Last updated on August 2017

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

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Craig McDonald, MD

University of California - Davis Medical Center - Devis Physical Medicin & Rehabilitation
Davis, CA United States
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Perry Shieh, MD

David Geffen School of Medicine - UCLA Neurology
Los Angeles, CA United States
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Carla Grosmann, MD

Rady Children's Hospital center - UCSD Department of Neuroscience
San Diego, CA United States
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Michele Yang, MD

Children's Hospital Colorado
Aurora, CO United States
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Gyula Acsadi, MD

Connecticut Children's Medical Center - Division Neurology
Hartford, CT United States
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Barry Byrne, MD

Child Health Research Institute - Department of Pediatrics
Gainesville, FL United States
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Richard Finkel, MD

Nemours Children's Hospital
Orlando, FL United States
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Katherine Mathews, MD

University of Iowa Children's Hospital
Iowa City, IA United States
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Huiyuan Jiang, MD

Wayne State University - University Pediatrics - Children's Hospital of Michigan
Detroit, MI United States
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Peter Manousakis, MD

University of Minnesota - Department of Neurology
Minneapolis, MN United States
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Anne Connolly, MD

Washington University School of Medicine in St Louis - Department of Neurology
Saint Louis, MO United States
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Erika Finanger, MD

Shriners Hospitals for Children
Portland, OR United States
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Gihan Tennekoon, MD

The Children's Hospital of Philadelphia
Philadelphia, PA United States
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Jean Harper, MD

Childrens Hospital of Richmond at Virginia Commonwealth University
Richmond, VA United States
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Nathalie Goemans, MD

University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology
Leuven, Belgium
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Laurent Servais, MD

Hospital de La Citadelle, Centre de R f rence des Maladies Neuromuscolaires (CRMN)
Liege, Belgium
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Jean Mah, MD

Alberta Children's Hospital
Calgary, AB Canada
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Kathryn Selby, MD

The University of British Columbia, Children's and Womens Health Centre of BC Branch
Vancouver, BC Canada
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Laura McAdam, MD

Holland Bloorview Kids Rehabilitation Hospital
Toronto, ON Canada
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Yann Pereon, MD

CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest
Nantes, France
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Teresa Gidaro, MD

Hopital Armand Trousseau I-Motion, Plateforme d'essais cliniques p diatriques
Paris, France
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Ulrike Schara, MD

Universitatsklinikum Essen - Kinder und Jugendmedizin Neuropadiatrie
Essen, Germany
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Jessika Johanssen, MD

Klinik un Policlinik fur Kinder und Jugendmedizin - Universitatsklinikum Hamburg Eppendorf
Hamburg, Germany
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Wolfgang Muller-Felber, MD

Klinikum der Uniersitat Munchen - Campus Innenstadt
Munchen, Germany
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Claudio Bruno, MD

IRCCS Istituto G.Gaslini, U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative
Genova, Italy
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Giuseppe Vita, MD

A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari
Messina, Italy
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Giacomo Comi, MD

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica
Milano, Italy
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Giovanni Baranello, MD

Fondazione IRCCS Istituto Neurologico Carlo Besta
Milano, Italy
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Enrico Bertini, MD

Ospedale Pediatrico Bambin Ges , Malattie Neuromuscolari e Neurodegenerative
Roma, Italy
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Eugenio Mercuri, MD

Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile
Roma, Italy
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Erik Niks, MD

Leiden University Medical Center LUMC
Leiden, Netherlands
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Imelda de Groot, MD

Radboud University Medical Centre
Nijmegen, Netherlands
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Andres Nascimento, MD

Hospital Sant Joan de D u - Neuromuscular Pathology Unit
Esplugas de Llobregat, Spain
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Francina Munell, MD

Hospital Materno-Infantil - Passeig de la Vall d'Hebron
Barcelona, Spain
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Ana Camacho Salas, MD

Hospital 12 de Octubre - Hospital Materno Infantil
Madrid, Spain
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Juan Jesus Vilchez, MD

Hospital Universitari i Polit cnic de la Fe - Servicio Neurologia
Valencia, Spain
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Stefan Spinty, MD

Alder Hey Children's Hospital NHS Trust
Liverpool, United Kingdom
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Francesco Muntoni, MD

UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD
London, United Kingdom
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Michela Guglieri, MD

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

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