Last updated on July 2018

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

Brief description of study

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:

  1. Deflazacort daily regimen
  2. Deflazacort intermittent regimen
  3. Other steroids daily regimen
  4. Other steroids intermittent regimen. The study duration is planned for 19 months.

Detailed Study Description

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.

Clinical Study Identifier: NCT02851797

Contact Investigators or Research Sites near you

Start Over

Craig McDonald, MD

University of California - Davis Medical Center - Devis Physical Medicin & Rehabilitation
Davis, CA United States
  Connect »

Perry Shieh, MD

David Geffen School of Medicine - UCLA Neurology
Los Angeles, CA United States
  Connect »

Carla Grosmann, MD

Rady Children's Hospital center - UCSD Department of Neuroscience
San Diego, CA United States
  Connect »

Michele Yang, MD

Children's Hospital Colorado
Aurora, CO United States
  Connect »

Gyula Acsadi, MD

Connecticut Children's Medical Center - Division Neurology
Hartford, CT United States
  Connect »

Barry Byrne, MD

Child Health Research Institute - Department of Pediatrics
Gainesville, FL United States
  Connect »

Richard Finkel, MD

Nemours Children's Hospital
Orlando, FL United States
  Connect »

Katherine Mathews, MD

University of Iowa Children's Hospital
Iowa City, IA United States
  Connect »

Reference Study ID Number EPYDIS ...

Wayne State University - University Pediatrics - Children's Hospital of Michigan
Detroit, MI United States
  Connect »

Georgios Manousakis, MD

University of Minnesota - Department of Neurology
Minneapolis, MN United States
  Connect »

Anne Connolly, MD

Washington University School of Medicine in St Louis - Department of Neurology
Saint Louis, MO United States
  Connect »

Erika Finanger, MD

Shriners Hospitals for Children
Portland, OR United States
  Connect »

Gihan Tennekoon, MD

The Children's Hospital of Philadelphia
Philadelphia, PA United States
  Connect »

Amy Harper, MD

Childrens Hospital of Richmond at Virginia Commonwealth University
Richmond, VA United States
  Connect »

Nathalie Goemans, MD

University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology
Leuven, Belgium
  Connect »

Laurent Servais, MD

Hospital de La Citadelle, Centre de R f rence des Maladies Neuromuscolaires (CRMN)
Liege, Belgium
  Connect »

Jean Mah, MD

Alberta Children's Hospital
Calgary, AB Canada
  Connect »

Kathryn Selby, MD

The University of British Columbia, Children's and Womens Health Centre of BC Branch
Vancouver, BC Canada
  Connect »

Laura McAdam, MD

Holland Bloorview Kids Rehabilitation Hospital
Toronto, ON Canada
  Connect »

Yann Pereon, MD

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

Teresa Gidaro, MD

Hopital Armand Trousseau I-Motion, Plateforme d'essais cliniques p diatriques
Paris, France
  Connect »

Ulrike Schara, MD

Universitatsklinikum Essen - Kinder und Jugendmedizin Neuropadiatrie
Essen, Germany
  Connect »

Jessika Johannssen, MD

Klinik un Policlinik fur Kinder und Jugendmedizin - Universitatsklinikum Hamburg Eppendorf
Hamburg, Germany
  Connect »

Wolfgang Muller-Felber, MD

Klinikum der Uniersitat Munchen - Campus Innenstadt
Munchen, Germany
  Connect »

Claudio Bruno, MD

IRCCS Istituto G.Gaslini, U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative
Genova, Italy
  Connect »

Giuseppe Vita, MD

A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari
Messina, Italy
  Connect »

Giacomo Comi, MD

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

Giovanni Baranello, MD

Fondazione IRCCS Istituto Neurologico Carlo Besta
Milano, Italy
  Connect »

Enrico Bertini, MD

Ospedale Pediatrico Bambin Ges , Malattie Neuromuscolari e Neurodegenerative
Roma, Italy
  Connect »

Eugenio Mercuri, MD

Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile
Roma, Italy
  Connect »

Erik Niks, MD

Leiden University Medical Center LUMC
Leiden, Netherlands
  Connect »

Imelda de Groot, MD

Radboud University Medical Centre
Nijmegen, Netherlands
  Connect »

Francina Munell, MD

Hospital Materno-Infantil - Passeig de la Vall d'Hebron
Barcelona, Spain
  Connect »

Ana Camacho Salas, MD

Hospital 12 de Octubre - Hospital Materno Infantil
Madrid, Spain
  Connect »

Juan Jesus Vilchez, MD

Hospital Universitari i Polit cnic de la Fe - Servicio Neurologia
Valencia, Spain
  Connect »

Stefan Spinty, MD

Alder Hey Children's Hospital NHS Trust
Liverpool, United Kingdom
  Connect »

Mariacristina Scoto, MD

UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD
London, United Kingdom
  Connect »

Volker Straub, MD

The John Walton Muscular Dystrophy Research Centre - Freeman Hospital - Newcastle University - Institute of Genetic Medicine
Newcastle upon Tyne, United Kingdom
  Connect »

Tracey Willis, MD

The Robert Jones and Agnes Hunt Orthopaedic Hospital - NHS Foundation Trust
Oswestry, United Kingdom
  Connect »

Andres Nascimento, MD

Hospital Sant Joan de D u - Neuromuscular Pathology Unit
Esplugues de Llobregat, Spain
  Connect »