vollb007@umn.edu | |
Principal Investigator: Georgios Manousakis, MD | |
United States, Missouri | |
Washington University School of Medicine in St Louis - Department of Neurology | Recruiting |
Saint Louis, Missouri, United States, 63110 | |
Contact: Alyssa Sonsoucie amsonsoucie@wustl.edu | |
Principal Investigator: Anne Connolly, MD | |
United States, Oregon | |
Shriners Hospitals for Children | Recruiting |
Portland, Oregon, United States, 97239 | |
Contact: Cassandra Black ceb@shcc.org | |
Principal Investigator: Erika Finanger, MD | |
United States, Pennsylvania | |
The Children's Hospital of Philadelphia | Not yet recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
Contact: Khrystine Ford FORDKA@email.chop.edu | |
Principal Investigator: Gihan Tennekoon, MD | |
United States, Virginia | |
Childrens Hospital of Richmond at Virginia Commonwealth University | Recruiting |
Richmond, Virginia, United States, 23298 | |
Contact: Kathryn O'Hara, MD kathryn.ohara@vcuhealth.org | |
Principal Investigator: Amy Harper, MD | |
Belgium | |
University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology | Recruiting |
Leuven, Belgium, 03000 | |
Contact: Nathalie Goemans, MD nathalie.goemans@uzleuven.be | |
Principal Investigator: Nathalie Goemans, MD | |
Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN) | Recruiting |
Liege, Belgium, 04000 | |
Contact: Laurent Servais, MD l.servais@institut-myologie.org | |
Principal Investigator: Laurent Servais, MD | |
Canada, Alberta | |
Alberta Children's Hospital | Recruiting |
Calgary, Alberta, Canada, T3B6A8 | |
Contact: Jean Mah, MD Jean.Mah@albertahealthservices.ca | |
Principal Investigator: Jean Mah, MD | |
Canada, British Columbia | |
The University of British Columbia, Children's and Womens Health Centre of BC Branch | Not yet recruiting |
Vancouver, British Columbia, Canada, V6H 3V4 | |
Contact: Kathryn Selby, MD kselby@cw.bc.ca | |
Principal Investigator: Kathryn Selby, MD | |
Canada, Ontario | |
Holland Bloorview Kids Rehabilitation Hospital | Not yet recruiting |
Toronto, Ontario, Canada, M4G1R8 | |
Contact: Gloria Lee glee@hollandbloorview.ca | |
Principal Investigator: Laura McAdam, MD | |
France | |
CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest | Recruiting |
Nantes, France, 44093 | |
Contact: Yann Pereon, MD Yann.Pereon@univ-nantes.fr | |
Principal Investigator: Yann Pereon, MD | |
Hopital Armand Trousseau I-Motion, Plateforme d'essais cliniques pédiatriques | Recruiting |
Paris, France, 75012 | |
Contact: Teresa Gidaro, MD t.gidaro@institut-myologie.org | |
Principal Investigator: Teresa Gidaro, MD | |
Germany | |
Universitatsklinikum Essen - Kinder und Jugendmedizin Neuropadiatrie | Recruiting |
Essen, Germany, 45122 | |
Contact: Ulrike Schara, MD ulrike.schara@uk-essen.de | |
Principal Investigator: Ulrike Schara, MD | |
Klinik un Policlinik fur Kinder und Jugendmedizin - Universitatsklinikum Hamburg Eppendorf | Recruiting |
Hamburg, Germany, 20246 | |
Contact: Jessika Johannssen, MD jjohannsen@uke.de | |
Principal Investigator: Jessika Johannssen, MD | |
Klinikum der Uniersitat Munchen - Campus Innenstadt | Recruiting |
Munchen, Germany, 80337 | |
Contact: Astrid Blaschek, MD Astrid.Blaschek@med.uni-muenchen.de | |
Principal Investigator: Wolfgang Muller-Felber, MD | |
Italy | |
IRCCS Istituto G.Gaslini, U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative | Recruiting |
Genova, Italy, 16147 | |
Contact: Claudio Bruno, MD claudiobruno@ospedale-gaslini.ge.it | |
Principal Investigator: Claudio Bruno, MD | |
A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari | Recruiting |
Messina, Italy, 98125 | |
Contact: Giuseppe Vita, MD vitag@unime.it | |
Principal Investigator: Giuseppe Vita, MD | |
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica | Recruiting |
Milano, Italy, 20122 | |
Contact: Giacomo Comi, MD giacomo.comi@unimi.it | |
Principal Investigator: Giacomo Comi, MD | |
Fondazione IRCCS Istituto Neurologico Carlo Besta | Recruiting |
Milano, Italy, 20133 | |
Contact: Giovanni Baranello, MD Giovanni.Baranello@istituto-besta.it | |
Principal Investigator: Giovanni Baranello, MD | |
Ospedale Pediatrico Bambin Gesù, Malattie Neuromuscolari e Neurodegenerative | Recruiting |
Roma, Italy, 00146 | |
Contact: Enrico Bertini, MD enricosilvio.bertini@opbg.net | |
Principal Investigator: Enrico Bertini, MD | |
Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile | Recruiting |
Roma, Italy, 00168 | |
Contact: Eugenio Mercuri, MD eumercuri@gmail.com | |
Principal Investigator: Eugenio Mercuri, MD | |
Netherlands | |
Leiden University Medical Center LUMC | Recruiting |
Leiden, Netherlands, ZH 2300 RC | |
Contact: Martha Mosselman, MD M.H.Mosselman@lumc.nl | |
Principal Investigator: Erik Niks, MD | |
Radboud University Medical Centre | Recruiting |
Nijmegen, Netherlands, 6500 | |
Contact: Imelda de Groot, MD Imelda.deGroot@radboudumc.nl | |
Principal Investigator: Imelda de Groot, MD | |
Spain | |
Hospital Sant Joan de Déu - Neuromuscular Pathology Unit | Recruiting |
Esplugues de Llobregat, Barcelona, Spain, 08950 | |
Contact: Andres Nascimento, MD anascimento@hsjdbcn.org | |
Principal Investigator: Andres Nascimento, MD | |
Hospital Materno-Infantil - Passeig de la Vall d'Hebron | Recruiting |
Barcelona, Spain, 08035 | |
Contact: Francina Munell, MD francina.munell@vhir.org | |
Principal Investigator: Francina Munell, MD | |
Hospital 12 de Octubre - Hospital Materno Infantil | Recruiting |
Madrid, Spain, 28041 | |
Contact: Ana Camacho Salas, MD acamacho@salud.madrid.org | |
Principal Investigator: Ana Camacho Salas, MD | |
Hospital Universitari i Politécnic de la Fe - Servicio Neurologia | Recruiting |
Valencia, Spain, 46026 | |
Contact: Juan Jesus Vilchez, MD vilchez_jje@gva.es | |
Principal Investigator: Juan Jesus Vilchez, MD | |
United Kingdom | |
Alder Hey Children's Hospital NHS Trust | Recruiting |
Liverpool, United Kingdom, L12 2AP | |
Contact NMR@alderhey.nhs.uk | |
Principal Investigator: Stefan Spinty, MD | |
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD | Not yet recruiting |
London, United Kingdom, EC1N 1EH | |
Contact: Mariacristina Scoto, MD m.scoto@ucl.ac.uk | |
Principal Investigator: Mariacristina Scoto, MD | |
The John Walton Muscular Dystrophy Research Centre - Freeman Hospital - Newcastle University - Institute of Genetic Medicine | Recruiting |
Newcastle upon Tyne, United Kingdom, NE1 3BZ | |
Contact: May Tiet may.tiet@newcastle.ac.uk | |
Principal Investigator: Volker Straub, MD | |
The Robert Jones and Agnes Hunt Orthopaedic Hospital - NHS Foundation Trust | Recruiting |
Oswestry, United Kingdom, SY 10 7AG | |
Contact: Sarah Turner, MD Sarah.Turner2@rjah.nhs.uk | |
Principal Investigator: Tracey Willis, MD |
Responsible Party: | Italfarmaco |
ClinicalTrials.gov Identifier: | NCT02851797 History of Changes |
Other Study ID Numbers: |
EPYDIS (DSC/14/2357/48) |
First Posted: | August 2, 2016 Key Record Dates |
Last Update Posted: | April 12, 2018 |
Last Verified: | April 2018 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | Yes | |
Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |